Trial · NCT02782741
Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease
NCT02782741, Phase 3, Completed, Genzyme, a Sanofi Company; results first posted 2021-04-08
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease. Trials, record NCT02782741. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02782741
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease
- NCT number
- NCT02782741
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 101 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
- 2016-05-25
- Last updated
- 2024-04-04
- Other registries' IDs
- eudract:2016-000942-77
- Results first posted
- 2021-04-08
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | PAP: Change From Baseline in Percent Predicted FVC in Upright Position at Week 49 | Baseline, Week 49 | FVC is a standard pulmonary function test used to quantify respiratory muscle weakness. FVC is the volume of air (in liters) that can be forcibly blown out after full inspiration in the upright position. Least square (LS) mean and standard error (SE) were derived from mixed model for repeated measure (MMRM) model with baseline FVC \[percent (%) predicted, as continuous\], sex, age (in years at baseline), treatment group, visit, interaction term between treatment group and visit as fixed effects. Percent of predicted FVC = (actual FVC measurement)/(predicted value of FVC) \* 100. After non-inferiority (NI) testing, a test for superiority of avalglucosidase alfa versus alglucosidase alfa was performed with an overall 2-sided 5% level of significance. |
| Secondary | PAP: Change From Baseline in Total Distance Walked During Six-minute Walk Test (6MWT) at Week 49 | Baseline, Week 49 | 6MWT was a standardized test that measured the distance (in meters) covered by the participant by walking on a flat, hard surface in a period of a 6-minute walk. Mean distance walked gives an indication of functional endurance. The greater the distance (that a participant could walk in 6 minutes), the greater the endurance. LS mean and SE were derived from MMRM model with baseline FVC (% predicted) and baseline 6MWT (distance walked in meter), age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects. |
| Secondary | PAP: Change From Baseline in Percent Predicted Maximal Inspiratory Pressure (MIP) in Upright Position at Week 49 | Baseline, Week 49 | MIP is a quick and non-invasive test to measure strength of inspiratory muscles, primarily diaphragm, and allows for assessment of ventilatory failure, restrictive lung disease and respiratory muscle strength. MIP refers to how much air pressure force an individual creates by inhaling through the mouth as hard as possible. LS mean and SE were derived from MMRM model for MIP % predicted adjusted for MIP % predicted at baseline, age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects. |
| Secondary | PAP: Change From Baseline in Percent Predicted Maximal Expiratory Pressure (MEP) in Upright Position at Week 49 | Baseline, Week 49 | MEP is a quick and non-invasive test to measure strength of expiratory muscles, primarily diaphragm, and allows for assessment of ventilatory failure, restrictive lung disease and respiratory muscle strength. MEP is the greater pressure generated during maximal expiration. LS mean and SE were derived from MMRM model for MEP % predicted adjusted for MEP % predicted at baseline, age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects. |
| Secondary | PAP: Change From Baseline in Lower Extremity Muscle Strength at Week 49 as Assessed by Hand-Held Dynamometry (HHD) | Baseline, Week 49 | HHD: a portable method for strength quantitation. To complete a make test, participant exerted maximal force against dynamometer with gradual increase in force and completed isometric hold for 4-5 seconds. Muscle strengths were collected in Newton. Every muscle group (hip: flexion, extension, abduction; knee: flexion, extension and ankle dorsiflexion) were measured 2 times and highest value was reported. Summary score was sum of 12 measurements (2 measurements per muscle group) from 6 muscle groups on each side (left and right). An increase from Baseline was reflective of increased muscle strength, whereas a decrease from Baseline was reflective of decreased muscle strength. LS mean and SE were derived from MMRM model for HHD lower extremity muscle strength composite score adjusted for summary HHD lower extremity score at baseline, baseline FVC (% predicted), age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects. |
| Secondary | PAP: Change From Baseline in Quick Motor Function Test (QMFT) Total Scores at Week 49 | Baseline, Week 49 | The QMFT was an observer administered test to evaluate changes in motor function. QMFT comprised of 16 items specifically difficult for participants with Pompe disease. Each item was scored separately on a 5-point ordinal scale (ranged from 0 to 4, higher score indicated better outcome). Total QMFT score was obtained by adding the scores of all items and ranged from 0 (unable to perform motor function tests) to 64 (normal muscle function), higher score represented better outcome. LS mean and SE were derived from MMRM models adjusted for total QMFT score at baseline, baseline FVC (% predicted), age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects. |
| Secondary | PAP: Change From Baseline in 12-Item Short-Form Health Survey (SF-12): Physical Component Summary (PCS) and Mental Component Summary (MCS) Scores at Week 49 | Baseline, Week 49 | SF-12, a 12 item-questionnaire, used to assess health-related quality of life in participants aged \>=18 years at screening/baseline. SF-12 consisted of 12 items, which were categorized into eight domains (subscales) of functioning and well-being: physical functioning, role-physical, role emotional, mental health, bodily pain, general health, vitality and social functioning, with each domain score ranged from 0 (poor health) to 100 (better health), higher scores indicated good health condition. These eight domains were further summarized into 2 summary scores, PCS and MCS. The score range for each of these 2 summary scores was from 0 (poor health) to 100 (better health), higher scores indicated a better health-related quality of life. LS mean and SE were derived from MMRM models adjusted for baseline score (PCS or MCS), baseline FVC (% predicted), age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects. |
| Secondary | PAP: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Infusion-Associated Reactions (IARs) | From Baseline up to Week 49 | AE: any untoward medical occurrence in participant who took study drug and not necessarily have to had causal relationship with treatment. TEAEs: AEs that developed/worsened in grade/became serious during TEAE period in PAP (from time of 1st treatment date to last treatment date+4 weeks for participants who didn't receive any treatment in open-label or to time just prior to 1st treatment in open-label for participants who received treatment in open-label). Protocol-defined IARs: AE of special interest (AESIs) that occurred during either infusion/observation period following infusion which were deemed to be related/possibly related to study drug. Algorithm-defined IARs: any TEAE meeting either 1 of 2 criteria: 1) event occurred from start to end of infusion + 24 hours, considered related to study drug, 2) If AE time component missed, compare AE start date with infusion start and end date. If AE start date was between infusion start and end date + 1 day and it was related to study drug. |
| Secondary | Open-label Period: Number of Participants With TEAEs and IARs | Week 50 to 289 in open-label long-term period | AE: any untoward medical occurrence in a participant who received study drug and did not necessarily have to had a causal relationship with treatment. TEAEs in open-label: AEs that developed/worsened in grade/became serious during TEAE period in open-label (from time of 1st open-label treatment to last treatment date + 4 weeks). Protocol-defined IARs: defined as AESIs that occurred during either infusion/observation period following infusion which were deemed to be related/possibly related to study drug. Algorithm-defined IARs: any TEAE meeting either 1 of 2 criteria: 1) event occurred from start to end of infusion plus 24 hours, considered related to study drug, 2) If AE time component missed, compare AE start date with infusion start and end date. If AE start date was between infusion start and end date plus 1 day and it was related to study drug. |
| Secondary | PAP: Percentage of Participants With Treatment-Emergent Antidrug Antibodies (ADA) Response | From Baseline up to Week 49 | ADA response categories: 1) Treatment-induced: ADAs developed following administration of the study drug. If the baseline ADA sample was missing or non-reportable and at least one reportable on-treatment ADA sample was available, the baseline sample was considered as "negative". 2) Treatment-boosted: Pre-existing ADAs that were boosted at least two titer steps from baseline (i.e., 4 fold increase in titers) following administration of the study drug (any time after the first drug administration). 3) Treatment emergent: combination of treatment induced and treatment boosted. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | PAP: Change From Baseline in Percent Predicted FVC in Upright Position at Week 49 | Baseline, Week 49 | Analysis was performed on mITT population. | percent predicted FVC | LEAST_SQUARES_MEAN |
| SECONDARY | PAP: Change From Baseline in Total Distance Walked During Six-minute Walk Test (6MWT) at Week 49 | Baseline, Week 49 | Analysis was performed on mITT population. | meters | LEAST_SQUARES_MEAN |
| SECONDARY | PAP: Change From Baseline in Percent Predicted Maximal Inspiratory Pressure (MIP) in Upright Position at Week 49 | Baseline, Week 49 | Analysis was performed on mITT population. | percent predicted MIP | LEAST_SQUARES_MEAN |
| SECONDARY | PAP: Change From Baseline in Percent Predicted Maximal Expiratory Pressure (MEP) in Upright Position at Week 49 | Baseline, Week 49 | Analysis was performed on mITT population. | percent predicted MEP | LEAST_SQUARES_MEAN |
| SECONDARY | PAP: Change From Baseline in Lower Extremity Muscle Strength at Week 49 as Assessed by Hand-Held Dynamometry (HHD) | Baseline, Week 49 | Analysis was performed on mITT population. | Newton | LEAST_SQUARES_MEAN |
| SECONDARY | PAP: Change From Baseline in Quick Motor Function Test (QMFT) Total Scores at Week 49 | Baseline, Week 49 | Analysis was performed on mITT population. | scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | PAP: Change From Baseline in 12-Item Short-Form Health Survey (SF-12): Physical Component Summary (PCS) and Mental Component Summary (MCS) Scores at Week 49 | Baseline, Week 49 | Analysis was performed on mITT population. Here, 'overall number of participants analyzed' = participants evaluable for this outcome measure. | scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | PAP: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Infusion-Associated Reactions (IARs) | From Baseline up to Week 49 | Analysis was performed on safety population which included participants who had received at least 1 infusion (partial or total) and were analyzed according to the treatment received. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Open-label Period: Number of Participants With TEAEs and IARs | Week 50 to 289 in open-label long-term period | Analysis was performed on safety population. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | PAP: Percentage of Participants With Treatment-Emergent Antidrug Antibodies (ADA) Response | From Baseline up to Week 49 | Analysis was performed on ADA evaluable population which consisted of 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. | percentage of participants | NUMBER |
Source ClinicalTrials.gov
Results by group37
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| PAP: Change From Baseline in Percent Predicted FVC in Upright Position at Week 49 | PAP: Avalglucosidase Alfa | 2.89 | Standard Error 0.88 | percent predicted FVC | |
| PAP: Change From Baseline in Percent Predicted FVC in Upright Position at Week 49 | PAP: Alglucosidase Alfa | 0.46 | Standard Error 0.93 | percent predicted FVC | |
| PAP: Change From Baseline in Total Distance Walked During Six-minute Walk Test (6MWT) at Week 49 | PAP: Avalglucosidase Alfa | 32.21 | Standard Error 9.93 | meters | |
| PAP: Change From Baseline in Total Distance Walked During Six-minute Walk Test (6MWT) at Week 49 | PAP: Alglucosidase Alfa | 2.19 | Standard Error 10.40 | meters | |
| PAP: Change From Baseline in Percent Predicted Maximal Inspiratory Pressure (MIP) in Upright Position at Week 49 | PAP: Avalglucosidase Alfa | 0.17 | Standard Error 3.60 | percent predicted MIP | |
| PAP: Change From Baseline in Percent Predicted Maximal Inspiratory Pressure (MIP) in Upright Position at Week 49 | PAP: Alglucosidase Alfa | -2.96 | Standard Error 3.79 | percent predicted MIP | |
| PAP: Change From Baseline in Percent Predicted Maximal Expiratory Pressure (MEP) in Upright Position at Week 49 | PAP: Avalglucosidase Alfa | 3.02 | Standard Error 3.87 | percent predicted MEP | |
| PAP: Change From Baseline in Percent Predicted Maximal Expiratory Pressure (MEP) in Upright Position at Week 49 | PAP: Alglucosidase Alfa | 4.95 | Standard Error 4.07 | percent predicted MEP | |
| PAP: Change From Baseline in Lower Extremity Muscle Strength at Week 49 as Assessed by Hand-Held Dynamometry (HHD) | PAP: Avalglucosidase Alfa | 260.69 | Standard Error 46.07 | Newton | |
| PAP: Change From Baseline in Lower Extremity Muscle Strength at Week 49 as Assessed by Hand-Held Dynamometry (HHD) | PAP: Alglucosidase Alfa | 153.72 | Standard Error 48.54 | Newton |
1 to 10 of 37Source ClinicalTrials.gov
See all 37 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Blinded Treatment Period: up to Week 49 | STARTED | Avalglucosidase Alfa | 51 | |
| Blinded Treatment Period: up to Week 49 | STARTED | Alglucosidase Alfa in PAP Then Avalglucosidase Alfa in Open-label | 49 | |
| Blinded Treatment Period: up to Week 49 | STARTED | Avalglucosidase Alfa in Open-label Only | 0 | |
| Blinded Treatment Period: up to Week 49 | Safety Population | Avalglucosidase Alfa | 51 | |
| Blinded Treatment Period: up to Week 49 | Safety Population | Alglucosidase Alfa in PAP Then Avalglucosidase Alfa in Open-label | 49 | |
| Blinded Treatment Period: up to Week 49 | Safety Population | Avalglucosidase Alfa in Open-label Only | 0 | |
| Blinded Treatment Period: up to Week 49 | COMPLETED | Avalglucosidase Alfa | 51 | |
| Blinded Treatment Period: up to Week 49 | COMPLETED | Alglucosidase Alfa in PAP Then Avalglucosidase Alfa in Open-label | 44 | |
| Blinded Treatment Period: up to Week 49 | COMPLETED | Avalglucosidase Alfa in Open-label Only | 0 | |
| Blinded Treatment Period: up to Week 49 | NOT COMPLETED | Avalglucosidase Alfa | 0 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events516
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 | Bronchitis | Infections and infestations | PAP: Avalglucosidase Alfa | 0 | 51 | 0 |
| other | Bronchitis | Infections and infestations | PAP: Alglucosidase Alfa | 2 | 49 | 2 |
| other | Bronchitis | Infections and infestations | Open-label: Avalglucosidase Alfa | 3 | 52 | 3 |
| other | Bronchitis | Infections and infestations | Open-label: Alglucosidase Alfa-PAP Then Avalglucosidase Alfa | 4 | 44 | 4 |
| other | Covid-19 | Infections and infestations | PAP: Avalglucosidase Alfa | 0 | 51 | 0 |
| other | Covid-19 | Infections and infestations | PAP: Alglucosidase Alfa | 0 | 49 | 0 |
| other | Covid-19 | Infections and infestations | Open-label: Avalglucosidase Alfa | 16 | 52 | 17 |
| other | Covid-19 | Infections and infestations | Open-label: Alglucosidase Alfa-PAP Then Avalglucosidase Alfa | 9 | 44 | 9 |
| other | Cystitis | Infections and infestations | PAP: Avalglucosidase Alfa | 3 | 51 | 4 |
| other | Cystitis | Infections and infestations | PAP: Alglucosidase Alfa | 0 | 49 | 0 |
1 to 10 of 516Source ClinicalTrials.gov
See all 516 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | PAP: Avalglucosidase Alfa | 0 | 51 |
| Total, all-cause mortality | PAP: Alglucosidase Alfa | 1 | 49 |
| Total, all-cause mortality | Open-label: Avalglucosidase Alfa | 0 | 52 |
| Total, all-cause mortality | Open-label: Alglucosidase Alfa-PAP Then Avalglucosidase Alfa | 2 | 44 |
| Total, other adverse events | PAP: Avalglucosidase Alfa | 40 | 51 |
| Total, other adverse events | PAP: Alglucosidase Alfa | 44 | 49 |
| Total, other adverse events | Open-label: Avalglucosidase Alfa | 50 | 52 |
| Total, other adverse events | Open-label: Alglucosidase Alfa-PAP Then Avalglucosidase Alfa | 40 | 44 |
| Total, serious adverse events | PAP: Avalglucosidase Alfa | 8 | 51 |
| Total, serious adverse events | PAP: Alglucosidase Alfa | 12 | 49 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers4
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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