Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Drug-drug Interaction Study

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: NCT01380743
Recruitment Status : Completed
First Posted : June 27, 2011
Results First Posted : August 17, 2018
Last Update Posted : October 2, 2018
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Pompe Disease
Interventions Drug: duvoglustat
Drug: rhGAA
Enrollment 25
Recruitment Details  
Pre-assignment Details A total of 27 participants were enrolled; 25 participants completed the study. Two participants were discontinued from the study prior to assignment to a cohort: 1 participant voluntarily withdrew before receiving study drug; 1 participant was screened twice and enrolled once, and was counted twice under the total number enrolled.
Arm/Group Title Cohort 1, Duvoglustat 50 mg + rhGAA Cohort 2, Duvoglustat 100 mg + rhGAA Cohort 3, Duvoglustat 250 mg + rhGAA Cohort 4, Duvoglustat 600 mg + rhGAA
Hide Arm/Group Description During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 50 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion. During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 100 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion. During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 250 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion. During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 600 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
Period Title: Overall Study
Started 6 6 6 7
Received at Least 1 Dose of Study Drug 6 6 6 7
Completed 6 6 6 7
Not Completed 0 0 0 0
Arm/Group Title Cohort 1, Duvoglustat 50 mg + rhGAA Cohort 2, Duvoglustat 100 mg + rhGAA Cohort 3, Duvoglustat 250 mg + rhGAA Cohort 4, Duvoglustat 600 mg + rhGAA Total
Hide Arm/Group Description During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 50 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion. During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 100 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion. During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 250 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion. During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 600 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion. Total of all reporting groups
Overall Number of Baseline Participants 6 6 6 7 25
Hide Baseline Analysis Population Description
All participants who were enrolled and received at least 1 dose of rhGAA or duvoglustat.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 6 participants 6 participants 6 participants 7 participants 25 participants
47.7  (5.35) 52.8  (5.71) 50.0  (12.90) 40.0  (6.61) 47.3  (9.13)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 6 participants 6 participants 7 participants 25 participants
Female
3
  50.0%
3
  50.0%
4
  66.7%
2
  28.6%
12
  48.0%
Male
3
  50.0%
3
  50.0%
2
  33.3%
5
  71.4%
13
  52.0%
1.Primary Outcome
Title Number Of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs)
Hide Description

A TEAE was defined as an adverse event (AE) with an onset date on or after the first dose of investigational medicinal product (IMP), or an AE with an onset date before the first dose date that worsened in severity after the first dose date. A severe AE was defined as an AE that was incapacitating and required medical intervention. The number of participants who experienced 1 or more severe TEAEs after dosing on Day 1 up to Day 60 (includes end of study follow-up period) is reported.

A summary of serious and all other non-serious AEs, regardless of causality, is located in the Reported Adverse Events module.

Time Frame Day 1 after dosing up to Day 60
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Population included all participants who were enrolled and received at least 1 dose of rhGAA or duvoglustat.
Arm/Group Title Cohort 1, Duvoglustat 50 mg + rhGAA Cohort 2, Duvoglustat 100 mg + rhGAA Cohort 3, Duvoglustat 250 mg + rhGAA Cohort 4, Duvoglustat 600 mg + rhGAA
Hide Arm/Group Description:
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 50 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 100 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 250 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 600 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
Overall Number of Participants Analyzed 6 6 6 7
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
1
  14.3%
2.Primary Outcome
Title Pharmacokinetics (PK): Maximum Measured Plasma Concentration (Cmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
Hide Description

The Cmax of total GAA and rhGAA protein in plasma was measured after a single rhGAA intravenous infusion and after pre-administration of single ascending oral doses of duvoglustat.

During Treatment Period 1, participants received a single intravenous infusion of rhGAA. During Treatment Period 2, participants received a single oral dose of duvoglustat 1 hour before initiation of a single rhGAA intravenous infusion. PK samples were taken at time points Predose, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 12, and 24 hours, and 3 or 7 days postdose during Periods 1 and 2, and 24 to 30 days postdose during Period 2.

Time Frame Predose, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 12, and 24 hours, and 3 or 7 days postdose during Periods 1 and 2, and 24 to 30 days postdose during Period 2
Hide Outcome Measure Data
Hide Analysis Population Description
The Per Protocol population included all participants who successfully completed both periods.
Arm/Group Title Cohort 1, Duvoglustat 50 mg + rhGAA Cohort 2, Duvoglustat 100 mg + rhGAA Cohort 3, Duvoglustat 250 mg + rhGAA Cohort 4, Duvoglustat 600 mg + rhGAA
Hide Arm/Group Description:
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 50 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 100 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 250 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 600 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
Overall Number of Participants Analyzed 6 6 6 7
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: nmol/mL/h
Total GAA, Period 1
17269
(25.6%)
22785
(18.1%)
18986
(19.5%)
18980
(34.6%)
Total GAA, Period 2
20539
(21.2%)
28607
(14.1%)
22651
(9.0%)
22989
(36.4%)
rhGAA, Period 1
328660
(40.7%)
399622
(33.2%)
405543
(11.4%)
507294
(47.9%)
rhGAA, Period 2
385475
(35.7%)
430738
(39.5%)
438795
(7.0%)
637571
(48.8%)
3.Primary Outcome
Title PK: Time To The Maximum Plasma Concentration (Tmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
Hide Description

The Tmax of total GAA and rhGAA protein in plasma was measured after a single rhGAA intravenous infusion and after pre-administration of single ascending oral doses of duvoglustat.

During Treatment Period 1, participants received a single intravenous infusion of rhGAA. During Treatment Period 2, participants received a single oral dose of duvoglustat 1 hour before initiation of a single rhGAA intravenous infusion. PK samples were taken at time points Predose, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 12, and 24 hours, and 3 or 7 days postdose during Periods 1 and 2, and 24 to 30 days postdose during Period 2.

Time Frame Predose, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 12, and 24 hours, and 3 or 7 days postdose during Periods 1 and 2, and 24 to 30 days postdose during Period 2
Hide Outcome Measure Data
Hide Analysis Population Description
The Per Protocol population included all participants who successfully completed both periods.
Arm/Group Title Cohort 1, Duvoglustat 50 mg + rhGAA Cohort 2, Duvoglustat 100 mg + rhGAA Cohort 3, Duvoglustat 250 mg + rhGAA Cohort 4, Duvoglustat 600 mg + rhGAA
Hide Arm/Group Description:
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 50 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 100 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 250 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 600 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
Overall Number of Participants Analyzed 6 6 6 7
Median (Full Range)
Unit of Measure: h
Total GAA, Period 1
4.74
(4.00 to 5.00)
4.00
(2.98 to 4.97)
4.00
(3.78 to 4.02)
4.00
(3.00 to 6.00)
Total GAA, Period 2
4.51
(3.95 to 5.98)
4.00
(3.00 to 5.98)
4.00
(4.00 to 4.00)
4.00
(3.00 to 6.00)
rhGAA, Period 1
4.98
(4.00 to 5.00)
4.01
(4.00 to 5.07)
4.01
(3.78 to 5.00)
4.00
(3.83 to 6.00)
rhGAA, Period 2
4.98
(4.00 to 6.02)
4.00
(4.00 to 7.00)
4.50
(4.00 to 7.02)
4.00
(3.00 to 6.00)
4.Primary Outcome
Title PK: Elimination Half-life (T1/2) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
Hide Description

The T1/2 of total GAA and rhGAA protein in plasma was measured after a single rhGAA intravenous infusion and after pre-administration of single ascending oral doses of duvoglustat.

During Treatment Period 1, participants received a single intravenous infusion of rhGAA. During Treatment Period 2, participants received a single oral dose of duvoglustat 1 hour before initiation of a single rhGAA intravenous infusion. PK samples were taken at time points Predose, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 12, and 24 hours, and 3 or 7 days postdose during Periods 1 and 2, and 24 to 30 days postdose during Period 2.

Values presented are arithmetic mean (percent coefficient of variation, [CV%]). The number of participants analyzed for some cohorts are reduced because the terminal phase of the concentration profile for these participants was not estimable.

Time Frame Predose, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 12, and 24 hours, and 3 or 7 days postdose during Periods 1 and 2, and 24 to 30 days postdose during Period 2
Hide Outcome Measure Data
Hide Analysis Population Description
The Per Protocol population included all participants who successfully completed both periods.
Arm/Group Title Cohort 1, Duvoglustat 50 mg + rhGAA Cohort 2, Duvoglustat 100 mg + rhGAA Cohort 3, Duvoglustat 250 mg + rhGAA Cohort 4, Duvoglustat 600 mg + rhGAA
Hide Arm/Group Description:
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 50 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 100 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 250 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 600 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
Overall Number of Participants Analyzed 6 6 6 7
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: h
Total GAA, Period 1 Number Analyzed 6 participants 6 participants 6 participants 7 participants
3.81
(12.7%)
3.82
(17.7%)
3.56
(13.7%)
3.70
(19.9%)
Total GAA, Period 2 Number Analyzed 6 participants 6 participants 6 participants 7 participants
4.42
(16.9%)
4.77
(14.2%)
5.36
(26.0%)
6.33
(22.8%)
rhGAA, Period 1 Number Analyzed 6 participants 5 participants 6 participants 4 participants
3.31
(39.6%)
2.33
(59.5%)
1.89
(46.9%)
2.33
(54.3%)
rhGAA, Period 2 Number Analyzed 6 participants 4 participants 6 participants 7 participants
4.43
(38.1%)
5.76
(55.6%)
4.23
(52.3%)
5.71
(45.6%)
5.Primary Outcome
Title PK: Area Under The Plasma Concentration Versus Time Curve From Time 0 To The Time Of The Last Measurable Concentration (AUC0-t) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
Hide Description

The AUC0-t of total GAA and rhGAA protein in plasma was measured after a single rhGAA intravenous infusion and after pre-administration of single ascending oral doses of duvoglustat.

During Treatment Period 1, participants received a single intravenous infusion of rhGAA. During Treatment Period 2, participants received a single oral dose of duvoglustat 1 hour before initiation of a single rhGAA intravenous infusion. PK samples were taken at time points Predose, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 12, and 24 hours, and 3 or 7 days postdose during Periods 1 and 2, and 24 to 30 days postdose during Period 2.

Time Frame Predose, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 12, and 24 hours, and 3 or 7 days postdose during Periods 1 and 2, and 24 to 30 days postdose during Period 2
Hide Outcome Measure Data
Hide Analysis Population Description
The Per Protocol population included all participants who successfully completed both periods.
Arm/Group Title Cohort 1, Duvoglustat 50 mg + rhGAA Cohort 2, Duvoglustat 100 mg + rhGAA Cohort 3, Duvoglustat 250 mg + rhGAA Cohort 4, Duvoglustat 600 mg + rhGAA
Hide Arm/Group Description:
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 50 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 100 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 250 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 600 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
Overall Number of Participants Analyzed 6 6 6 7
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: nmol/mL/h
Total GAA, Period 1
108578
(24.1%)
141515
(27.9%)
107489
(21.7%)
118483
(43.1%)
Total GAA, Period 2
159994
(18.1%)
226198
(25.4%)
201044
(8.3%)
228413
(38.4%)
rhGAA, Period 1
1891079
(35.6%)
2227356
(62.8%)
2248866
(24.7%)
2326168
(55.5%)
rhGAA, Period 2
2914884
(49.2%)
3466263
(59.5%)
3618948
(26.4%)
5293233
(68.2%)
6.Primary Outcome
Title PK: AUC From Time 0 Extrapolated To Infinity (AUCinf) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
Hide Description

The AUCinf of total GAA and rhGAA protein in plasma was measured after a single rhGAA intravenous infusion and after pre-administration of single ascending oral doses of duvoglustat.

During Treatment Period 1, participants received a single intravenous infusion of rhGAA. During Treatment Period 2, participants received a single oral dose of duvoglustat 1 hour before initiation of a single rhGAA intravenous infusion. PK samples were taken at time points Predose, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 12, and 24 hours, and 3 or 7 days postdose during Periods 1 and 2, and 24 to 30 days postdose during Period 2.

The number of participants analyzed for some cohorts are reduced because the terminal phase of the concentration profile for these participants was not estimable.

Time Frame Predose, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 12, and 24 hours, and 3 or 7 days postdose during Periods 1 and 2, and 24 to 30 days postdose during Period 2
Hide Outcome Measure Data
Hide Analysis Population Description
The Per Protocol population included all participants who successfully completed both periods.
Arm/Group Title Cohort 1, Duvoglustat 50 mg + rhGAA Cohort 2, Duvoglustat 100 mg + rhGAA Cohort 3, Duvoglustat 250 mg + rhGAA Cohort 4, Duvoglustat 600 mg + rhGAA
Hide Arm/Group Description:
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 50 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 100 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 250 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 600 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
Overall Number of Participants Analyzed 6 6 6 7
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: h*(nmol/mL/h)
Total GAA, Period 1 Number Analyzed 6 participants 6 participants 6 participants 7 participants
110388
(24.5%)
144056
(28.6%)
108862
(22.1%)
120604
(44.3%)
Total GAA, Period 2 Number Analyzed 6 participants 6 participants 6 participants 7 participants
165983
(19.1%)
237613
(27.6%)
215276
(9.5%)
254074
(42.0%)
rhGAA, Period 1 Number Analyzed 6 participants 5 participants 6 participants 4 participants
2274897
(35.5%)
2232099
(54.0%)
2468980
(26.4%)
2452414
(70.5%)
rhGAA, Period 2 Number Analyzed 6 participants 3 participants 6 participants 7 participants
3603068
(37.4%)
3427088
(71.1%)
4602726
(31.3%)
6691323
(46.6%)
7.Secondary Outcome
Title Total GAA Activity In Skeletal Muscle
Hide Description The total GAA activity in skeletal muscle was measured after a single intravenous administration of rhGAA alone and after pre-administration of single ascending oral doses of duvoglustat. Participants were assessed using skeletal muscle biopsies at either Day 3 or Day 7 during Treatment Periods 1 and 2.
Time Frame Day 3 or Day 7
Hide Outcome Measure Data
Hide Analysis Population Description
The Per Protocol population included all participants who successfully completed both periods.
Arm/Group Title Cohort 1, Duvoglustat 50 mg + rhGAA Cohort 2, Duvoglustat 100 mg + rhGAA Cohort 3, Duvoglustat 250 mg + rhGAA Cohort 4, Duvoglustat 600 mg + rhGAA
Hide Arm/Group Description:
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 50 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 100 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 250 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 600 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
Overall Number of Participants Analyzed 6 6 6 6
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: pmol/mg/h
Total GAA, Period 1, Day 3 Number Analyzed 2 participants 3 participants 3 participants 3 participants
1409
(130%)
1926
(67%)
2622
(14%)
1887
(47%)
Total GAA, Period 2, Day 3 Number Analyzed 2 participants 3 participants 3 participants 3 participants
1952
(8%)
2320
(77%)
2746
(35%)
2710
(37%)
Total GAA, Period 1, Day 7 Number Analyzed 4 participants 3 participants 3 participants 3 participants
1216
(24%)
861
(16%)
NA [1] 
(91%)
916
(25%)
Total GAA, Period 2, Day 7 Number Analyzed 4 participants 3 participants 3 participants 3 participants
1197
(18%)
1064
(39%)
1690
(22%)
1085
(14%)
[1]
Value not calculated because 1 participant’s value was below the limit of quantification.
8.Secondary Outcome
Title Duvoglustat Concentration In Skeletal Muscle
Hide Description

The concentration of duvoglustat in skeletal muscle tissue homogenate was measured after pre-administration of single ascending oral doses of duvoglustat during Treatment Period 2. Participants had skeletal muscle biopsies at either Day 3 or Day 7 during Treatment Period 2.

Three participants were excluded from this analysis due to the following reasons: treatment sequence was inadvertently switched due to study site error, follow-up biopsy sample could not be conclusively identified, or muscle biopsies were mislabeled at the clinical site.

Values presented are arithmetic mean (percent coefficient of variation, [CV%]) because of the prevalence of participants with values below the limit of quantification. Concentrations below the limit of quantification were treated as zero.

Time Frame Day 3 or Day 7
Hide Outcome Measure Data
Hide Analysis Population Description
The Per Protocol population included all participants who successfully completed both periods.
Arm/Group Title Cohort 1, Duvoglustat 50 mg + rhGAA Cohort 2, Duvoglustat 100 mg + rhGAA Cohort 3, Duvoglustat 250 mg + rhGAA Cohort 4, Duvoglustat 600 mg + rhGAA
Hide Arm/Group Description:
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 50 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 100 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 250 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 600 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
Overall Number of Participants Analyzed 6 6 6 7
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: ng/g
Duvoglustat Concentration, Day 3 Number Analyzed 1 participants 3 participants 3 participants 1 participants
0 [1] 
(NA%)
5.9
(173%)
38.8
(87%)
83.6 [1] 
(NA%)
Duvoglustat Concentration, Day 7 Number Analyzed 4 participants 3 participants 2 participants 3 participants
8.7
(68%)
0 [1] 
(NA%)
28.0
(0.5%)
54.1
(25%)
[1]
Value was not calculated because participant's value was below the limit of quantification.
Time Frame Day 1 after dosing up to Day 60
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Cohort 1, Duvoglustat 50 mg + rhGAA Cohort 2, Duvoglustat 100 mg + rhGAA Cohort 3, Duvoglustat 250 mg + rhGAA Cohort 4, Duvoglustat 600 mg + rhGAA
Hide Arm/Group Description During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 50 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion. During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 100 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion. During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 250 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion. During Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each participant received a single 600 mg oral dose of duvoglustat 1 hour prior to initiation of a single rhGAA infusion.
All-Cause Mortality
Cohort 1, Duvoglustat 50 mg + rhGAA Cohort 2, Duvoglustat 100 mg + rhGAA Cohort 3, Duvoglustat 250 mg + rhGAA Cohort 4, Duvoglustat 600 mg + rhGAA
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Cohort 1, Duvoglustat 50 mg + rhGAA Cohort 2, Duvoglustat 100 mg + rhGAA Cohort 3, Duvoglustat 250 mg + rhGAA Cohort 4, Duvoglustat 600 mg + rhGAA
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/6 (0.00%)   1/6 (16.67%)   0/6 (0.00%)   0/7 (0.00%) 
Investigations         
Electrocardiogram QT prolonged  1  0/6 (0.00%)  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 14.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Cohort 1, Duvoglustat 50 mg + rhGAA Cohort 2, Duvoglustat 100 mg + rhGAA Cohort 3, Duvoglustat 250 mg + rhGAA Cohort 4, Duvoglustat 600 mg + rhGAA
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   5/6 (83.33%)   3/6 (50.00%)   4/6 (66.67%)   4/7 (57.14%) 
Cardiac disorders         
Palpitations  1  0/6 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%) 
Ear and labyrinth disorders         
Vertigo  1  0/6 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%) 
Gastrointestinal disorders         
Dental caries  1  1/6 (16.67%)  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%) 
Constipation  1  0/6 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%) 
Oral pain  1  1/6 (16.67%)  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%) 
General disorders         
Oedema peripheral  1  0/6 (0.00%)  0/6 (0.00%)  2/6 (33.33%)  0/7 (0.00%) 
Fatigue  1  1/6 (16.67%)  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%) 
Nodule  1  0/6 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  1/7 (14.29%) 
Infections and infestations         
Oral candidiasis  1  0/6 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%) 
Otitis media  1  0/6 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%) 
Respiratory tract infection  1  0/6 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%) 
Sinusitis  1  0/6 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%) 
Tooth abscess  1  0/6 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%) 
Upper respiratory tract infection  1  1/6 (16.67%)  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%) 
Urinary tract infection  1  0/6 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%) 
Injury, poisoning and procedural complications         
Post procedural haematoma  1  0/6 (0.00%)  1/6 (16.67%)  1/6 (16.67%)  0/7 (0.00%) 
Procedural pain  1  0/6 (0.00%)  2/6 (33.33%)  4/6 (66.67%)  0/7 (0.00%) 
Back injury  1  0/6 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%) 
Fall  1  0/6 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  1/7 (14.29%) 
Muscle strain  1  0/6 (0.00%)  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%) 
Procedural complication  1  0/6 (0.00%)  0/6 (0.00%)  2/6 (33.33%)  0/7 (0.00%) 
Tooth fracture  1  0/6 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%) 
Investigations         
Weight increased  1  0/6 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%) 
Musculoskeletal and connective tissue disorders         
Pain in extremity  1  2/6 (33.33%)  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%) 
Back pain  1  0/6 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  1/7 (14.29%) 
Neck pain  1  0/6 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Melanocytic naevus  1  0/6 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%) 
Ovarian neoplasm  1  0/6 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%) 
Nervous system disorders         
Headache  1  0/6 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%) 
Hyperaesthesia  1  1/6 (16.67%)  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%) 
Somnolence  1  1/6 (16.67%)  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%) 
Renal and urinary disorders         
Dysuria  1  0/6 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%) 
Nephrolithiasis  1  1/6 (16.67%)  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%) 
Pollakiuria  1  1/6 (16.67%)  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%) 
Urinary incontinence  1  0/6 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%) 
Reproductive system and breast disorders         
Uterine enlargement  1  0/3 (0.00%)  0/3 (0.00%)  0/4 (0.00%)  1/2 (50.00%) 
Respiratory, thoracic and mediastinal disorders         
Dyspnoea  1  0/6 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%) 
Sinus congestion  1  1/6 (16.67%)  0/6 (0.00%)  0/6 (0.00%)  0/7 (0.00%) 
Skin and subcutaneous tissue disorders         
Dermatitis contact  1  0/6 (0.00%)  1/6 (16.67%)  0/6 (0.00%)  0/7 (0.00%) 
Erythema  1  0/6 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%) 
Hyperhidrosis  1  0/6 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%) 
Scar pain  1  0/6 (0.00%)  0/6 (0.00%)  0/6 (0.00%)  1/7 (14.29%) 
Vascular disorders         
Haematoma  1  0/6 (0.00%)  0/6 (0.00%)  1/6 (16.67%)  0/7 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 14.0
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 investigator can only publish the results from this trial provided they supply the sponsor (or authorized entity) a copy of any proposed publication for review. If requested, the investigator will remove information deemed confidential or proprietary by the sponsor and will withhold publication for an additional period of time to allow the sponsor to take appropriate measures to establish and preserve its proprietary rights.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Amicus Therapeutics
Organization: Patient Advocacy
Phone: +1-609-662-2000
EMail: clinicaltrials@amicusrx.com
Layout table for additonal information
Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT01380743     History of Changes
Other Study ID Numbers: AT2220-010
First Submitted: June 23, 2011
First Posted: June 27, 2011
Results First Submitted: July 23, 2018
Results First Posted: August 17, 2018
Last Update Posted: October 2, 2018