Study 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

PHASE1

Study Classification

INTERVENTIONAL

Study Start Date

2000-09-30

Study Completion Date

2005-11-30

Brief Summary

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The purpose of the study is to evaluate the safety, efficacy and pharmacokinetics of two dose levels of weekly intravenous infusions of recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) for a minimum of 24 weeks in patients diagnosed with MPS VI.

Detailed Description

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Conditions

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Mucopolysaccharidosis VI

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

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 diagnosis of MPS VI, confirmed at screening by measurable clinical signs and symptoms of MPS VI
* Leukocyte ASB enzyme activity level less than 20% of the normal range
* Clinical evidence of significant MPS VI disease that provides adequate opportunity to achieve quantitative, short-term therapeutic benefit in three or more of the following parameters: endurance (as measured by a six-minute walk test), forced vital capacity (as measured by spirometry), joint range of motion, urinary glycosaminoglycans, and hepatomegaly.
* Ability to perform all protocol tests
* Ability to stand independently for six minutes
* Sexually active subjects must agree to use an adequate form of contraception

Exclusion Criteria

* History of bone marrow transplantation
* Pregnant or lactating patient
* Use of an investigational drug or device within 30 days prior to study participation.
* A medical condition, serious intercurrent illness, or other extenuating circumstances that may significantly decrease study compliance including prescribed follow-up
* Known hypersensitivity to rhASB or to components of the study drug
* History of cancer (except low grade and fully resolved skin malignancy)

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Principal Investigators

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Stuart J Swiedler, MD, Ph.D.

Role: STUDY_DIRECTOR

BioMarin Pharmaceutical

Locations

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BioMarin Pharmaceutical Inc.

Novato, California, United States

Site Status

Countries

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United States

Other Identifiers

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ASB-00-01

Identifier Type: -

Identifier Source: org_study_id