Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT00701415 · ClinicalTrials.gov registry record · Phase 3
A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms
A Phase 3 study, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 3
- Development phase
- 31
- Enrollment target
NCT00701415: Completed Phase 3 study, sponsored by Genzyme, a Sanofi Company.
NCT00701415 is a Phase 3 study that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 31 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (96% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00701415, a Phase 3 study, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 31 participants
- Enrollment target
Study Summary
The purpose of this study was to determine whether 2 alternative dosing regimens of Fabrazyme (Agalsidase beta) (1.0 mg/kg every 4 weeks or 0.5 mg/kg every 2 weeks) were effective in treatment-naïve pediatric participants without severe symptoms. Participants were to be treated for 5 years.
Primary Outcome
Skin biopsies were taken at Baseline, Week 52, Week 156 and Week 260 or early withdrawal and analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Each biopsy was scored for GL-3 accumulation on a severity score-scale of none, mild, moderate, severe (0-1-2-3). Scores are categorized as normal (score = 0) or abnormal (score = 1, 2 or 3). Data was summarized in terms of number of participants with none/trace, mild, moderate and severe biopsy scores.
Interventions
- BIOLOGICAL Agalsidase beta
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 31 participants |
| Start Date | 2008-09 |
| Est. Completion | 2015-06 |
| Phase | Phase 3 |
What the finished NCT00701415 record still lists
NCT00701415 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 31 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (96% lower).
The record links to 0 conditions, and to 1 intervention - of which Agalsidase beta is the first listed.
NCT00701415 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00701415 about?
NCT00701415 is a clinical study titled "A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms". The purpose of this study was to determine whether 2 alternative dosing regimens of Fabrazyme (Agalsidase beta) (1.0 mg/kg every 4 weeks or 0.5 mg/kg every 2 weeks) were effective in treatment-naïve pediatric participants without severe symptoms. Participants were to be treated for 5 years.
What is the current status of trial NCT00701415?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 31 participants. The study started on 2008-09. Estimated completion is 2015-06.
What interventions are being tested in trial NCT00701415?
The interventions under investigation include: Agalsidase beta (BIOLOGICAL).
Who is sponsoring clinical trial NCT00701415?
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 NCT00701415's enrollment target sits among peer trials
31 1914th of 2000 higher than 84 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 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
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT02932618 · 31 participants · Phase 3
A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)
- NCT03482297 · 31 participants · Phase 1
Automated Abdominal Binder for Orthostatic Hypotension
- NCT04016389 · 31 participants · NA
FIGO 2018 Stage IB2 Cervical Cancer Treated With Neoadjuvant Chemotherapy Followed by Fertility Sparing Surgery
- NCT04211675 · 31 participants · Phase 1
NK Cells Infusions With Irinotecan, Temozolomide, and Dinutuximab
Similar registry start date
- NCT05988463 · started 2027-01-01 · Phase 1
Dose-Escalation Study of Artesunate Patients With IPF
- NCT07219706 · started 2026-12-10 · NA
Dietary Fiber and Time Restricted Eating
- NCT02831335 · started 2026-12-01
Technical Development of Multi-Parametric Renocerebral MRI
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia