Study of rhASB in Patients With Mucopolysaccharidosis VI

Mucopolysaccharidosis VI Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00104234
• Other study ID #: ASB-03-06
• Status: Completed
• Phase: Phase 3
• First received: February 24, 2005
• Last updated: January 26, 2010
• Start date: February 2004
• Est. completion date: October 2006

Study information

• Verified date: January 2010
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 39
• Est. completion date: October 2006
• Est. primary completion date: October 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 7 Years and older

Eligibility

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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis VI

Intervention

Drug:
• N-acetylgalactosamine 4-sulfatase

• Placebo/rhASB

Locations

Country	Name	City	State
United States	BioMarin Pharmaceutical Inc.	Novato	California

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	12-Minute Walk Test		Baseline of ASB-03-05 through week 96 of ASB-03-06	No
Secondary	3-Minute Stair Climb		Baseline ASB-03-05 through week 96 of ASB-03-06.	No
Secondary	Change in Urinary Glycosaminoglycans (GAG) Level		72 weeks	No