Trial · NCT01380743
Drug-drug Interaction Study
NCT01380743, Phase 2, Completed, Amicus Therapeutics; results first posted 2018-08-17
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ConductScience Library. Drug-drug Interaction Study. Trials, record NCT01380743. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01380743
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Drug-drug Interaction Study
- NCT number
- NCT01380743
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 25 participants, actual
- Lead sponsor
- Amicus Therapeutics
- Conditions
- Pompe Disease
- Interventions
- duvoglustat, rhGAA
- First posted
- 2011-06-27
- Last updated
- 2025-06-26
- Results first posted
- 2018-08-17
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number Of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs) | Day 1 after dosing up to Day 60 | 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. |
| Primary | Pharmacokinetics (PK): Maximum Measured Plasma Concentration (Cmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease | 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 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. |
| Primary | PK: Time To The Maximum Plasma Concentration (Tmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease | 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 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. |
| Primary | PK: Elimination Half-life (T1/2) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease | 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 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. |
| Primary | 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 | 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 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. |
| Primary | PK: AUC From Time 0 Extrapolated To Infinity (AUCinf) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease | 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 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. |
| Secondary | Total GAA Activity In Skeletal Muscle | Day 3 or Day 7 | 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. |
| Secondary | Duvoglustat Concentration In Skeletal Muscle | Day 3 or Day 7 | 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. |
Source ClinicalTrials.gov
Outcomes8
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number Of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs) | Day 1 after dosing up to Day 60 | The Safety Population included all participants who were enrolled and received at least 1 dose of rhGAA or duvoglustat. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Pharmacokinetics (PK): Maximum Measured Plasma Concentration (Cmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease | 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 Per Protocol population included all participants who successfully completed both periods. | nmol/mL/h | GEOMETRIC_MEAN |
| PRIMARY | PK: Time To The Maximum Plasma Concentration (Tmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease | 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 Per Protocol population included all participants who successfully completed both periods. | h | MEDIAN |
| PRIMARY | PK: Elimination Half-life (T1/2) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease | 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 Per Protocol population included all participants who successfully completed both periods. | h | GEOMETRIC_MEAN |
| PRIMARY | 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 | 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 Per Protocol population included all participants who successfully completed both periods. | nmol/mL/h | GEOMETRIC_MEAN |
| PRIMARY | PK: AUC From Time 0 Extrapolated To Infinity (AUCinf) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease | 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 Per Protocol population included all participants who successfully completed both periods. | h*(nmol/mL/h) | GEOMETRIC_MEAN |
| SECONDARY | Total GAA Activity In Skeletal Muscle | Day 3 or Day 7 | The Per Protocol population included all participants who successfully completed both periods. | pmol/mg/h | GEOMETRIC_MEAN |
| SECONDARY | Duvoglustat Concentration In Skeletal Muscle | Day 3 or Day 7 | The Per Protocol population included all participants who successfully completed both periods. | ng/g | GEOMETRIC_MEAN |
Source ClinicalTrials.gov
Results by group108
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number Of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs) | Cohort 1, Duvoglustat 50 mg + rhGAA | 0 | Participants | ||
| Number Of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs) | Cohort 2, Duvoglustat 100 mg + rhGAA | 0 | Participants | ||
| Number Of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs) | Cohort 3, Duvoglustat 250 mg + rhGAA | 0 | Participants | ||
| Number Of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs) | Cohort 4, Duvoglustat 600 mg + rhGAA | 1 | Participants | ||
| Pharmacokinetics (PK): Maximum Measured Plasma Concentration (Cmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease | Cohort 1, Duvoglustat 50 mg + rhGAA | Total GAA, Period 1 | 17269 | Geometric Coefficient of Variation 25.6 | nmol/mL/h |
| Pharmacokinetics (PK): Maximum Measured Plasma Concentration (Cmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease | Cohort 2, Duvoglustat 100 mg + rhGAA | Total GAA, Period 1 | 22785 | Geometric Coefficient of Variation 18.1 | nmol/mL/h |
| Pharmacokinetics (PK): Maximum Measured Plasma Concentration (Cmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease | Cohort 3, Duvoglustat 250 mg + rhGAA | Total GAA, Period 1 | 18986 | Geometric Coefficient of Variation 19.5 | nmol/mL/h |
| Pharmacokinetics (PK): Maximum Measured Plasma Concentration (Cmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease | Cohort 4, Duvoglustat 600 mg + rhGAA | Total GAA, Period 1 | 18980 | Geometric Coefficient of Variation 34.6 | nmol/mL/h |
| Pharmacokinetics (PK): Maximum Measured Plasma Concentration (Cmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease | Cohort 1, Duvoglustat 50 mg + rhGAA | Total GAA, Period 2 | 20539 | Geometric Coefficient of Variation 21.2 | nmol/mL/h |
| Pharmacokinetics (PK): Maximum Measured Plasma Concentration (Cmax) Of Total GAA And rhGAA Protein In Plasma In Participants With Pompe Disease | Cohort 2, Duvoglustat 100 mg + rhGAA | Total GAA, Period 2 | 28607 | Geometric Coefficient of Variation 14.1 | nmol/mL/h |
1 to 10 of 108Source ClinicalTrials.gov
See all 108 results by groupParticipant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1, Duvoglustat 50 mg + rhGAA | 6 | |
| Overall Study | STARTED | Cohort 2, Duvoglustat 100 mg + rhGAA | 6 | |
| Overall Study | STARTED | Cohort 3, Duvoglustat 250 mg + rhGAA | 6 | |
| Overall Study | STARTED | Cohort 4, Duvoglustat 600 mg + rhGAA | 7 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Cohort 1, Duvoglustat 50 mg + rhGAA | 6 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Cohort 2, Duvoglustat 100 mg + rhGAA | 6 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Cohort 3, Duvoglustat 250 mg + rhGAA | 6 | |
| Overall Study | Received at Least 1 Dose of Study Drug | Cohort 4, Duvoglustat 600 mg + rhGAA | 7 | |
| Overall Study | COMPLETED | Cohort 1, Duvoglustat 50 mg + rhGAA | 6 | |
| Overall Study | COMPLETED | Cohort 2, Duvoglustat 100 mg + rhGAA | 6 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events176
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Dental caries | Gastrointestinal disorders | Cohort 1, Duvoglustat 50 mg + rhGAA | 1 | 6 | |
| other | Dental caries | Gastrointestinal disorders | Cohort 2, Duvoglustat 100 mg + rhGAA | 0 | 6 | |
| other | Dental caries | Gastrointestinal disorders | Cohort 3, Duvoglustat 250 mg + rhGAA | 0 | 6 | |
| other | Dental caries | Gastrointestinal disorders | Cohort 4, Duvoglustat 600 mg + rhGAA | 0 | 7 | |
| other | Oedema peripheral | General disorders | Cohort 1, Duvoglustat 50 mg + rhGAA | 0 | 6 | |
| other | Oedema peripheral | General disorders | Cohort 2, Duvoglustat 100 mg + rhGAA | 0 | 6 | |
| other | Oedema peripheral | General disorders | Cohort 3, Duvoglustat 250 mg + rhGAA | 2 | 6 | |
| other | Oedema peripheral | General disorders | Cohort 4, Duvoglustat 600 mg + rhGAA | 0 | 7 | |
| other | Post procedural haematoma | Injury, poisoning and procedural complications | Cohort 1, Duvoglustat 50 mg + rhGAA | 0 | 6 | |
| other | Post procedural haematoma | Injury, poisoning and procedural complications | Cohort 2, Duvoglustat 100 mg + rhGAA | 1 | 6 |
1 to 10 of 176Source ClinicalTrials.gov
See all 176 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1, Duvoglustat 50 mg + rhGAA | ||
| Total, all-cause mortality | Cohort 2, Duvoglustat 100 mg + rhGAA | ||
| Total, all-cause mortality | Cohort 3, Duvoglustat 250 mg + rhGAA | ||
| Total, all-cause mortality | Cohort 4, Duvoglustat 600 mg + rhGAA | ||
| Total, other adverse events | Cohort 1, Duvoglustat 50 mg + rhGAA | 5 | 6 |
| Total, other adverse events | Cohort 2, Duvoglustat 100 mg + rhGAA | 3 | 6 |
| Total, other adverse events | Cohort 3, Duvoglustat 250 mg + rhGAA | 4 | 6 |
| Total, other adverse events | Cohort 4, Duvoglustat 600 mg + rhGAA | 4 | 7 |
| Total, serious adverse events | Cohort 1, Duvoglustat 50 mg + rhGAA | 0 | 6 |
| Total, serious adverse events | Cohort 2, Duvoglustat 100 mg + rhGAA | 1 | 6 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01380743
GET https://conductscience.org/api/trials/v1/NCT01380743/results?page=2