Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

2002-03-31

Study Completion Date

2006-06-30

Brief Summary

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The purpose of the study is evaluate the efficacy, safety, and pharmacokinetics of weekly intravenous infusions of 1 mg/kg recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) in patients diagnosed with Mucopolysaccharidosis VI (MPS VI)

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Detailed Description

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Conditions

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Mucopolysaccharidosis VI

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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N-acetylgalactosamine 4-sulfatase

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Patient consent
* Patient must be five years of age or older
* Patient must have documented biochemical or genetic proof of MPS VI
* Patient must walk at least 1 meter, but less than 250 meters, in the first 6 minutes of the baseline 12-minute walk test
* If female of childbearing potential, patient must have a negative pregnancy test

Exclusion Criteria

* Patient is under consideration for or has undergone a successful bone marrow transplant (BMT).
* Pregnant or lactating patient
* Patient has received an investigational drug within 30 days prior to study enrollment
* Patient has been previously treated with rhASB
* Patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that may significantly confound study results or decrease study compliance
* Patient has a known hypersensitivity to rhASB or to components of the study drug
* History of cancer (except low grade and fully resolved skin malignancy)

Minimum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No