Study of rhASB in Patients With Mucopolysaccharidosis VI

This study has been completed.

Sponsor:

Information provided by:

BioMarin Pharmaceutical

ClinicalTrials.gov Identifier:

NCT00104234

First received: February 24, 2005

Last updated: January 26, 2010

Last verified: January 2010

History of Changes

Purpose

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

Condition	Intervention	Phase
Mucopolysaccharidosis VI	Drug: N-acetylgalactosamine 4-sulfatase Drug: Placebo/rhASB	Phase 3


Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	A Multicenter, Multinational Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With Mucopolysaccharidosis VI

Resource links provided by NLM:

Genetics Home Reference related topics: Schindler disease mucopolysaccharidosis type VI

Genetic and Rare Diseases Information Center resources: Mucopolysaccharidosis Mucopolysaccharidosis Type VI

U.S. FDA Resources

Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:

12-Minute Walk Test [ Time Frame: Baseline of ASB-03-05 through week 96 of ASB-03-06 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

3-Minute Stair Climb [ Time Frame: Baseline ASB-03-05 through week 96 of ASB-03-06. ] [ Designated as safety issue: No ]
Change in Urinary Glycosaminoglycans (GAG) Level [ Time Frame: 72 weeks ] [ Designated as safety issue: No ]


Enrollment:	39
Study Start Date:	February 2004
Study Completion Date:	October 2006
Primary Completion Date:	October 2006 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
rhASB/rhASB N-acetylgalactosamine 4-sulfatase	Drug: N-acetylgalactosamine 4-sulfatase Other Name: rhASB
Placebo/rhASB	Drug: Placebo/rhASB

Eligibility


Ages Eligible for Study:	7 Years and older (Child, Adult, Senior)
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00104234

Locations


United States, California
BioMarin Pharmaceutical Inc.
Novato, California, United States, 94949

Sponsors and Collaborators

BioMarin Pharmaceutical

More Information


ClinicalTrials.gov Identifier:	NCT00104234 History of Changes
Other Study ID Numbers:	ASB-03-06
Study First Received:	February 24, 2005
Results First Received:	February 4, 2009
Last Updated:	January 26, 2010
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:


Mucopolysaccharidoses Mucopolysaccharidosis VI Carbohydrate Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn	Lysosomal Storage Diseases Mucinoses Connective Tissue Diseases Metabolic Diseases

ClinicalTrials.gov processed this record on September 23, 2016

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