Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI

NCT00048620 | Completed Phase 1

• 1 other identifier: ASB-00-01
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Mucopolysaccharidosis VI

Outcome Measures

Primary Outcomes (1)

• Lysosomal storage disease

Interventions

N-acetylgalactosamine 4-sulfatase DRUG

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (1)

BioMarin Pharmaceutical Inc.

Novato, California, 94949, United States

Location

Related Links

• Related Info

MeSH Terms

Conditions

Mucopolysaccharidosis VI

Interventions

N-Acetylgalactosamine-4-Sulfatase

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Arylsulfatases; Sulfatases; Esterases; Hydrolases; Enzymes; Enzymes and Coenzymes; Chondroitinsulfatases; Chondroitinases and Chondroitin Lyases

Study Officials

• Stuart J Swiedler, MD, Ph.D.

  BioMarin Pharmaceutical

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: November 4, 2002
• First Posted: November 6, 2002
• Study Start: September 1, 2000
• Study Completion: November 1, 2005
• Last Updated: November 3, 2006

Record last verified: 2006-11

Locations

US (1)