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NCT00067470 · ClinicalTrials.gov registry record · Phase 3

Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

A Phase 3 study, sponsored by BioMarin Pharmaceutical.

Completed
Registry status
Phase 3
Development phase
39
Enrollment target

NCT00067470: Completed Phase 3 study, sponsored by BioMarin Pharmaceutical.

NCT00067470 is a Phase 3 study that has completed, run by BioMarin Pharmaceutical. The registered enrollment target is 39 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (95% lower). According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT00067470, a Phase 3 study, has completed, sponsored by BioMarin Pharmaceutical.

COMPLETED
Registry status
Phase 3
Development phase
39 participants
Enrollment target

Study Summary

The purpose of the study is to evaluate the ability of rhASB versus placebo to enhance endurance in patients with Mucopolysaccharidosis VI (MPS VI), as evidenced by an increase in the number of meters walked in the 12 minute walk test at Week 24 compared with baseline.

Primary Outcome

Number of meters walked in 12 minutes at week 24 minus number of meters walked in 12 minutes at baseline

Interventions

  • DRUG Placebo
  • DRUG N-acetylgalactosamine 4-sulfatase

Trial Details

FieldValue
Enrollment Target 39 participants
Start Date 2003-09
Est. Completion 2004-03
Phase Phase 3
BioMarin Pharmaceutical

80 total trials

What the finished NCT00067470 record still lists

NCT00067470 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 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (95% lower).

The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.

NCT00067470 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT00067470 about?

NCT00067470 is a clinical study titled "Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI". The purpose of the study is to evaluate the ability of rhASB versus placebo to enhance endurance in patients with Mucopolysaccharidosis VI (MPS VI), as evidenced by an increase in the number of meters walked in the 12 minute walk test at Week 24 compared with baseline.

What is the current status of trial NCT00067470?

This trial is currently completed. It is a Phase 3 study. The enrollment target is 39 participants. The study started on 2003-09. Estimated completion is 2004-03.

What interventions are being tested in trial NCT00067470?

The interventions under investigation include: Placebo (DRUG), N-acetylgalactosamine 4-sulfatase (DRUG).

Who is sponsoring clinical trial NCT00067470?

This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT00067470's enrollment target sits among peer trials

39 1898th of 2000 higher than 101 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

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Source: ClinicalTrials.gov NCT00067470, the US trial registry maintained by the National Library of Medicine. NCT00067470 (small enrollment · none site footprint · completed) retrieved and formatted by PlainTrial, see methodology.