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NCT00701701 · ClinicalTrials.gov registry record · Phase 4
Immune Tolerance Induction Study
A Phase 4 study, sponsored by Genzyme, a Sanofi Company.
- Terminated
- Registry status
- Phase 4
- Development phase
- 4
- Enrollment target
NCT00701701: Clinical Trial Phase 4 study, sponsored by Genzyme, a Sanofi Company.
NCT00701701 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
NCT00701701, 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
An exploratory, open-labeled study of participants with Pompe disease, who had previously received Myozyme® (alglucosidase alfa) treatment, to evaluate the efficacy, safety and clinical benefit of 2 Immune Tolerance Induction (ITI) regimens in combination with Myozyme®. Eligible participants who were then receiving Myozyme® therapy were enrolled into the study, and were followed for a minimum of 18 months on-study (a 6-month ITI treatment module and a 12-month follow-up module on Myozyme® alone). Eligible participants were followed for a minimum of 18 months on treatment or, if a participant was \<6 months of age at the time of enrollment, until the participant was 2 years of age. Both cross-reacting immunologic material (CRIM)-negative and CRIM-positive participants were eligible for Regimen A depending if they met the required criteria. Regimen B, however, was limited to CRIM-negative participants.
Primary Outcome
An Adverse event (AE) was defined as any undesirable physical, psychological, or behavioral effect experienced by participant during his/her participation in an investigational study, in conjunction with the use of the drug or biologic, whether or not product-related. TEAEs were defined as AEs that occurred or worsened during the on-treatment period (time from the start of investigational medicinal product \[IMP\] administration up to 18 months). Serious AE (SAE) was any AE that resulted in any
Interventions
- BIOLOGICAL Myozyme® (alglucosidase alfa)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2008-12-14 |
| Est. Completion | 2020-02-18 |
| Phase | Phase 4 |
Why NCT00701701 stopped before completion
NCT00701701 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 Myozyme® (alglucosidase alfa) is the first listed.
NCT00701701 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00701701 about?
NCT00701701 is a clinical study titled "Immune Tolerance Induction Study". An exploratory, open-labeled study of participants with Pompe disease, who had previously received Myozyme® (alglucosidase alfa) treatment, to evaluate the efficacy, safety and clinical benefit of 2 Immune Tolerance Induction (ITI) regimens in combination with Myozyme®. Eligible participants who wer...
What is the current status of trial NCT00701701?
This trial is currently terminated. It is a Phase 4 study. The enrollment target is 4 participants. The study started on 2008-12-14. Estimated completion is 2020-02-18.
What interventions are being tested in trial NCT00701701?
The interventions under investigation include: Myozyme® (alglucosidase alfa) (BIOLOGICAL).
Who is sponsoring clinical trial NCT00701701?
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 NCT00701701'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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