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Completed

NCT Number: NCT00104234

Study of rhASB in Patients With Mucopolysaccharidosis VI

The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.

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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 have successfully completed study ASB-03-05, including having received at least 20 of 24 scheduled weekly infusions and having missed no more then two consecutive infusions
  • If female of childbearing potential, patient must have a negative pregnancy test

Exclusion criteria

  • Pregnant or lactating patient
  • Patient has received an investigational drug within 30 days prior to study enrollment
  • Patient is unwilling or unable to travel to the primary site for periodic assessments

Treatment and study plan

N-acetylgalactosamine 4-sulfatase

Drug

Other names: rhASB

Placebo/rhASB

Drug

Primary outcomes

  1. 12-Minute Walk Test

    Time frame: Baseline of ASB-03-05 through week 96 of ASB-03-06

    Mean change in meters walked in 12 minutes. Mean change is the mean difference between the 12-Minute Walk Test at 96 weeks and that measured before first ever treatment with rhASB. For the rhASB/rhASB group, mean change is calculated for Week 96 - Baseline. For the placebo/rhASB group, mean change is calculated for Week 96 - Week 24.

Secondary outcomes

  1. 3-Minute Stair Climb

    Time frame: Baseline ASB-03-05 through week 96 of ASB-03-06.

    Mean change in number of stairs climbed per minute in 3 minutes. Mean change is the mean difference between the 3-Minute Stair Climb at 96 weeks and that measured before first ever treatment with rhASB. Mean change is calculated for Week 96 - Baseline for the rhASB/rhASB group and for Week 96 - Week 24 for the placebo/rhASB group.

  2. Change in Urinary Glycosaminoglycans (GAG) Level

    Time frame: 72 weeks

    Mean change in urinary GAG level for the first 72 weeks of rhASB treatment. For the rhASB/rhASB group, mean change was calculated for Week 72 -Baseline. For the placebo/rhASB, mean change was calculated for Week 96 - Week 24.

Sponsors and collaborators

Lead sponsor

BioMarin Pharmaceutical

Industry

Registry information

Official study title

A Multicenter, Multinational Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With Mucopolysaccharidosis VI

Important dates

Study start
2004
Primary completion
2006
Study completion
2006
First posted
Feb 25, 2005
Registry last updated
Feb 2, 2010

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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