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OpenTrials
Completed

NCT Number: NCT00067470

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.

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Key information

Conditions

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

Age range

7 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

BioMarin Pharmaceutical Inc.

Novato, California, 94949, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patient consent
  • Patient must be seven years of age or older
  • Patient must have documented biochemical or genetic proof of MPS VI
  • The patient must be able to walk independently at least 5 meters and no more than 270 meters in the first 6 minutes, or no more then 400 meters total in 12 minutes, in the screening 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)
  • Patient refuses or is unable to complete all screening evaluations
  • 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
  • The patient has clinically significant spinal cord compression
  • The patient has known hypersensitivity to rhASB or to components of the active or placebo test solutions

Treatment and study plan

Placebo

Drug

N-acetylgalactosamine 4-sulfatase

Drug

Other names: rhASB

Primary outcomes

  1. Change From Baseline in 12-minute Walk Test at 24 Weeks

    Time frame: baseline and 24 weeks

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

Secondary outcomes

  1. Change From Baseline in 3-minute Stair Climb at 24 Weeks

    Time frame: baseline and 24 weeks

    Number of stairs climbed per minute in 3 minutes at 24 weeks minus number of stairs climbed per minute in 3 minutes at baseline

  2. Change From Baseline in Urinary GAG (uGAG) at 24 Weeks

    Time frame: baseline and 24 weeks

    Glycosaminoglycan (GAG) level measured in urine

Sponsors and collaborators

Lead sponsor

BioMarin Pharmaceutical

Industry

Registry information

Important dates

Study start
2003
Study completion
2004
First posted
Aug 25, 2003
Registry last updated
Dec 24, 2009

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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