Trial · NCT01597596
A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
NCT01597596, Phase 4, Terminated, Genzyme, a Sanofi Company; results first posted 2016-01-18
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease. Trials, record NCT01597596. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01597596
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
- NCT number
- NCT01597596
- Status
- Terminated
- Phases
- Phase 4
- Enrollment
- 4 participants, actual
- Lead sponsor
- Genzyme, a Sanofi Company
- Conditions
- Pompe Disease (Infantile-Onset), Glycogen Storage Disease Type II (GSD II), Glycogenosis 2, Acid Maltase Deficiency
- Interventions
- alglucosidase alfa, alglucosidase alfa
- First posted
- 2012-05-14
- Last updated
- 2016-01-18
- Other registries' IDs
- eudract:2011-005595-42
- Results first posted
- 2016-01-18
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Cardiac Function at Week 52 | Baseline, Week 52 | Cardiac function was measured by the left ventricular mass Z-score (LVM-Z). Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline indicates an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. |
| Secondary | Percentage of Participants With Estimated Probability of Survival | Up to Week 52 | |
| Secondary | Number of Participants With Invasive Ventilator-Free Survival | Up to Week 52 | Invasive ventilator-free survival was defined as the time during which the participant is alive and not invasively ventilated. Number of Participants with invasive ventilator-free survival were reported. |
| Secondary | Change From Baseline in Motor Development Status at Week 52 | Baseline, Week 52 | Motor development status was assessed by the Gross Motor Function Measure - 88 Scale (GMFM-88) total percent scores. GMFM-88 is an 88-item measure to detect gross motor function. It consists of 5 categories: lying and rolling; sitting; crawling and kneeling; standing; walking, running and jumping. Each item was scored on a 4-point Likert scale (0 = cannot do; 1 = initiates \[\<10% of the task\]; 2 = partially completes \[10% to \<100% of the task\]; 3 = task completion). The score for each dimension was expressed as a percentage of the maximum score for that dimension. Total score ranges from 0% to 100%, where higher scores indicate better motor functions. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Cardiac Function at Week 52 | Baseline, Week 52 | Full analysis population defined as all participants who receive at least 1 infusion of alglucosidase alfa. For this endpoint no participants were analyzed in 'Algucosidase Alfa 4000 L material' arm at Baseline and Week 52. One participant from 'Algucosidase Alfa 160 L Material' arm was discontinued from study at Week 31 due to physician's decision | Z-score | MEAN |
| SECONDARY | Percentage of Participants With Estimated Probability of Survival | Up to Week 52 | Full analysis population. | percentage of participants | NUMBER |
| SECONDARY | Number of Participants With Invasive Ventilator-Free Survival | Up to Week 52 | Full analysis population. | participants | NUMBER |
| SECONDARY | Change From Baseline in Motor Development Status at Week 52 | Baseline, Week 52 | Full analysis population. For this endpoint no participants were analyzed in 'Algucosidase Alfa 4000 L material' arm at Baseline and Week 52. One participant from 'Algucosidase Alfa 160 L Material' arm was discontinued from study at Week 31 due to physician's decision. | percentage of maximum total score | MEAN |
Source ClinicalTrials.gov
Results by group6
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Cardiac Function at Week 52 | Alglucosidase Alfa 160 L Material (US Participants) | -5.06 | Standard Deviation 1.103 | Z-score | |
| Percentage of Participants With Estimated Probability of Survival | Alglucosidase Alfa 4000 L Material (Non-US Participants) | 100 | percentage of participants | ||
| Percentage of Participants With Estimated Probability of Survival | Alglucosidase Alfa 160 L Material (US Participants) | 100 | percentage of participants | ||
| Number of Participants With Invasive Ventilator-Free Survival | Alglucosidase Alfa 4000 L Material (Non-US Participants) | 1 | participants | ||
| Number of Participants With Invasive Ventilator-Free Survival | Alglucosidase Alfa 160 L Material (US Participants) | 2 | participants | ||
| Change From Baseline in Motor Development Status at Week 52 | Alglucosidase Alfa 160 L Material (US Participants) | 48.65 | Standard Deviation 17.183 | percentage of maximum total score |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Alglucosidase Alfa 4000 L Material (Non-US Participants) | 1 | |
| Overall Study | STARTED | Alglucosidase Alfa 160 L Material (US Participants) | 3 | |
| Overall Study | COMPLETED | Alglucosidase Alfa 4000 L Material (Non-US Participants) | 0 | |
| Overall Study | COMPLETED | Alglucosidase Alfa 160 L Material (US Participants) | 2 | |
| Overall Study | NOT COMPLETED | Alglucosidase Alfa 4000 L Material (Non-US Participants) | 1 | |
| Overall Study | NOT COMPLETED | Alglucosidase Alfa 160 L Material (US Participants) | 1 |
Source ClinicalTrials.gov
Adverse events100
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 | Anaemia | Blood and lymphatic system disorders | Alglucosidase Alfa 4000 L Material (Non-US Participants) | 0 | 1 | |
| other | Anaemia | Blood and lymphatic system disorders | Alglucosidase Alfa 160 L Material (US Participants) | 1 | 3 | |
| other | Cardiac failure | Cardiac disorders | Alglucosidase Alfa 4000 L Material (Non-US Participants) | 0 | 1 | |
| other | Cardiac failure | Cardiac disorders | Alglucosidase Alfa 160 L Material (US Participants) | 2 | 3 | |
| other | Cardiac failure congestive | Cardiac disorders | Alglucosidase Alfa 4000 L Material (Non-US Participants) | 0 | 1 | |
| other | Cardiac failure congestive | Cardiac disorders | Alglucosidase Alfa 160 L Material (US Participants) | 1 | 3 | |
| other | Cardiomyopathy | Cardiac disorders | Alglucosidase Alfa 4000 L Material (Non-US Participants) | 0 | 1 | |
| other | Cardiomyopathy | Cardiac disorders | Alglucosidase Alfa 160 L Material (US Participants) | 1 | 3 | |
| other | Nodal rhythm | Cardiac disorders | Alglucosidase Alfa 4000 L Material (Non-US Participants) | 0 | 1 | |
| other | Nodal rhythm | Cardiac disorders | Alglucosidase Alfa 160 L Material (US Participants) | 1 | 3 |
1 to 10 of 100Source ClinicalTrials.gov
See all 100 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Alglucosidase Alfa 4000 L Material (Non-US Participants) | ||
| Total, all-cause mortality | Alglucosidase Alfa 160 L Material (US Participants) | ||
| Total, other adverse events | Alglucosidase Alfa 4000 L Material (Non-US Participants) | 1 | 1 |
| Total, other adverse events | Alglucosidase Alfa 160 L Material (US Participants) | 3 | 3 |
| Total, serious adverse events | Alglucosidase Alfa 4000 L Material (Non-US Participants) | 0 | 1 |
| Total, serious adverse events | Alglucosidase Alfa 160 L Material (US Participants) | 3 | 3 |
Source ClinicalTrials.gov
Papers0
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Alglucosidase Alfa 4000 L Material (Non-US Participants) vs Alglucosidase Alfa 160 L Material (US Participants): Number of Participants With Invasive Ventilator-Free Survival | Trial comparisons |
| Alglucosidase Alfa 4000 L Material (Non-US Participants) vs Alglucosidase Alfa 160 L Material (US Participants): serious adverse events | Trial comparisons |
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/NCT01597596
GET https://conductscience.org/api/trials/v1/NCT01597596/events?page=2