Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI - Tabular View

Tracking Information

First Received Date ^ICMJE

November 6, 2002

Last Updated Date

August 24, 2009

Start Date ^ICMJE

March 2002

Primary Completion Date

Current Primary Outcome Measures ^ICMJE

12-minute walk test [ Time Frame: weeks 6, 12, 24, 48, 96 and 144 ] [ Designated as safety issue: No ]
urinary glycoaminoglycan(uGAG)levels [ Time Frame: weeks 1, 4, 6, 8, 12 ] [ Designated as safety issue: No ]

Original Primary Outcome Measures ^ICMJE

Lysosomal Storage Disease

Change History

Complete list of historical versions of study NCT00048711 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Original Secondary Outcome Measures ^ICMJE

Descriptive Information

Brief Title ^ICMJE

Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

Official Title ^ICMJE

Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI

Brief Summary

The purpose of the study is evaluate the efficacy, safety, and pharmacokinetics of weekly intravenous infusions of 1 mg/kg recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) in patients diagnosed with Mucopolysaccharidosis VI (MPS VI)

Detailed Description

Study Phase

Phase II

Study Type ^ICMJE

Interventional

Study Design ^ICMJE

Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Condition ^ICMJE

Mucopolysaccharidosis VI

Intervention ^ICMJE

Drug: N-acetylgalactosamine 4-sulfatase

Study Arms / Comparison Groups

Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Completed

Enrollment ^ICMJE

Completion Date

June 2006

Primary Completion Date

Eligibility Criteria ^ICMJE

Inclusion Criteria:

Patient consent
Patient must be five years of age or older
Patient must have documented biochemical or genetic proof of MPS VI
Patient must walk at least 1 meter, but less than 250 meters, in the first 6 minutes of the baseline 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).
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
Patient has a known hypersensitivity to rhASB or to components of the study drug
History of cancer (except low grade and fully resolved skin malignancy)

Gender

Both

Ages

5 Years and older

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

United States

Administrative Information

NCT ID ^ICMJE

NCT00048711

Responsible Party

Study ID Numbers ^ICMJE

ASB-01-04

Study Sponsor ^ICMJE

BioMarin Pharmaceutical

Collaborators ^ICMJE

Investigators ^ICMJE

Study Director:

Stuart J Sweidler, MD, Ph.D.

BioMarin Pharmaceutical

Information Provided By

BioMarin Pharmaceutical

Verification Date

August 2009

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP