Trial Outcomes & Findings for Drug-drug Interaction Study (NCT NCT01380743)
NCT ID: NCT01380743
Last Updated: 2025-06-26
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
25 participants
Primary outcome timeframe
Day 1 after dosing up to Day 60
Results posted on
2025-06-26
Participant Flow
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.
Participant milestones
| Measure |
Cohort 4, Duvoglustat 600 mg + rhGAA
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.
|
Cohort 1, Duvoglustat 50 mg + rhGAA
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.
|
Cohort 2, Duvoglustat 100 mg + rhGAA
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.
|
Cohort 3, Duvoglustat 250 mg + rhGAA
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.
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
7
|
6
|
6
|
6
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
7
|
6
|
6
|
6
|
|
Overall Study
COMPLETED
|
7
|
6
|
6
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Drug-drug Interaction Study
Baseline characteristics by cohort
| Measure |
Cohort 1, Duvoglustat 50 mg + rhGAA
n=6 Participants
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.
|
Cohort 2, Duvoglustat 100 mg + rhGAA
n=6 Participants
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.
|
Cohort 3, Duvoglustat 250 mg + rhGAA
n=6 Participants
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.
|
Cohort 4, Duvoglustat 600 mg + rhGAA
n=7 Participants
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
n=25 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Continuous
|
47.7 years
STANDARD_DEVIATION 5.35 • n=5 Participants
|
52.8 years
STANDARD_DEVIATION 5.71 • n=7 Participants
|
50.0 years
STANDARD_DEVIATION 12.90 • n=5 Participants
|
40.0 years
STANDARD_DEVIATION 6.61 • n=4 Participants
|
47.3 years
STANDARD_DEVIATION 9.13 • n=21 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
12 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
13 Participants
n=21 Participants
|
PRIMARY outcome
Timeframe: Day 1 after dosing up to Day 60Population: The Safety Population included all participants who were enrolled and received at least 1 dose of rhGAA or duvoglustat.
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.
Outcome measures
| Measure |
Cohort 1, Duvoglustat 50 mg + rhGAA
n=6 Participants
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.
|
Cohort 2, Duvoglustat 100 mg + rhGAA
n=6 Participants
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.
|
Cohort 3, Duvoglustat 250 mg + rhGAA
n=6 Participants
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.
|
Cohort 4, Duvoglustat 600 mg + rhGAA
n=7 Participants
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.
|
|---|---|---|---|---|
|
Number Of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs)
|
0 Participants
|
0 Participants
|
0 Participants
|
1 Participants
|
PRIMARY outcome
Timeframe: 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 2Population: The Per Protocol population included all participants who successfully completed both periods.
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.
Outcome measures
| Measure |
Cohort 1, Duvoglustat 50 mg + rhGAA
n=6 Participants
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.
|
Cohort 2, Duvoglustat 100 mg + rhGAA
n=6 Participants
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.
|
Cohort 3, Duvoglustat 250 mg + rhGAA
n=6 Participants
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.
|
Cohort 4, Duvoglustat 600 mg + rhGAA
n=7 Participants
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.
|
|---|---|---|---|---|
|
Pharmacokinetics (PK): Maximum Measured Plasma Concentration (Cmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
Total GAA, Period 1
|
17269 nmol/mL/h
Geometric Coefficient of Variation 25.6
|
22785 nmol/mL/h
Geometric Coefficient of Variation 18.1
|
18986 nmol/mL/h
Geometric Coefficient of Variation 19.5
|
18980 nmol/mL/h
Geometric Coefficient of Variation 34.6
|
|
Pharmacokinetics (PK): Maximum Measured Plasma Concentration (Cmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
Total GAA, Period 2
|
20539 nmol/mL/h
Geometric Coefficient of Variation 21.2
|
28607 nmol/mL/h
Geometric Coefficient of Variation 14.1
|
22651 nmol/mL/h
Geometric Coefficient of Variation 9.0
|
22989 nmol/mL/h
Geometric Coefficient of Variation 36.4
|
|
Pharmacokinetics (PK): Maximum Measured Plasma Concentration (Cmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
rhGAA, Period 1
|
328660 nmol/mL/h
Geometric Coefficient of Variation 40.7
|
399622 nmol/mL/h
Geometric Coefficient of Variation 33.2
|
405543 nmol/mL/h
Geometric Coefficient of Variation 11.4
|
507294 nmol/mL/h
Geometric Coefficient of Variation 47.9
|
|
Pharmacokinetics (PK): Maximum Measured Plasma Concentration (Cmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
rhGAA, Period 2
|
385475 nmol/mL/h
Geometric Coefficient of Variation 35.7
|
430738 nmol/mL/h
Geometric Coefficient of Variation 39.5
|
438795 nmol/mL/h
Geometric Coefficient of Variation 7.0
|
637571 nmol/mL/h
Geometric Coefficient of Variation 48.8
|
PRIMARY outcome
Timeframe: 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 2Population: The Per Protocol population included all participants who successfully completed both periods.
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.
Outcome measures
| Measure |
Cohort 1, Duvoglustat 50 mg + rhGAA
n=6 Participants
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.
|
Cohort 2, Duvoglustat 100 mg + rhGAA
n=6 Participants
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.
|
Cohort 3, Duvoglustat 250 mg + rhGAA
n=6 Participants
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.
|
Cohort 4, Duvoglustat 600 mg + rhGAA
n=7 Participants
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.
|
|---|---|---|---|---|
|
PK: Time To The Maximum Plasma Concentration (Tmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
Total GAA, Period 1
|
4.74 h
Interval 4.0 to 5.0
|
4.00 h
Interval 2.98 to 4.97
|
4.00 h
Interval 3.78 to 4.02
|
4.00 h
Interval 3.0 to 6.0
|
|
PK: Time To The Maximum Plasma Concentration (Tmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
Total GAA, Period 2
|
4.51 h
Interval 3.95 to 5.98
|
4.00 h
Interval 3.0 to 5.98
|
4.00 h
Interval 4.0 to 4.0
|
4.00 h
Interval 3.0 to 6.0
|
|
PK: Time To The Maximum Plasma Concentration (Tmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
rhGAA, Period 1
|
4.98 h
Interval 4.0 to 5.0
|
4.01 h
Interval 4.0 to 5.07
|
4.01 h
Interval 3.78 to 5.0
|
4.00 h
Interval 3.83 to 6.0
|
|
PK: Time To The Maximum Plasma Concentration (Tmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
rhGAA, Period 2
|
4.98 h
Interval 4.0 to 6.02
|
4.00 h
Interval 4.0 to 7.0
|
4.50 h
Interval 4.0 to 7.02
|
4.00 h
Interval 3.0 to 6.0
|
PRIMARY outcome
Timeframe: 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 2Population: The Per Protocol population included all participants who successfully completed both periods.
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.
Outcome measures
| Measure |
Cohort 1, Duvoglustat 50 mg + rhGAA
n=6 Participants
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.
|
Cohort 2, Duvoglustat 100 mg + rhGAA
n=6 Participants
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.
|
Cohort 3, Duvoglustat 250 mg + rhGAA
n=6 Participants
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.
|
Cohort 4, Duvoglustat 600 mg + rhGAA
n=7 Participants
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.
|
|---|---|---|---|---|
|
PK: Elimination Half-life (T1/2) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
Total GAA, Period 1
|
3.81 h
Geometric Coefficient of Variation 12.7
|
3.82 h
Geometric Coefficient of Variation 17.7
|
3.56 h
Geometric Coefficient of Variation 13.7
|
3.70 h
Geometric Coefficient of Variation 19.9
|
|
PK: Elimination Half-life (T1/2) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
Total GAA, Period 2
|
4.42 h
Geometric Coefficient of Variation 16.9
|
4.77 h
Geometric Coefficient of Variation 14.2
|
5.36 h
Geometric Coefficient of Variation 26.0
|
6.33 h
Geometric Coefficient of Variation 22.8
|
|
PK: Elimination Half-life (T1/2) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
rhGAA, Period 1
|
3.31 h
Geometric Coefficient of Variation 39.6
|
2.33 h
Geometric Coefficient of Variation 59.5
|
1.89 h
Geometric Coefficient of Variation 46.9
|
2.33 h
Geometric Coefficient of Variation 54.3
|
|
PK: Elimination Half-life (T1/2) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
rhGAA, Period 2
|
4.43 h
Geometric Coefficient of Variation 38.1
|
5.76 h
Geometric Coefficient of Variation 55.6
|
4.23 h
Geometric Coefficient of Variation 52.3
|
5.71 h
Geometric Coefficient of Variation 45.6
|
PRIMARY outcome
Timeframe: 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 2Population: The Per Protocol population included all participants who successfully completed both periods.
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.
Outcome measures
| Measure |
Cohort 1, Duvoglustat 50 mg + rhGAA
n=6 Participants
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.
|
Cohort 2, Duvoglustat 100 mg + rhGAA
n=6 Participants
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.
|
Cohort 3, Duvoglustat 250 mg + rhGAA
n=6 Participants
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.
|
Cohort 4, Duvoglustat 600 mg + rhGAA
n=7 Participants
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.
|
|---|---|---|---|---|
|
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
Total GAA, Period 1
|
108578 nmol/mL/h
Geometric Coefficient of Variation 24.1
|
141515 nmol/mL/h
Geometric Coefficient of Variation 27.9
|
107489 nmol/mL/h
Geometric Coefficient of Variation 21.7
|
118483 nmol/mL/h
Geometric Coefficient of Variation 43.1
|
|
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
Total GAA, Period 2
|
159994 nmol/mL/h
Geometric Coefficient of Variation 18.1
|
226198 nmol/mL/h
Geometric Coefficient of Variation 25.4
|
201044 nmol/mL/h
Geometric Coefficient of Variation 8.3
|
228413 nmol/mL/h
Geometric Coefficient of Variation 38.4
|
|
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
rhGAA, Period 1
|
1891079 nmol/mL/h
Geometric Coefficient of Variation 35.6
|
2227356 nmol/mL/h
Geometric Coefficient of Variation 62.8
|
2248866 nmol/mL/h
Geometric Coefficient of Variation 24.7
|
2326168 nmol/mL/h
Geometric Coefficient of Variation 55.5
|
|
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
rhGAA, Period 2
|
2914884 nmol/mL/h
Geometric Coefficient of Variation 49.2
|
3466263 nmol/mL/h
Geometric Coefficient of Variation 59.5
|
3618948 nmol/mL/h
Geometric Coefficient of Variation 26.4
|
5293233 nmol/mL/h
Geometric Coefficient of Variation 68.2
|
PRIMARY outcome
Timeframe: 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 2Population: The Per Protocol population included all participants who successfully completed both periods.
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.
Outcome measures
| Measure |
Cohort 1, Duvoglustat 50 mg + rhGAA
n=6 Participants
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.
|
Cohort 2, Duvoglustat 100 mg + rhGAA
n=6 Participants
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.
|
Cohort 3, Duvoglustat 250 mg + rhGAA
n=6 Participants
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.
|
Cohort 4, Duvoglustat 600 mg + rhGAA
n=7 Participants
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.
|
|---|---|---|---|---|
|
PK: AUC From Time 0 Extrapolated To Infinity (AUCinf) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
Total GAA, Period 1
|
110388 h*(nmol/mL/h)
Geometric Coefficient of Variation 24.5
|
144056 h*(nmol/mL/h)
Geometric Coefficient of Variation 28.6
|
108862 h*(nmol/mL/h)
Geometric Coefficient of Variation 22.1
|
120604 h*(nmol/mL/h)
Geometric Coefficient of Variation 44.3
|
|
PK: AUC From Time 0 Extrapolated To Infinity (AUCinf) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
Total GAA, Period 2
|
165983 h*(nmol/mL/h)
Geometric Coefficient of Variation 19.1
|
237613 h*(nmol/mL/h)
Geometric Coefficient of Variation 27.6
|
215276 h*(nmol/mL/h)
Geometric Coefficient of Variation 9.5
|
254074 h*(nmol/mL/h)
Geometric Coefficient of Variation 42.0
|
|
PK: AUC From Time 0 Extrapolated To Infinity (AUCinf) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
rhGAA, Period 1
|
2274897 h*(nmol/mL/h)
Geometric Coefficient of Variation 35.5
|
2232099 h*(nmol/mL/h)
Geometric Coefficient of Variation 54.0
|
2468980 h*(nmol/mL/h)
Geometric Coefficient of Variation 26.4
|
2452414 h*(nmol/mL/h)
Geometric Coefficient of Variation 70.5
|
|
PK: AUC From Time 0 Extrapolated To Infinity (AUCinf) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease
rhGAA, Period 2
|
3603068 h*(nmol/mL/h)
Geometric Coefficient of Variation 37.4
|
3427088 h*(nmol/mL/h)
Geometric Coefficient of Variation 71.1
|
4602726 h*(nmol/mL/h)
Geometric Coefficient of Variation 31.3
|
6691323 h*(nmol/mL/h)
Geometric Coefficient of Variation 46.6
|
SECONDARY outcome
Timeframe: Day 3 or Day 7Population: The Per Protocol population included all participants who successfully completed both periods.
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.
Outcome measures
| Measure |
Cohort 1, Duvoglustat 50 mg + rhGAA
n=6 Participants
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.
|
Cohort 2, Duvoglustat 100 mg + rhGAA
n=6 Participants
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.
|
Cohort 3, Duvoglustat 250 mg + rhGAA
n=6 Participants
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.
|
Cohort 4, Duvoglustat 600 mg + rhGAA
n=6 Participants
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 GAA Activity In Skeletal Muscle
Total GAA, Period 1, Day 3
|
1409 pmol/mg/h
Geometric Coefficient of Variation 130
|
1926 pmol/mg/h
Geometric Coefficient of Variation 67
|
2622 pmol/mg/h
Geometric Coefficient of Variation 14
|
1887 pmol/mg/h
Geometric Coefficient of Variation 47
|
|
Total GAA Activity In Skeletal Muscle
Total GAA, Period 2, Day 3
|
1952 pmol/mg/h
Geometric Coefficient of Variation 8
|
2320 pmol/mg/h
Geometric Coefficient of Variation 77
|
2746 pmol/mg/h
Geometric Coefficient of Variation 35
|
2710 pmol/mg/h
Geometric Coefficient of Variation 37
|
|
Total GAA Activity In Skeletal Muscle
Total GAA, Period 1, Day 7
|
1216 pmol/mg/h
Geometric Coefficient of Variation 24
|
861 pmol/mg/h
Geometric Coefficient of Variation 16
|
NA pmol/mg/h
Geometric Coefficient of Variation 91
Value not calculated because 1 participant's value was below the limit of quantification.
|
916 pmol/mg/h
Geometric Coefficient of Variation 25
|
|
Total GAA Activity In Skeletal Muscle
Total GAA, Period 2, Day 7
|
1197 pmol/mg/h
Geometric Coefficient of Variation 18
|
1064 pmol/mg/h
Geometric Coefficient of Variation 39
|
1690 pmol/mg/h
Geometric Coefficient of Variation 22
|
1085 pmol/mg/h
Geometric Coefficient of Variation 14
|
SECONDARY outcome
Timeframe: Day 3 or Day 7Population: The Per Protocol population included all participants who successfully completed both periods.
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.
Outcome measures
| Measure |
Cohort 1, Duvoglustat 50 mg + rhGAA
n=6 Participants
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.
|
Cohort 2, Duvoglustat 100 mg + rhGAA
n=6 Participants
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.
|
Cohort 3, Duvoglustat 250 mg + rhGAA
n=6 Participants
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.
|
Cohort 4, Duvoglustat 600 mg + rhGAA
n=7 Participants
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.
|
|---|---|---|---|---|
|
Duvoglustat Concentration In Skeletal Muscle
Duvoglustat Concentration, Day 3
|
0 ng/g
Geometric Coefficient of Variation NA
Value was not calculated because participant's value was below the limit of quantification.
|
5.9 ng/g
Geometric Coefficient of Variation 173
|
38.8 ng/g
Geometric Coefficient of Variation 87
|
83.6 ng/g
Geometric Coefficient of Variation NA
Value was not calculated because participant's value was below the limit of quantification.
|
|
Duvoglustat Concentration In Skeletal Muscle
Duvoglustat Concentration, Day 7
|
8.7 ng/g
Geometric Coefficient of Variation 68
|
0 ng/g
Geometric Coefficient of Variation NA
Value was not calculated because participant's value was below the limit of quantification.
|
28.0 ng/g
Geometric Coefficient of Variation 0.5
|
54.1 ng/g
Geometric Coefficient of Variation 25
|
Adverse Events
Cohort 1, Duvoglustat 50 mg + rhGAA
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Cohort 2, Duvoglustat 100 mg + rhGAA
Serious events: 1 serious events
Other events: 3 other events
Deaths: 0 deaths
Cohort 3, Duvoglustat 250 mg + rhGAA
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Cohort 4, Duvoglustat 600 mg + rhGAA
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cohort 1, Duvoglustat 50 mg + rhGAA
n=6 participants at risk
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.
|
Cohort 2, Duvoglustat 100 mg + rhGAA
n=6 participants at risk
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.
|
Cohort 3, Duvoglustat 250 mg + rhGAA
n=6 participants at risk
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.
|
Cohort 4, Duvoglustat 600 mg + rhGAA
n=7 participants at risk
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.
|
|---|---|---|---|---|
|
Investigations
Electrocardiogram QT prolonged
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
Other adverse events
| Measure |
Cohort 1, Duvoglustat 50 mg + rhGAA
n=6 participants at risk
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.
|
Cohort 2, Duvoglustat 100 mg + rhGAA
n=6 participants at risk
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.
|
Cohort 3, Duvoglustat 250 mg + rhGAA
n=6 participants at risk
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.
|
Cohort 4, Duvoglustat 600 mg + rhGAA
n=7 participants at risk
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.
|
|---|---|---|---|---|
|
Gastrointestinal disorders
Dental caries
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
General disorders
Oedema peripheral
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
33.3%
2/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Injury, poisoning and procedural complications
Post procedural haematoma
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Injury, poisoning and procedural complications
Procedural pain
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
33.3%
2/6 • Day 1 after dosing up to Day 60
|
66.7%
4/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
33.3%
2/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
14.3%
1/7 • Day 1 after dosing up to Day 60
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Melanocytic naevus
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
14.3%
1/7 • Day 1 after dosing up to Day 60
|
|
Renal and urinary disorders
Dysuria
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
14.3%
1/7 • Day 1 after dosing up to Day 60
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Skin and subcutaneous tissue disorders
Erythema
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
14.3%
1/7 • Day 1 after dosing up to Day 60
|
|
Skin and subcutaneous tissue disorders
Scar pain
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
14.3%
1/7 • Day 1 after dosing up to Day 60
|
|
Vascular disorders
Haematoma
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Cardiac disorders
Palpitations
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
14.3%
1/7 • Day 1 after dosing up to Day 60
|
|
Ear and labyrinth disorders
Vertigo
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
14.3%
1/7 • Day 1 after dosing up to Day 60
|
|
Gastrointestinal disorders
Oral pain
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
General disorders
Fatigue
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
General disorders
Nodule
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
14.3%
1/7 • Day 1 after dosing up to Day 60
|
|
Infections and infestations
Oral candidiasis
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Infections and infestations
Otitis media
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Infections and infestations
Respiratory tract infection
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
14.3%
1/7 • Day 1 after dosing up to Day 60
|
|
Infections and infestations
Sinusitis
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Infections and infestations
Tooth abscess
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Infections and infestations
Upper respiratory tract infection
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Injury, poisoning and procedural complications
Back injury
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
14.3%
1/7 • Day 1 after dosing up to Day 60
|
|
Injury, poisoning and procedural complications
Muscle strain
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Injury, poisoning and procedural complications
Procedural complication
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
33.3%
2/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Injury, poisoning and procedural complications
Tooth fracture
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Investigations
Weight increased
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
14.3%
1/7 • Day 1 after dosing up to Day 60
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
14.3%
1/7 • Day 1 after dosing up to Day 60
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
14.3%
1/7 • Day 1 after dosing up to Day 60
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Ovarian neoplasm
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
14.3%
1/7 • Day 1 after dosing up to Day 60
|
|
Nervous system disorders
Headache
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Nervous system disorders
Hyperaesthesia
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Nervous system disorders
Somnolence
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Renal and urinary disorders
Nephrolithiasis
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Renal and urinary disorders
Pollakiuria
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
|
Renal and urinary disorders
Urinary incontinence
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
14.3%
1/7 • Day 1 after dosing up to Day 60
|
|
Reproductive system and breast disorders
Uterine enlargement
|
0.00%
0/3 • Day 1 after dosing up to Day 60
|
0.00%
0/3 • Day 1 after dosing up to Day 60
|
0.00%
0/4 • Day 1 after dosing up to Day 60
|
50.0%
1/2 • Day 1 after dosing up to Day 60
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
16.7%
1/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/6 • Day 1 after dosing up to Day 60
|
0.00%
0/7 • Day 1 after dosing up to Day 60
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER