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NCT01597596 · ClinicalTrials.gov registry record · Phase 4
A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
A Phase 4 study, sponsored by Genzyme, a Sanofi Company.
- Terminated
- Registry status
- Phase 4
- Development phase
- 4
- Enrollment target
NCT01597596: Clinical Trial Phase 4 study, sponsored by Genzyme, a Sanofi Company.
NCT01597596 is a Phase 4 study that was terminated before completion, run by Genzyme, a Sanofi Company. The registered enrollment target is 4 participants, below the 518-participant average among 9,235 other Phase 4 trials with a reported enrollment target (99% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01597596, a Phase 4 study, was terminated before completion, sponsored by Genzyme, a Sanofi Company.
- TERMINATED
- Registry status
- Phase 4
- Development phase
- 4 participants
- Enrollment target
Study Summary
A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease. Participants were treated with alglucosidase alfa 160 L scale product in the United States (US) and 4000 L scale product in the regions outside the US.
Primary Outcome
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.
Interventions
- BIOLOGICAL alglucosidase alfa
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2012-08 |
| Est. Completion | 2014-12 |
| Phase | Phase 4 |
Why NCT01597596 stopped before completion
NCT01597596 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 4 participants, a relatively small participant target, below the 518-participant average among 9,235 other Phase 4 trials with a reported enrollment target (99% lower).
The record links to 0 conditions, and to 1 intervention - of which alglucosidase alfa is the first listed.
NCT01597596 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01597596 about?
NCT01597596 is a clinical study titled "A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease". A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease. Participants were treated with alglucosidase alfa 160 L scale product in ...
What is the current status of trial NCT01597596?
This trial is currently terminated. It is a Phase 4 study. The enrollment target is 4 participants. The study started on 2012-08. Estimated completion is 2014-12.
What interventions are being tested in trial NCT01597596?
The interventions under investigation include: alglucosidase alfa (BIOLOGICAL).
Who is sponsoring clinical trial NCT01597596?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01597596's enrollment target sits among peer trials
4 1994th of 2000 higher than 6 of 2,000 other Phase 4 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 4 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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