Trial · NCT03019406
A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa
NCT03019406, Phase 2, Active not recruiting, Genzyme, a Sanofi Company; results first posted 2021-11-22
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa. Trials, record NCT03019406. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03019406
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa
- NCT number
- NCT03019406
- Status
- Active not recruiting
- Phases
- Phase 2
- Enrollment
- 22 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Glycogen Storage Disease Type II-Pompe's Disease
- Interventions
- Avalglucosidase alfa (GZ402666), Alglucosidase alfa (GZ419829)
- First posted
- 2017-01-12
- Last updated
- 2026-01-30
- Other registries' IDs
- eudract:2016-003475-21
- Results first posted
- 2021-11-22
Source ClinicalTrials.gov
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| 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 | Adverse event (AE): any untoward medical occurrence in participant received study drug \& did not necessarily had to have causal relationship with treatment. TEAEs: AEs developed/worsened in grade/become serious during PAP period (from the time of 1st study drug dose up to Week 25). Serious AE(SAE): any untoward medical occurrence at any dose resulted in death, was life-threatening, required inpatient hospitalization, prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was congenital anomaly/birth defect or was medically important event. TEAEs included SAEs \& non-SAEs. AESI:AE (serious/non-serious) of scientific \& medical concern specific to Sponsor's product/program, for which ongoing monitoring \& immediate notification by Investigator to Sponsor required. |
| Primary | PAP: Number of Participants With Infusion-associated Reactions (IARs) | From Baseline to Week 25 | IARs were defined as AESIs that occurred during either the infusion or the observation period following the infusion which were deemed to be related or possibly related to the study drug. Protocol-defined IARs: An AESIs that occurred during either the infusion or the observation period following the infusion which were deemed to be related or possibly related to study drug. Algorithm-defined IARs: any TEAE meeting either 1 or 2 criteria: 1) event occurred from the start of infusion to the end of infusion plus 24 hours, and considered related to study drug, 2) If an AE time component was missing, compared AE Start date with infusion start date and infusion end date. If an AE Start date was between infusion start date and infusion end date plus one day, and it was related to study drug. |
| Secondary | PAP: Number of Participants With Anti-drug Antibody (ADA) Response | From Baseline to Week 25 | Anti-drug antibody response was categorized as: Treatment induced ADAs: ADAs developed de novo (seroconversion) following administration of study drug. Treatment boosted ADAs: pre-existing ADAs that were boosted at least two titer steps from Baseline (i.e., 4-fold increase in titers) followed by administration of study drug. |
| 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 | Cmax is the maximum observed plasma concentration. |
| 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 | Tmax is the time to achieve maximum plasma concentration. |
| 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 | AUC0-last is the area under the plasma concentration versus time curve from time 0 to the time of last quantifiable concentration. |
| 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 | t1/2 is the time required for the plasma concentration of a drug to decrease by half of its initial concentration. |
| 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 | CL is defined as a quantitative measure of the rate at which a drug substance is removed from the body. |
| 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 | Steady state volume of distribution (Vss) is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. |
| Secondary | PAP: Change From Baseline in Gross Motor Function Measure-88 (GMFM-88) Test Scores at Week 25 | Baseline, Week 25 | GMFM-88 was developed specifically to detect quantitative changes in gross motor function. The GMFM-88 consisted of 88 items organized into 5 dimensions; lying and rolling (17 items); sitting (20 items); crawling and kneeling (14 items); standing (13 items) and walking, running and jumping (24 items). Each item was scored on a 4-point Likert scale with scores range: 0= cannot do; 1 = initiates less then \[\<\] 10 percentage \[%\] of the task; 2 = partially completes \[10% to \<100% of the task\] and 3 = task completion. The score for each dimension was expressed as a % of the maximum score for that dimension.Total percentage score was obtained by adding the percentage scores for each dimension and dividing the sum by the total number of dimensions. Total scores ranged from 0% to 100%; where higher scores indicated better motor functions. |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 registered outcomesOutcomes20
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| 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 |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 outcomesResults by group156
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI) | PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg | Any TEAEs | 5 | Participants | |
| PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI) | PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg | Any TEAEs | 5 | Participants | |
| PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI) | PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg | Any TEAEs | 5 | Participants | |
| PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI) | PAP: Cohort 3b: Alglucosidase Alfa | Any TEAEs | 5 | Participants | |
| PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI) | PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg | Any Serious TEAEs | 1 | Participants | |
| PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI) | PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg | Any Serious TEAEs | 3 | Participants | |
| PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI) | PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg | Any Serious TEAEs | 0 | Participants | |
| PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI) | PAP: Cohort 3b: Alglucosidase Alfa | Any Serious TEAEs | 2 | Participants | |
| PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI) | PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg | Any AESI | 0 | Participants | |
| PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI) | PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg | Any AESI | 2 | Participants |
1 to 10 of 156Source ClinicalTrials.gov
See all 156 results by groupParticipant flow28
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| PAP: up to 25 Weeks | STARTED | Cohort 1: Avalglucosidase Alfa 20 mg/kg | 6 | |
| PAP: up to 25 Weeks | STARTED | Cohort 2: Avalglucosidase Alfa 40 mg/kg | 5 | |
| PAP: up to 25 Weeks | STARTED | Cohort 3a: Avalglucosidase Alfa 40 mg//kg | 5 | |
| PAP: up to 25 Weeks | STARTED | Cohort 3b: Alglucosidase Alfa in PAP | 6 | |
| PAP: up to 25 Weeks | Treated | Cohort 1: Avalglucosidase Alfa 20 mg/kg | 6 | |
| PAP: up to 25 Weeks | Treated | Cohort 2: Avalglucosidase Alfa 40 mg/kg | 5 | |
| PAP: up to 25 Weeks | Treated | Cohort 3a: Avalglucosidase Alfa 40 mg//kg | 5 | |
| PAP: up to 25 Weeks | Treated | Cohort 3b: Alglucosidase Alfa in PAP | 6 | |
| PAP: up to 25 Weeks | COMPLETED | Cohort 1: Avalglucosidase Alfa 20 mg/kg | 6 | |
| PAP: up to 25 Weeks | COMPLETED | Cohort 2: Avalglucosidase Alfa 40 mg/kg | 5 |
1 to 10 of 28Source ClinicalTrials.gov
See all 28 participant flowAdverse events1,064
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 | Lymph Node Pain | Blood and lymphatic system disorders | PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg | 0 | 6 | 0 |
| other | Lymph Node Pain | Blood and lymphatic system disorders | PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg | 0 | 5 | 0 |
| other | Lymph Node Pain | Blood and lymphatic system disorders | PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg | 0 | 5 | 0 |
| other | Lymph Node Pain | Blood and lymphatic system disorders | PAP: Cohort 3b: Alglucosidase Alfa | 0 | 6 | 0 |
| other | Lymph Node Pain | Blood and lymphatic system disorders | ETP: Avalglucosidase Alfa 20 mg/kg | 1 | 6 | 1 |
| other | Lymph Node Pain | Blood and lymphatic system disorders | Pooled Arm ETP: Avalglucosidase Alfa 40 mg/kg | 0 | 10 | 0 |
| other | Lymph Node Pain | Blood and lymphatic system disorders | Alglucosidase Alfa in PAP Then Avalglucosidase Alfa 40 mg/kg in ETP | 0 | 3 | 0 |
| other | Neutropenia | Blood and lymphatic system disorders | PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg | 0 | 6 | 0 |
| other | Neutropenia | Blood and lymphatic system disorders | PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg | 1 | 5 | 1 |
| other | Neutropenia | Blood and lymphatic system disorders | PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg | 0 | 5 | 0 |
1 to 10 of 1,064Source ClinicalTrials.gov
See all 1,064 adverse eventsAdverse event totals21
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg | 0 | 6 |
| Total, all-cause mortality | PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg | 0 | 5 |
| Total, all-cause mortality | PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg | 0 | 5 |
| Total, all-cause mortality | PAP: Cohort 3b: Alglucosidase Alfa | 0 | 6 |
| Total, all-cause mortality | ETP: Avalglucosidase Alfa 20 mg/kg | 0 | 6 |
| Total, all-cause mortality | Pooled Arm ETP: Avalglucosidase Alfa 40 mg/kg | 0 | 10 |
| Total, all-cause mortality | Alglucosidase Alfa in PAP Then Avalglucosidase Alfa 40 mg/kg in ETP | 0 | 3 |
| Total, other adverse events | PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg | 5 | 6 |
| Total, other adverse events | PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg | 5 | 5 |
| Total, other adverse events | PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg | 5 | 5 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 adverse event totalsPapers2
The papers the trial lists on ClinicalTrials.gov.
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.
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