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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 is a Phase 4 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 4 participants.
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00701129
The ClinicalTrials.gov registry entry for NCT00701129 describes a study currently listed as completed, categorized as Phase 4. The registered enrollment target is 4 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Genzyme, a Sanofi Company, which has 196 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 3 interventions - of which Rituximab is the first listed.
NCT00701129 reports 0 study locations.
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.
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