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NCT00701129 · ClinicalTrials.gov registry record · Phase 4
An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease
A Phase 4 study, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 4
- Development phase
- 4
- Enrollment target
NCT00701129: Completed Phase 4 study, sponsored by Genzyme, a Sanofi Company.
NCT00701129 is a Phase 4 study that has completed, 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
NCT00701129, a Phase 4 study, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 4
- Development phase
- 4 participants
- Enrollment target
Study Summary
The purpose of this study was to evaluate the efficacy, clinical benefits and safety of a prophylactic immunomodulatory regimen given prior to first treatment with alglucosidase alfa (Myozyme®) in patients with infantile-onset Pompe disease. The objectives were to assess the efficacy of a prophylactic immunomodulatory regimen given prior to first treatment with alglucosidase alfa, as assessed by anti-recombinant human acid alpha-glucosidase (anti-rhGAA) antibody titers, and antibodies that inhibit the activity and/or uptake of alglucosidase alfa; to evaluate the clinical benefit as measured by overall survival, ventilator-free survival, left ventricular mass index (LVMI), gross motor function and development, disability index and the incidence of adverse events (AEs), serious adverse events (SAEs), and clinical laboratory abnormalities.
Primary Outcome
Serum samples from patients were analyzed for the presence of anti-rhGAA IgG antibodies. End of study (EOS) refers to the last post baseline observation during study period (up to Week 79).
Interventions
- DRUG Rituximab
- DRUG Methotrexate
- BIOLOGICAL Alglucosidase Alfa
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2009-10 |
| Est. Completion | 2013-03 |
| Phase | Phase 4 |
What the finished NCT00701129 record still lists
NCT00701129 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 3 interventions - of which Rituximab is the first listed.
NCT00701129 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00701129 about?
NCT00701129 is a clinical study titled "An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease". The purpose of this study was to evaluate the efficacy, clinical benefits and safety of a prophylactic immunomodulatory regimen given prior to first treatment with alglucosidase alfa (Myozyme®) in patients with infantile-onset Pompe disease. The objectives were to assess the efficacy of a prophylac...
What is the current status of trial NCT00701129?
This trial is currently completed. It is a Phase 4 study. The enrollment target is 4 participants. The study started on 2009-10. Estimated completion is 2013-03.
What interventions are being tested in trial NCT00701129?
The interventions under investigation include: Rituximab (DRUG), Methotrexate (DRUG), Alglucosidase Alfa (BIOLOGICAL).
Who is sponsoring clinical trial NCT00701129?
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 NCT00701129'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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