Trial · NCT03019406
Outcomes20
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
1 to 20 of 20
Download this page as JSON| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI) | From Baseline to Week 25 | Analysis was performed on safety population. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | PAP: Number of Participants With Infusion-associated Reactions (IARs) | From Baseline to Week 25 | Analysis was performed on safety population. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | PAP: Number of Participants With Anti-drug Antibody (ADA) Response | From Baseline to Week 25 | Analysis was performed on the ADA evaluable population which included participants who had received at least 1 infusion (partial or total) and had at least one ADA sample taken post-baseline after drug administration that was appropriate for ADA testing with a reportable result. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | PAP: Pharmacokinetic (PK) Parameter: Maximum Observed Plasma Concentration (Cmax) of Avalglucosidase Alfa | Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 and 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25 | Analysis was performed on PK population which included PAP participants who had received at least 1 infusion (partial or total) and had evaluable drug concentration data. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm. | nanograms per milliliter | MEAN |
| SECONDARY | PAP: Pharmacokinetic Parameter: Time to Achieve Maximum Observed Plasma Concentration (Tmax) of Avalglucosidase Alfa | Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 and 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25 | Analysis was performed on PK population. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm. | hours | MEDIAN |
| SECONDARY | PAP: Pharmacokinetic Parameter: Area Under the Plasma Concentration Versus Time Curve From Time 0 to the Time of Last Quantifiable Concentration (AUC0-last) of Avalglucosidase Alfa | Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 & 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25 | Analysis was performed on PK population. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm. | nanograms*hours per milliliter | MEAN |
| SECONDARY | PAP: Pharmacokinetic Parameter: Terminal Half-life (t1/2) of Avalglucosidase Alfa | Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 & 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25 | Analysis was performed on PK population. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm. | hours | MEAN |
| SECONDARY | PAP: Pharmacokinetic Parameter: Clearance (CL) of Avalglucosidase Alfa | Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 and 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25 | Analysis was performed on PK population. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm. | milliliters per hours | MEAN |
| SECONDARY | PAP: Pharmacokinetic Parameter: Volume of Distribution at Steady State (Vss) of Avalglucosidase Alfa | Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 & 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25 | Analysis was performed on PK population. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm. | milliliters | MEAN |
| SECONDARY | PAP: Change From Baseline in Gross Motor Function Measure-88 (GMFM-88) Test Scores at Week 25 | Baseline, Week 25 | Analysis was performed on safety population. Here, 'overall number of participants analyzed' = participants with available data for this OM. | percentage of total score | MEAN |
| SECONDARY | PAP: Number of Participants in Gross Motor Function Classification System-Expanded and Revised (GMFCS-E and R) Scores at Baseline and Week 25 | Baseline, Week 25 | Analysis was performed on safety population. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | PAP: Change From Baseline in Pompe Pediatric Evaluation of Disability Inventory (Pompe-PEDI) Functional Skills Scale: Mobility Domain Test Score-Scaled Score at Week 25 | Baseline, Week 25 | Analysis was performed on safety population. Here, 'overall number of participants analyzed' = participants with available data for this OM. | score on a scale | MEAN |
| SECONDARY | PAP: Change From Baseline in Quick Motor Function Test (QMFT) Scores at Week 25 | Baseline, Week 25 | Analysis was performed on safety population. Here, 'overall number of participants analyzed' = participants with available data for this OM. | score on a scale | MEAN |
| SECONDARY | PAP: Echo-Left Ventricular Mass Z-Score (LVM Z-score) M-mode at Baseline, Week 25, and Change From Baseline at Week 25 | Baseline, Week 25 | Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category. | Z-score | MEAN |
| SECONDARY | PAP: Change From Baseline in Eyelid Position Measurements: Interpalpebral Fissure Distance (IPFD) - Left Non-Flash and Right Non-Flash at Week 25 | Baseline, Week 25 | Analysis was performed on safety population. | millimeters | MEAN |
| SECONDARY | PAP: Change From Baseline in Eyelid Position Measurements: Left and Right Margin Reflex Distance (MRD) at Week 25 | Baseline, Week 25 | Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category. | millimeters | MEAN |
| SECONDARY | PAP: Change From Baseline in Eyelid Position Measurements Assessed by Margin Pupil Distance (MPD) - Left Non-Flash and Right Non-Flash at Week 25 | Baseline, Week 25 | Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category. | millimeters | MEAN |
| SECONDARY | PAP: Change From Baseline in Creatine Kinase Value at Week 25 | Baseline, Week 25 | Analysis was performed on safety population. | international units per liters | MEAN |
| SECONDARY | ETP: Number of Participants With Treatment Emergent Adverse Events, Serious Treatment Emergent Adverse Events, and Adverse Event of Special Interest | Up to 9 years and 4 months | |||
| SECONDARY | ETP: Number of Participants With Infusion-associated Reactions | Up to 9 years and 4 months |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
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.
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