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Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa

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ClinicalTrials.gov Identifier: NCT01288027
Recruitment Status : Completed
First Posted : February 2, 2011
Results First Posted : December 2, 2014
Last Update Posted : December 19, 2014
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Pompe Disease (Late-Onset)
Glycogen Storage Disease Type II (GSD II)
Glycogenesis 2 Acid Maltase Deficiency
Intervention Biological: Alglucosidase Alfa
Enrollment 16
Recruitment Details The study was conducted at 11 centers between July 06, 2011 and December 19, 2013.
Pre-assignment Details  
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
Period Title: Overall Study
Started 16
Full Analysis Set (FAS) 16 [1]
Completed 16
Not Completed 0
[1]
All participants who received at least 1 complete infusion of alglucosidase alfa.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
Overall Number of Baseline Participants 16
Hide Baseline Analysis Population Description
FAS population included all participants who received at least one complete infusion of alglucosidase alfa.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 16 participants
51.6  (13.69)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants
Female
9
  56.3%
Male
7
  43.8%
1.Primary Outcome
Title Change From Baseline in Tissue Glycogen Content in Quadriceps Muscle Biopsy Samples at Week 26
Hide Description Tissue glycogen content was measured by quadriceps biopsies as ‘percent area of tissue occupied by glycogen'.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
FAS population included all participants who received at least one complete infusion of alglucosidase alfa. Here, n = number of participants with both Baseline and Week 26 assessment of tissue glycogen content.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
Overall Number of Participants Analyzed 16
Mean (Standard Deviation)
Unit of Measure: percent area occupied by glycogen
Baseline (n=14) 5.3  (4.59)
Change at Week 26 (n=13) -1.6  (4.11)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Alglucosidase Alfa
Comments Statistical significance for change from Baseline was measured using one sample t-test
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1860
Comments [Not Specified]
Method one sample t-test
Comments [Not Specified]
2.Secondary Outcome
Title Glycogen Distribution
Hide Description [Not Specified]
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
No quantitative data could be reported for this outcome as the assessment was qualitative in nature.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
3.Secondary Outcome
Title Muscle Fiber Morphology
Hide Description [Not Specified]
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
No quantitative data could be reported for this outcome as the assessment was qualitative in nature.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
4.Secondary Outcome
Title Lysosomal Inclusions
Hide Description [Not Specified]
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
No quantitative data could be reported for this outcome as the assessment was qualitative in nature.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
5.Secondary Outcome
Title Percent Change From Baseline in Muscle Involvement Using Mercuri Scoring at Week 26
Hide Description Muscle involvement was assessed by T1-weighted magnetic resonance imaging (MRI). T1-weighted MRI data was analyzed using the Mercuri scoring in both legs (Total score = 1-4; where 1=Normal appearance, 2=Mild involvement, 3=Moderate involvement, and 4=Severe involvement). For each participants, the average for each the upper (thigh) and lower leg was computed for Mercuri grading.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
FAS population included all participants who received at least one complete infusion of alglucosidase alfa. Here, Number of participants analyzed= participants with both Baseline and Week 26 assessment of muscle involvement, n= number of participants with both Baseline and Week 26 assessment of muscle involvement for specified category.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
Overall Number of Participants Analyzed 14
Mean (Standard Deviation)
Unit of Measure: percent change
Mercuri Scoring - Upper Leg (n=13) 2.6  (9.24)
Mercuri Scoring - Lower Leg (n=14) 0.0  (0.0)
6.Secondary Outcome
Title Percent Change From Baseline in Degree of Fatty Infiltration Using 3-Point 3-Dimensional (3D) Dixon at Week 26
Hide Description Degree of Fatty Infiltration was assessed by 3-point 3D Dixon acquisition using skeletal muscle MRI in a subset of participants.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
FAS population included all participants who received at least one complete infusion of alglucosidase alfa. Here, number of participants analyzed = number of participants with both Baseline and Week 26 assessment of degree of fatty infiltration.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
Overall Number of Participants Analyzed 3
Mean (Standard Deviation)
Unit of Measure: percent change
2.0  (12.83)
7.Secondary Outcome
Title Percent Change From Baseline in Disease Activity Using T2 Magnetic Resonance Imaging (MRI) at Week 26
Hide Description Disease activity (inflammation and/or water content within muscles) was quantitatively assessed by T2 MRI values in a subset of participants. A T2 MRI value of greater than (>) 39 millisecond (ms) was defined as abnormal. T2 estimation normally requires an additional acquisition for computing the B1 spatial deviation however, can still be estimated if this acquisition is missing.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
FAS population included all participants who received at least one complete infusion of alglucosidase alfa. Here Number of participants analyzed = number of participants with both baseline and Week 26 assessment of disease activity.
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description:
Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: percent change
T2 with B1 8.1  (14.19)
T2 without B1 7.2  (13.58)
Time Frame From signature of the informed consent form up to the final visit (Week 26)
Adverse Event Reporting Description In the event a single participant has experienced both serious and non-serious form of the same adverse event (AE), individual has been included in numerator of both AE tables. Analysis was performed on the safety population: all participant who received any amount of alglucosidase alfa. AEs are listed independent of relationship to treatment.
 
Arm/Group Title Alglucosidase Alfa
Hide Arm/Group Description Alglucosidase alfa intravenous infusion 20 milligram per kilogram (mg/kg) every other week for 24 weeks.
All-Cause Mortality
Alglucosidase Alfa
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Alglucosidase Alfa
Affected / at Risk (%)
Total   1/16 (6.25%) 
Eye disorders   
Retinal vascular thrombosis  1  1/16 (6.25%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 16.1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Alglucosidase Alfa
Affected / at Risk (%)
Total   15/16 (93.75%) 
Cardiac disorders   
Bradycardia  1  1/16 (6.25%) 
Ear and labyrinth disorders   
Deafness  1  1/16 (6.25%) 
Tympanic membrane perforation  1  1/16 (6.25%) 
Eye disorders   
Ocular hyperaemia  1  1/16 (6.25%) 
Gastrointestinal disorders   
Abdominal discomfort  1  1/16 (6.25%) 
Diarrhoea  1  2/16 (12.50%) 
Nausea  1  1/16 (6.25%) 
Vomiting  1  1/16 (6.25%) 
General disorders   
Adverse drug reaction  1  1/16 (6.25%) 
Fatigue  1  2/16 (12.50%) 
Feeling cold  1  1/16 (6.25%) 
Influenza like illness  1  1/16 (6.25%) 
Injection site extravasation  1  1/16 (6.25%) 
Malaise  1  1/16 (6.25%) 
Infections and infestations   
Bronchitis  1  2/16 (12.50%) 
Ear infection  1  1/16 (6.25%) 
Nasopharyngitis  1  5/16 (31.25%) 
Rhinitis  1  1/16 (6.25%) 
Subcutaneous abscess  1  1/16 (6.25%) 
Upper respiratory tract infection  1  2/16 (12.50%) 
Injury, poisoning and procedural complications   
Arthropod bite  1  1/16 (6.25%) 
Ligament sprain  1  1/16 (6.25%) 
Procedural pain  1  3/16 (18.75%) 
Investigations   
Heart rate increased  1  1/16 (6.25%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  2/16 (12.50%) 
Back pain  1  2/16 (12.50%) 
Muscle spasms  1  1/16 (6.25%) 
Myalgia  1  1/16 (6.25%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Melanocytic naevus  1  1/16 (6.25%) 
Nervous system disorders   
Dizziness  1  3/16 (18.75%) 
Headache  1  4/16 (25.00%) 
Muscle contractions involuntary  1  1/16 (6.25%) 
Paraesthesia  1  1/16 (6.25%) 
Sciatica  1  1/16 (6.25%) 
Psychiatric disorders   
Insomnia  1  2/16 (12.50%) 
Reproductive system and breast disorders   
Epididymitis  1  1/16 (6.25%) 
Respiratory, thoracic and mediastinal disorders   
Dyspnoea  1  1/16 (6.25%) 
Nasal congestion  1  1/16 (6.25%) 
Oropharyngeal pain  1  2/16 (12.50%) 
Pharyngeal oedema  1  1/16 (6.25%) 
Tachypnoea  1  1/16 (6.25%) 
Skin and subcutaneous tissue disorders   
Dry skin  1  1/16 (6.25%) 
Rash  1  2/16 (12.50%) 
Urticaria  1  1/16 (6.25%) 
Vascular disorders   
Flushing  1  2/16 (12.50%) 
Hypotension  1  1/16 (6.25%) 
Peripheral coldness  1  1/16 (6.25%) 
Thrombosis  1  1/16 (6.25%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 16.1
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.
If no publication has occurred within 12 months of the completion of the study, the Investigator shall have the right to publish/present independently the results of the study. The Investigator shall provide the Sponsor with a copy of any such presentation/publication for comment at least 30 days before any presentation/submission for publication. If requested by the Sponsor, any presentation/submission shall be delayed up to 90 days, to allow the Sponsor to preserve its proprietary rights.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Trial Transparency Team
Organization: Sanofi
EMail: Contact-us@sanofi.com
Layout table for additonal information
Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT01288027     History of Changes
Other Study ID Numbers: AGLU07310
2010-020611-36 ( EudraCT Number )
MSC12823 ( Other Identifier: Sanofi )
First Submitted: January 27, 2011
First Posted: February 2, 2011
Results First Submitted: November 24, 2014
Results First Posted: December 2, 2014
Last Update Posted: December 19, 2014