Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT00104234 · ClinicalTrials.gov registry record · Phase 3
Study of rhASB in Patients With Mucopolysaccharidosis VI
A Phase 3 study of Mucopolysaccharidosis VI, sponsored by BioMarin Pharmaceutical.
- Completed
- Registry status
- Phase 3
- Development phase
- 39
- Enrollment target
- 1
- Study location
NCT00104234: Completed Phase 3 study of Mucopolysaccharidosis VI, sponsored by BioMarin Pharmaceutical.
NCT00104234 is a Phase 3 study of Mucopolysaccharidosis VI that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 39 participants, below the 62-participant average among 6 other Mucopolysaccharidosis VI trials with a reported enrollment target (37% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00104234, a Phase 3 study of Mucopolysaccharidosis VI, has completed, sponsored by BioMarin Pharmaceutical.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 39 participants
- Enrollment target
- 1
- Study location
Study Summary
The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.
Primary Outcome
Mean change in meters walked in 12 minutes. Mean change is the mean difference between the 12-Minute Walk Test at 96 weeks and that measured before first ever treatment with rhASB. For the rhASB/rhASB group, mean change is calculated for Week 96 - Baseline. For the placebo/rhASB group, mean change is calculated for Week 96 - Week 24.
Conditions Studied
Interventions
- DRUG N-acetylgalactosamine 4-sulfatase
- DRUG Placebo/rhASB
Study Locations (1)
California
- BioMarin Pharmaceutical Inc. - Novato
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 39 participants |
| Start Date | 2004-02 |
| Est. Completion | 2006-10 |
| Phase | Phase 3 |
What the finished NCT00104234 record still lists
NCT00104234 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 39 participants, a relatively small participant target, below the 62-participant average among 6 other Mucopolysaccharidosis VI trials with a reported enrollment target (37% lower).
The record links to 1 condition, with Mucopolysaccharidosis VI appearing as the primary indexed condition, and to 2 interventions - of which N-acetylgalactosamine 4-sulfatase is the first listed.
NCT00104234 reports a single indexed study location in California.
Frequently Asked Questions
What is clinical trial NCT00104234 about?
NCT00104234 is a clinical study titled "Study of rhASB in Patients With Mucopolysaccharidosis VI". The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.
What is the current status of trial NCT00104234?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 39 participants. The study started on 2004-02. Estimated completion is 2006-10.
What conditions does trial NCT00104234 study?
This clinical trial studies the following conditions: Mucopolysaccharidosis VI.
What interventions are being tested in trial NCT00104234?
The interventions under investigation include: N-acetylgalactosamine 4-sulfatase (DRUG), Placebo/rhASB (DRUG).
Who is sponsoring clinical trial NCT00104234?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00104234 being conducted?
This trial has 1 study location across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Mucopolysaccharidosis VI
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
Registry of Patients Diagnosed With Lysosomal Storage Diseases
RECRUITING
-
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI
ACTIVE NOT RECRUITING · Phase 1
-
Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
COMPLETED
-
Stem Cell Transplantation for Hurler
COMPLETED · Phase 2
-
Human Placental-Derived Stem Cell Transplantation
COMPLETED · Phase 1
-
A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
COMPLETED · Phase 4
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
- NCT03465592 · 39 participants · Phase 1
Trial of Nivolumab Following Partially Human Leukocyte Antigen (HLA) Mismatched BMT in Children & Adults With Sarcoma
- NCT03961776 · 39 participants
Monitoring Treatment Response With On-board DWI During Neo-adjuvant Chemo-radiation for Rectal Cancer Using Magnetic Resonance-guided-radiotherapy Systems
- NCT04050280 · 39 participants · Phase 2
CLAG-GO for Patients With Persistent, Relapsed or Refractory AML
- NCT04190862 · 39 participants · Phase 1
E-CEL UVEC as an Adjunct Cell Therapy for Treatment of Anal Fistulas
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