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A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa (Mini-COMET)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03019406
Recruitment Status : Active, not recruiting
First Posted : January 12, 2017
Results First Posted : November 22, 2021
Last Update Posted : May 25, 2022
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Glycogen Storage Disease Type II-Pompe's Disease
Interventions Drug: Avalglucosidase alfa (GZ402666)
Drug: Alglucosidase alfa (GZ419829)
Enrollment 22
Recruitment Details The study was conducted at 10 active centers in 5 countries. Total of 22 participants were screened between 12 October 2017 and 03 April 2019. None of the participants were screen failed. Participants were assigned to treatment by using interactive response technology system.
Pre-assignment Details Participants in Cohorts 1 received avalglucosidase alfa 20 milligrams per kilogram (mg/kg) every other week (qow) and participants in Cohort 2 received avalglucosidase alfa 40 mg/kg qow. No randomization process was applied for Cohort 1 and 2. Participants in Cohort 3 were randomized (stratified by gender) to receive either avalglucosidase alfa 40 mg/kg qow (3a) or their current stable alglucosidase alfa dose regimen (3b). Data reported based on primary completion date, i.e., 30 September 2019.
Arm/Group Title Cohort 1: Avalglucosidase Alfa 20 mg/kg Cohort 2: Avalglucosidase Alfa 40 mg/kg Cohort 3a: Avalglucosidase Alfa 40 mg//kg Cohort 3b: Alglucosidase Alfa in PAP
Hide Arm/Group Description Avalglucosidase alfa, 20 mg/kg intravenous (IV) infusion qow for 25 weeks in the Primary Analysis Period (PAP), followed by same treatment from Week 26 up to Week 371 in extension treatment period (ETP). Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in the PAP, followed by same treatment from Week 26 up to Week 371 in ETP. After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP, followed by same treatment from Week 26 up to Week 371 in ETP. After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP. After PAP, participants received avalglucosidase alfa 40mg/kg IV infusion qow from Week 26 up to Week 371 in ETP.
Period Title: PAP: up to 25 Weeks
Started 6 5 5 6
Treated 6 5 5 6
Completed 6 5 5 6
Not Completed 0 0 0 0
Period Title: ETP: Ongoing From Week 26
Started 6 5 5 3 [1]
Completed 0 0 0 0
Not Completed 6 5 5 3
Reason Not Completed
Ongoing             6             5             5             3
[1]
Participants who completed PAP and accepted to receive avalglucosidase alfa treatment in ETP.
Arm/Group Title Cohort 1: Avalglucosidase Alfa 20 mg/kg Cohort 2: Avalglucosidase Alfa 40 mg/kg Cohort 3a: Avalglucosidase Alfa 40 mg//kg Cohort 3b: Alglucosidase Alfa in PAP Total
Hide Arm/Group Description Avalglucosidase alfa, 20 mg/kg IV infusion qow for 25 weeks in the PAP, followed by same treatment from Week 26 up to Week 371 in extension treatment period (ETP). Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in the PAP, followed by same treatment from Week 26 up to Week 371 in ETP. After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP, followed by same treatment from Week 26 up to Week 371 in ETP. After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP. After PAP, participants received avalglucosidase alfa 40mg/kg IV infusion qow from Week 26 up to Week 371 in ETP. Total of all reporting groups
Overall Number of Baseline Participants 6 5 5 6 22
Hide Baseline Analysis Population Description
Analysis was performed on safety population which included participants who had received at least 1 infusion (partial or total) and were analyzed according to treatment received.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 6 participants 5 participants 5 participants 6 participants 22 participants
7.6  (3.4) 8.1  (4.1) 6.9  (2.7) 4.7  (3.2) 6.8  (3.4)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 5 participants 5 participants 6 participants 22 participants
Female
1
  16.7%
2
  40.0%
3
  60.0%
4
  66.7%
10
  45.5%
Male
5
  83.3%
3
  60.0%
2
  40.0%
2
  33.3%
12
  54.5%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 5 participants 5 participants 6 participants 22 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
3
  50.0%
3
  60.0%
2
  40.0%
0
   0.0%
8
  36.4%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
2
  33.3%
2
   9.1%
White
3
  50.0%
2
  40.0%
3
  60.0%
4
  66.7%
12
  54.5%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI)
Hide Description Adverse event (AE): any untoward medical occurrence in participant received study drug & did not necessarily had to have causal relationship with treatment. TEAEs: AEs developed/worsened in grade/become serious during PAP period (from the time of 1st study drug dose up to Week 25). Serious AE(SAE): any untoward medical occurrence at any dose resulted in death, was life-threatening, required inpatient hospitalization, prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was congenital anomaly/birth defect or was medically important event. TEAEs included SAEs & non-SAEs. AESI:AE (serious/non-serious) of scientific & medical concern specific to Sponsor's product/program, for which ongoing monitoring & immediate notification by Investigator to Sponsor required.
Time Frame From Baseline to Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3b: Alglucosidase Alfa
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 5 6
Measure Type: Count of Participants
Unit of Measure: Participants
Any TEAEs
5
  83.3%
5
 100.0%
5
 100.0%
5
  83.3%
Any Serious TEAEs
1
  16.7%
3
  60.0%
0
   0.0%
2
  33.3%
Any AESI
0
   0.0%
2
  40.0%
1
  20.0%
1
  16.7%
2.Primary Outcome
Title PAP: Number of Participants With Infusion-associated Reactions (IARs)
Hide Description IARs were defined as AESIs that occurred during either the infusion or the observation period following the infusion which were deemed to be related or possibly related to the study drug. Protocol-defined IARs: An AESIs that occurred during either the infusion or the observation period following the infusion which were deemed to be related or possibly related to study drug. Algorithm-defined IARs: any TEAE meeting either 1 or 2 criteria: 1) event occurred from the start of infusion to the end of infusion plus 24 hours, and considered related to study drug, 2) If an AE time component was missing, compared AE Start date with infusion start date and infusion end date. If an AE Start date was between infusion start date and infusion end date plus one day, and it was related to study drug.
Time Frame From Baseline to Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3b: Alglucosidase Alfa
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 5 6
Measure Type: Count of Participants
Unit of Measure: Participants
Protocol-defined IARs
0
   0.0%
2
  40.0%
1
  20.0%
1
  16.7%
Algorithm-defined IARs
0
   0.0%
2
  40.0%
1
  20.0%
1
  16.7%
3.Secondary Outcome
Title PAP: Number of Participants With Anti-drug Antibody (ADA) Response
Hide Description Anti-drug antibody response was categorized as: Treatment induced ADAs: ADAs developed de novo (seroconversion) following administration of study drug. Treatment boosted ADAs: pre-existing ADAs that were boosted at least two titer steps from Baseline (i.e., 4-fold increase in titers) followed by administration of study drug.
Time Frame From Baseline to Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on the ADA evaluable population which included participants who had received at least 1 infusion (partial or total) and had at least one ADA sample taken post-baseline after drug administration that was appropriate for ADA testing with a reportable result.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3b: Alglucosidase Alfa
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 5 6
Measure Type: Count of Participants
Unit of Measure: Participants
Treatment boosted ADA response 0 0 1 2
Treatment induced ADA response 0 1 3 1
4.Secondary Outcome
Title PAP: Pharmacokinetic (PK) Parameter: Maximum Observed Plasma Concentration (Cmax) of Avalglucosidase Alfa
Hide Description Cmax is the maximum observed plasma concentration.
Time Frame Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 and 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on PK population which included PAP participants who had received at least 1 infusion (partial or total) and had evaluable drug concentration data. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 5
Mean (Standard Deviation)
Unit of Measure: nanograms per milliliter
Week 1 Number Analyzed 5 participants 4 participants 4 participants
189000  (56700) 403000  (171000) 250000  (45100)
Week 25 Number Analyzed 5 participants 5 participants 5 participants
175000  (65900) 297000  (60100) 356000  (84700)
5.Secondary Outcome
Title PAP: Pharmacokinetic Parameter: Time to Achieve Maximum Observed Plasma Concentration (Tmax) of Avalglucosidase Alfa
Hide Description Tmax is the time to achieve maximum plasma concentration.
Time Frame Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 and 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on PK population. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 5
Median (Full Range)
Unit of Measure: hours
Week 1 Number Analyzed 5 participants 4 participants 4 participants
4.43
(3.90 to 5.33)
7.00
(6.00 to 7.25)
6.83
(6.65 to 7.22)
Week 25 Number Analyzed 5 participants 5 participants 5 participants
3.97
(3.77 to 4.75)
7.13
(5.67 to 7.98)
6.87
(5.03 to 7.43)
6.Secondary Outcome
Title PAP: Pharmacokinetic Parameter: Area Under the Plasma Concentration Versus Time Curve From Time 0 to the Time of Last Quantifiable Concentration (AUC0-last) of Avalglucosidase Alfa
Hide Description AUC0-last is the area under the plasma concentration versus time curve from time 0 to the time of last quantifiable concentration.
Time Frame Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 & 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on PK population. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 5
Mean (Standard Deviation)
Unit of Measure: nanograms*hours per milliliter
Week 1 Number Analyzed 5 participants 4 participants 4 participants
923000  (352000) 2630000  (972000) 1720000  (255000)
Week 25 Number Analyzed 5 participants 5 participants 5 participants
805000  (295000) 1930000  (348000) 2200000  (533000)
7.Secondary Outcome
Title PAP: Pharmacokinetic Parameter: Terminal Half-life (t1/2) of Avalglucosidase Alfa
Hide Description t1/2 is the time required for the plasma concentration of a drug to decrease by half of its initial concentration.
Time Frame Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 & 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on PK population. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 5
Mean (Standard Deviation)
Unit of Measure: hours
Week 1 Number Analyzed 5 participants 4 participants 4 participants
0.703  (0.291) 1.15  (0.523) 0.806  (0.248)
Week 25 Number Analyzed 5 participants 5 participants 5 participants
0.601  (0.256) 1.04  (0.248) 1.19  (0.472)
8.Secondary Outcome
Title PAP: Pharmacokinetic Parameter: Clearance (CL) of Avalglucosidase Alfa
Hide Description CL is defined as a quantitative measure of the rate at which a drug substance is removed from the body.
Time Frame Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 and 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on PK population. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 5
Mean (Standard Deviation)
Unit of Measure: milliliters per hours
Week 1 Number Analyzed 5 participants 4 participants 4 participants
673  (222) 562  (152) 529  (150)
Week 25 Number Analyzed 5 participants 5 participants 5 participants
696  (203) 683  (345) 526  (125)
9.Secondary Outcome
Title PAP: Pharmacokinetic Parameter: Volume of Distribution at Steady State (Vss) of Avalglucosidase Alfa
Hide Description Steady state volume of distribution (Vss) is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug.
Time Frame Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 & 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on PK population. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 5
Mean (Standard Deviation)
Unit of Measure: milliliters
Week 1 Number Analyzed 5 participants 4 participants 4 participants
3550  (927) 4500  (882) 4300  (1420)
Week 25 Number Analyzed 5 participants 5 participants 5 participants
3520  (1180) 5350  (2270) 4020  (1390)
10.Secondary Outcome
Title PAP: Change From Baseline in Gross Motor Function Measure-88 (GMFM-88) Test Scores at Week 25
Hide Description GMFM-88 was developed specifically to detect quantitative changes in gross motor function. The GMFM-88 consisted of 88 items organized into 5 dimensions; lying and rolling (17 items); sitting (20 items); crawling and kneeling (14 items); standing (13 items) and walking, running and jumping (24 items). Each item was scored on a 4-point Likert scale with scores range: 0= cannot do; 1 = initiates less then [<] 10 percentage [%] of the task; 2 = partially completes [10% to <100% of the task] and 3 = task completion. The score for each dimension was expressed as a % of the maximum score for that dimension.Total percentage score was obtained by adding the percentage scores for each dimension and dividing the sum by the total number of dimensions. Total scores ranged from 0% to 100%; where higher scores indicated better motor functions.
Time Frame Baseline, Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population. Here, 'overall number of participants analyzed' = participants with available data for this OM.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3b: Alglucosidase Alfa
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 4 6
Mean (Standard Deviation)
Unit of Measure: percentage of total score
6.50  (22.24) 9.80  (13.99) 11.00  (10.80) 17.00  (8.44)
11.Secondary Outcome
Title PAP: Number of Participants in Gross Motor Function Classification System-Expanded and Revised (GMFCS-E and R) Scores at Baseline and Week 25
Hide Description GMFCS-E&R was a 5 level classification system for specific age ranges; observations were performed on 5 levels based on self-initiated movement, with emphasis on sitting, transfers, and mobility: Level I (walks without limitations), Level II (walks with limitations), Level III (walks using a hand-held mobility device), Level IV (self-mobility with limitations; may use powered mobility) and level V (transported in a manual wheel chair) (I to V). The distinctions between levels were based on functional limitations, the need for assistive mobility devices, and to a much lesser extent, quality of movement, and were designed to be meaningful in daily life. The lower level represented good motor functioning and higher level represented low motor functioning. Number of participants in each level of classification at Baseline and Week 25 were reported.
Time Frame Baseline, Week 25
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Hide Analysis Population Description
Analysis was performed on safety population.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3b: Alglucosidase Alfa
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 5 6
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline: Level I
1
  16.7%
1
  20.0%
2
  40.0%
3
  50.0%
Baseline: Level II
1
  16.7%
2
  40.0%
1
  20.0%
0
   0.0%
Baseline: Level III
1
  16.7%
0
   0.0%
1
  20.0%
1
  16.7%
Baseline: Level IV
2
  33.3%
2
  40.0%
0
   0.0%
1
  16.7%
Baseline: Level V
1
  16.7%
0
   0.0%
1
  20.0%
1
  16.7%
Week 25: Level I
1
  16.7%
1
  20.0%
2
  40.0%
3
  50.0%
Week 25: Level II
1
  16.7%
2
  40.0%
1
  20.0%
0
   0.0%
Week 25: Level III
2
  33.3%
1
  20.0%
0
   0.0%
1
  16.7%
Week 25: Level IV
1
  16.7%
1
  20.0%
1
  20.0%
1
  16.7%
Week 25: Level V
1
  16.7%
0
   0.0%
1
  20.0%
1
  16.7%
12.Secondary Outcome
Title PAP: Change From Baseline in Pompe Pediatric Evaluation of Disability Inventory (Pompe-PEDI) Functional Skills Scale: Mobility Domain Test Score-Scaled Score at Week 25
Hide Description Pompe-PEDI: disease specific version to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. It comprised of Functional Skills Scale and Caregiver Assistance Scale; both scales had 3 domains: Self Care, Mobility, and Social Function. Mobility domain was used to measure change in mobility due to changes in muscle strength; consisted of 160 mobility items for participant/legal guardian. The total number of mobility items the child was capable of, was converted to a scaled score with a range of 0 to 100, where scores near "0" indicated low capability and scores near "100" indicated high capability), where higher score was indicative of greater functional ability. Scaled scores were used to interpret individual function and progress over time.
Time Frame Baseline, Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population. Here, 'overall number of participants analyzed' = participants with available data for this OM.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3b: Alglucosidase Alfa
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 3 6
Mean (Standard Deviation)
Unit of Measure: score on a scale
6.19  (10.55) 2.12  (4.04) 2.60  (1.72) 5.20  (5.95)
13.Secondary Outcome
Title PAP: Change From Baseline in Quick Motor Function Test (QMFT) Scores at Week 25
Hide Description QMFT was observer administered test comprised of 16 items specifically difficult for participants with Pompe disease. Each item was scored on 5-point ordinal scale ranged from 0 to 4 (higher score indicated better outcome). Total QMFT score was obtained by adding the scores of all items and ranged from 0 (unable to perform motor function tests) to 64 (normal muscle function), where higher score indicated better outcome/greater motor function.
Time Frame Baseline, Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population. Here, 'overall number of participants analyzed' = participants with available data for this OM.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3b: Alglucosidase Alfa
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 4 6
Mean (Standard Deviation)
Unit of Measure: score on a scale
-0.17  (4.45) 3.20  (4.55) 4.25  (3.30) 5.17  (4.54)
14.Secondary Outcome
Title PAP: Echo-Left Ventricular Mass Z-Score (LVM Z-score) M-mode at Baseline, Week 25, and Change From Baseline at Week 25
Hide Description Cardiac function was evaluated using LVM Z-score as assessed by echocardiogram in M-mode. Z-Scores indicated the number of standard deviations (SD) from the mean in a normal distribution. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. A negative change from Baseline indicated a decrease and positive change from Baseline indicated an increase in LVM Z-score. In this OM, absolute scores at Baseline and Week 25 along with Change from Baseline at Week 25 in LVM Z-score were reported.
Time Frame Baseline, Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3b: Alglucosidase Alfa
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 5 6
Mean (Standard Deviation)
Unit of Measure: Z-score
Baseline Number Analyzed 6 participants 3 participants 5 participants 4 participants
-1.10  (1.07) 0.13  (2.39) -0.78  (0.70) -0.43  (0.92)
Week 25 Number Analyzed 5 participants 4 participants 5 participants 4 participants
-1.74  (2.01) -1.05  (2.61) -1.36  (0.99) 0.15  (1.07)
Change from Baseline at Week 25 Number Analyzed 5 participants 2 participants 5 participants 3 participants
-0.60  (2.16) -0.60  (0.71) -0.58  (0.76) 0.47  (1.76)
15.Secondary Outcome
Title PAP: Change From Baseline in Eyelid Position Measurements: Interpalpebral Fissure Distance (IPFD) - Left Non-Flash and Right Non-Flash at Week 25
Hide Description IPFD is the widest vertical distance (in millimeters) between the upper eyelid and the lower eyelid when the participant is looking in "primary gaze" (i.e. normal gaze when looking straight forward). Images were taken while the participants was wearing a pair of empty eyeglass frames with millimeters rulers attached as a standardized tool to measure eyelid position without camera flash. A negative change from Baseline indicated a decrease and positive change from Baseline indicated an increase in measured distance.
Time Frame Baseline, Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3b: Alglucosidase Alfa
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 5 6
Mean (Standard Deviation)
Unit of Measure: millimeters
Left non-Flash -0.67  (1.54) 1.30  (1.48) 1.30  (0.76) -0.50  (0.77)
Right non-Flash -0.92  (1.66) 0.70  (1.25) 0.90  (1.14) -0.25  (1.13)
16.Secondary Outcome
Title PAP: Change From Baseline in Eyelid Position Measurements: Left and Right Margin Reflex Distance (MRD) at Week 25
Hide Description The MRD is the vertical distance (in millimeters) between the light reflex and the upper eyelid when the participant was looking in "primary gaze" while fixating on a light source. Images were taken while the participants was wearing a pair of empty eyeglass frames with millimeters rulers attached as a standardized tool to measure eyelid position. A negative change from Baseline indicated a decrease and positive change from Baseline indicated an increase in measured distance.
Time Frame Baseline, Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3b: Alglucosidase Alfa
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 5 6
Mean (Standard Deviation)
Unit of Measure: millimeters
Left MRD Number Analyzed 6 participants 4 participants 4 participants 6 participants
-0.08  (1.53) -0.38  (0.85) 0.88  (1.11) 0.08  (0.74)
Right MRD Number Analyzed 6 participants 5 participants 4 participants 6 participants
-0.50  (1.61) -0.50  (1.27) 0.63  (1.03) -0.08  (0.74)
17.Secondary Outcome
Title PAP: Change From Baseline in Eyelid Position Measurements Assessed by Margin Pupil Distance (MPD) - Left Non-Flash and Right Non-Flash at Week 25
Hide Description The MPD is the vertical distance (in millimeters) between the center of the pupil and the upper eyelid margin. Images were taken while the participants was wearing a pair of empty eyeglass frames with millimeters rulers attached as a standardized tool to measure eyelid position without camera flash. A negative change from Baseline indicated a decrease and positive change from Baseline indicated an increase in measured distance.
Time Frame Baseline, Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3b: Alglucosidase Alfa
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 5 6
Mean (Standard Deviation)
Unit of Measure: millimeters
MPD Left Non-Flash Number Analyzed 6 participants 4 participants 5 participants 6 participants
-0.83  (1.44) 0.75  (1.19) 0.40  (0.42) -0.50  (0.32)
MPD Right Non-Flash Number Analyzed 6 participants 5 participants 5 participants 6 participants
-0.25  (1.41) 0.50  (1.06) 0.30  (0.91) 0.00  (0.71)
18.Secondary Outcome
Title PAP: Change From Baseline in Creatine Kinase Value at Week 25
Hide Description Change from Baseline in Creatine kinase value (to assess muscle damage) at Week 25 were reported in this OM.
Time Frame Baseline, Week 25
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population.
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3b: Alglucosidase Alfa
Hide Arm/Group Description:
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Overall Number of Participants Analyzed 6 5 5 6
Mean (Standard Deviation)
Unit of Measure: international units per liters
-208.67  (382.34) -476.60  (467.20) -421.40  (413.39) -1.33  (51.20)
19.Secondary Outcome
Title ETP: Number of Participants With Treatment Emergent Adverse Events, Serious Treatment Emergent Adverse Events, and Adverse Event of Special Interest
Hide Description Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (December 2025).
Time Frame From Week 26 to Week 371
Outcome Measure Data Not Reported
20.Secondary Outcome
Title ETP: Number of Participants With Infusion-associated Reactions
Hide Description Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (December 2025).
Time Frame From Week 26 to Week 371
Outcome Measure Data Not Reported
Time Frame From Day 1 to Week 25 in PAP and from Week 26 to 102 in ETP. Safety data were planned to be collected and analyzed for pooled population of participants who received avalglucosidase alfa 40 mg/kg (Cohorts 2 and 3a of PAP) in ETP. AE data collection is still ongoing and will be updated upon study completion.
Adverse Event Reporting Description TEAEs: AEs that developed/worsened in grade/become serious during treatment epoch (for PAP:time just prior to 1st study drug dose in ETP or 4 weeks after last infusion for those who didn't get into ETP or 2 weeks after last infusion in PAP if participant enrolled in another study/received ERT) (for ETP:time from 1st administration of study drug in ETP to last administration of study drug +4 weeks /2 weeks if participant got enrolled in another study/received ERT). Analyzed on safety population.
 
Arm/Group Title PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3b: Alglucosidase Alfa ETP: Avalglucosidase Alfa 20 mg/kg Pooled Arm ETP: Avalglucosidase Alfa 40 mg/kg Alglucosidase Alfa in PAP Then Avalglucosidase Alfa 40 mg/kg in ETP
Hide Arm/Group Description Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP. Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP. After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP. After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP. Included all participants of Cohort 1 who received avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP, followed by same treatment from Week 26 up to Week 371 in ETP. Pooled arm included all participants of Cohorts 2 and 3a who received avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP, followed by same treatment from Week 26 up to Week 371 in ETP. Included all participants of Cohort 3b who received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in the PAP and after PAP, switched to receive avalglucosidase alfa 40 mg/kg IV infusion qow from Week 26 up to Week 371 in ETP.
All-Cause Mortality
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3b: Alglucosidase Alfa ETP: Avalglucosidase Alfa 20 mg/kg Pooled Arm ETP: Avalglucosidase Alfa 40 mg/kg Alglucosidase Alfa in PAP Then Avalglucosidase Alfa 40 mg/kg in ETP
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/6 (0.00%)      0/5 (0.00%)      0/5 (0.00%)      0/6 (0.00%)      0/6 (0.00%)      0/10 (0.00%)      0/3 (0.00%)    
Hide Serious Adverse Events
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3b: Alglucosidase Alfa ETP: Avalglucosidase Alfa 20 mg/kg Pooled Arm ETP: Avalglucosidase Alfa 40 mg/kg Alglucosidase Alfa in PAP Then Avalglucosidase Alfa 40 mg/kg in ETP
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/6 (16.67%)      3/5 (60.00%)      0/5 (0.00%)      2/6 (33.33%)      4/6 (66.67%)      2/10 (20.00%)      1/3 (33.33%)    
Cardiac disorders               
Atrial Thrombosis † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Ear and labyrinth disorders               
Tympanic Membrane Perforation † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Eye disorders               
Eyelid Ptosis † 1  0/6 (0.00%)  0 2/5 (40.00%)  3 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Strabismus † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
General disorders               
Pyrexia † 1  0/6 (0.00%)  0 1/5 (20.00%)  3 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  3 0/3 (0.00%)  0
Infections and infestations               
Gastroenteritis † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  2 0/3 (0.00%)  0
Influenza † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Lung Infection Pseudomonal † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Otitis Media † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Pneumonia † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 2/6 (33.33%)  2 1/10 (10.00%)  1 0/3 (0.00%)  0
Respiratory Tract Infection Viral † 1  1/6 (16.67%)  2 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Urinary Tract Infection † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Injury, poisoning and procedural complications               
Femur Fracture † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 1/3 (33.33%)  1
Joint Dislocation † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Post Procedural Haemorrhage † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Product Issues               
Device Malfunction † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Respiratory, thoracic and mediastinal disorders               
Adenoidal Hypertrophy † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Lung Consolidation † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Respiratory Distress † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Tonsillar Hypertrophy † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
1
Term from vocabulary, MedDRA 22.0
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg PAP: Cohort 3b: Alglucosidase Alfa ETP: Avalglucosidase Alfa 20 mg/kg Pooled Arm ETP: Avalglucosidase Alfa 40 mg/kg Alglucosidase Alfa in PAP Then Avalglucosidase Alfa 40 mg/kg in ETP
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   5/6 (83.33%)      5/5 (100.00%)      5/5 (100.00%)      4/6 (66.67%)      6/6 (100.00%)      7/10 (70.00%)      3/3 (100.00%)    
Blood and lymphatic system disorders               
Lymph Node Pain † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Neutropenia † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Cardiac disorders               
Pericardial Effusion † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Ear and labyrinth disorders               
Cerumen Impaction † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Deafness † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Ear Pain † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Excessive Cerumen Production † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 1/6 (16.67%)  3 0/10 (0.00%)  0 0/3 (0.00%)  0
Middle Ear Effusion † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Otorrhoea † 1  1/6 (16.67%)  2 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Eye disorders               
Conjunctivochalasis † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Eye Discharge † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Eye Irritation † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 2/5 (40.00%)  2 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Keratopathy † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
Lacrimation Increased † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Gastrointestinal disorders               
Abdominal Hernia † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Abdominal Pain † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 1/5 (20.00%)  1 0/6 (0.00%)  0 1/6 (16.67%)  1 1/10 (10.00%)  1 0/3 (0.00%)  0
Aphthous Ulcer † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Constipation † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Dental Caries † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Diarrhoea † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 2/5 (40.00%)  2 0/6 (0.00%)  0 1/6 (16.67%)  4 1/10 (10.00%)  3 0/3 (0.00%)  0
Dysphagia † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
Faeces Soft † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Loose Tooth † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Mucous Stools † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Nausea † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 1/6 (16.67%)  1 1/6 (16.67%)  6 0/10 (0.00%)  0 0/3 (0.00%)  0
Regurgitation † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Teething † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Toothache † 1  1/6 (16.67%)  1 1/5 (20.00%)  5 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
Vomiting † 1  0/6 (0.00%)  0 1/5 (20.00%)  3 2/5 (40.00%)  2 3/6 (50.00%)  3 1/6 (16.67%)  3 2/10 (20.00%)  3 0/3 (0.00%)  0
General disorders               
Asthenia † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Catheter Site Oedema † 1  0/6 (0.00%)  0 1/5 (20.00%)  2 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Device Related Thrombosis † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Fatigue † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Feeling Hot † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Infusion Site Swelling † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
Injection Site Pain † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Malaise † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Oedema Peripheral † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Pyrexia † 1  2/6 (33.33%)  4 1/5 (20.00%)  4 2/5 (40.00%)  2 1/6 (16.67%)  1 2/6 (33.33%)  9 2/10 (20.00%)  3 0/3 (0.00%)  0
Infections and infestations               
Bronchitis † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 2/6 (33.33%)  3 0/10 (0.00%)  0 0/3 (0.00%)  0
Bronchitis Viral † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
Conjunctivitis † 1  1/6 (16.67%)  2 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Enterovirus Infection † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Fungal Skin Infection † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 1/3 (33.33%)  1
Gastroenteritis † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Hand-Foot-And-Mouth Disease † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Influenza † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Lower Respiratory Tract Infection † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Lung Infection † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 1/3 (33.33%)  1
Lung Infection Pseudomonal † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Nail Infection † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 1/3 (33.33%)  1
Nasopharyngitis † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
Otitis Externa † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Otitis Media † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 2/6 (33.33%)  2 0/10 (0.00%)  0 1/3 (33.33%)  1
Parainfluenzae Virus Infection † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Pneumonia † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Pneumonia Pseudomonal † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Rhinitis † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Sepsis † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
Stoma Site Infection † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Tinea Pedis † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
Tonsillitis † 1  1/6 (16.67%)  2 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Upper Respiratory Tract Infection † 1  2/6 (33.33%)  5 0/5 (0.00%)  0 2/5 (40.00%)  3 1/6 (16.67%)  1 1/6 (16.67%)  3 1/10 (10.00%)  1 0/3 (0.00%)  0
Urinary Tract Infection † 1  2/6 (33.33%)  2 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
Urinary Tract Infection Bacterial † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Viral Infection † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 2/6 (33.33%)  2 0/6 (0.00%)  0 0/10 (0.00%)  0 1/3 (33.33%)  1
Viral Rash † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Injury, poisoning and procedural complications               
Arthropod Bite † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Arthropod Sting † 1  0/6 (0.00%)  0 1/5 (20.00%)  2 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  3 0/3 (0.00%)  0
Contusion † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
Craniocerebral Injury † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Fall † 1  2/6 (33.33%)  2 1/5 (20.00%)  2 0/5 (0.00%)  0 0/6 (0.00%)  0 2/6 (33.33%)  2 2/10 (20.00%)  2 1/3 (33.33%)  1
Ligament Sprain † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Muscle Strain † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Procedural Pain † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Skin Abrasion † 1  0/6 (0.00%)  0 1/5 (20.00%)  3 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Sunburn † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Thermal Burn † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Vascular Access Complication † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 1/3 (33.33%)  1
Wound † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Investigations               
Alpha-1 Acid Glycoprotein Increased † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Faecal Calprotectin Increased † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Gamma-Glutamyltransferase Increased † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Haemoglobin Decreased † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Heart Rate Increased † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Musculoskeletal and connective tissue disorders               
Arthralgia † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Back Pain † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  2 1/10 (10.00%)  1 0/3 (0.00%)  0
Foot Deformity † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
Musculoskeletal Chest Pain † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Musculoskeletal Pain † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Myalgia † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Osteopenia † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 1/3 (33.33%)  1
Pain In Extremity † 1  1/6 (16.67%)  3 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 1/3 (33.33%)  1
Nervous system disorders               
Headache † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 2/5 (40.00%)  2 0/6 (0.00%)  0 1/6 (16.67%)  14 1/10 (10.00%)  1 0/3 (0.00%)  0
Presyncope † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Seizure † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
Product Issues               
Device Occlusion † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 2/5 (40.00%)  2 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Psychiatric disorders               
Enuresis † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Nightmare † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
Staring † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Renal and urinary disorders               
Dysuria † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Haematuria † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Neurogenic Bladder † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Reproductive system and breast disorders               
Genital Erosion † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Respiratory, thoracic and mediastinal disorders               
Asthma † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  3 0/10 (0.00%)  0 0/3 (0.00%)  0
Atelectasis † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Bronchiectasis † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Cough † 1  1/6 (16.67%)  1 1/5 (20.00%)  1 2/5 (40.00%)  4 0/6 (0.00%)  0 1/6 (16.67%)  13 0/10 (0.00%)  0 0/3 (0.00%)  0
Epistaxis † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  2 2/10 (20.00%)  3 0/3 (0.00%)  0
Increased Upper Airway Secretion † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Laryngeal Oedema † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
Lower Respiratory Tract Congestion † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Nasal Congestion † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Oropharyngeal Pain † 1  0/6 (0.00%)  0 2/5 (40.00%)  3 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Productive Cough † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  3 0/10 (0.00%)  0 0/3 (0.00%)  0
Pulmonary Pain † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Respiratory Tract Congestion † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Rhinorrhoea † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 2/5 (40.00%)  2 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Tachypnoea † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  1 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
Tonsillar Hypertrophy † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 1/10 (10.00%)  1 0/3 (0.00%)  0
Upper Respiratory Tract Congestion † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 1/3 (33.33%)  1
Skin and subcutaneous tissue disorders               
Alopecia Areata † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Blister † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 1/3 (33.33%)  1
Dermatitis Atopic † 1  1/6 (16.67%)  1 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Dermatitis Contact † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 1/5 (20.00%)  2 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Dermatitis Diaper † 1  0/6 (0.00%)  0 1/5 (20.00%)  3 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  2 0/10 (0.00%)  0 0/3 (0.00%)  0
Eczema † 1  1/6 (16.67%)  2 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  2 0/10 (0.00%)  0 0/3 (0.00%)  0
Erythema † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Intertrigo † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 0/6 (0.00%)  0 1/6 (16.67%)  1 0/10 (0.00%)  0 0/3 (0.00%)  0
Pruritus † 1  0/6 (0.00%)  0 0/5 (0.00%)  0 0/5 (0.00%)  0 1/6 (16.67%)  1 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
Rash † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 2/5 (40.00%)  3 1/6 (16.67%)  2 1/6 (16.67%)  1 2/10 (20.00%)  3 1/3 (33.33%)  1
Urticaria † 1  0/6 (0.00%)  0 1/5 (20.00%)  1 0/5 (0.00%)  0 0/6 (0.00%)  0 0/6 (0.00%)  0 0/10 (0.00%)  0 0/3 (0.00%)  0
1
Term from vocabulary, MedDRA 22.0
†
Indicates events were collected by systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Trial Transparency Team
Organization: Genzyme, a Sanofi Company
Phone: 800-633-1610 ext 6#
EMail: Contact-US@sanofi.com
Layout table for additonal information
Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT03019406    
Other Study ID Numbers: ACT14132
2016-003475-21 ( EudraCT Number )
U1111-1179-4616 ( Other Identifier: UTN )
First Submitted: December 20, 2016
First Posted: January 12, 2017
Results First Submitted: August 24, 2021
Results First Posted: November 22, 2021
Last Update Posted: May 25, 2022