NCT00048711

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)

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Timeline
Completed

Started Mar 2002

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2002

Completed
8 months until next milestone

First Submitted

Initial submission to the registry

November 6, 2002

Completed
2 days until next milestone

First Posted

Study publicly available on registry

November 8, 2002

Completed
3.6 years until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2006

Completed
Last Updated

August 25, 2009

Status Verified

August 1, 2009

First QC Date

November 6, 2002

Last Update Submit

August 24, 2009

Conditions

Mucopolysaccharidosis VI

Outcome Measures

Primary Outcomes (2)

  • 12-minute walk test

    weeks 6, 12, 24, 48, 96 and 144

  • urinary glycoaminoglycan(uGAG)levels

    weeks 1, 4, 6, 8, 12

Interventions

N-acetylgalactosamine 4-sulfataseDRUG

Eligibility Criteria

Age5 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

BioMarin Pharmaceutical Inc.

Novato, California, 94949, United States

Location

Related Links

MeSH Terms

Conditions

Mucopolysaccharidosis VI

Interventions

N-Acetylgalactosamine-4-Sulfatase

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

ArylsulfatasesSulfatasesEsterasesHydrolasesEnzymesEnzymes and CoenzymesChondroitinsulfatasesChondroitinases and Chondroitin Lyases

Study Officials

  • Stuart J Sweidler, MD, Ph.D.

    BioMarin Pharmaceutical

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

November 6, 2002

First Posted

November 8, 2002

Study Start

March 1, 2002

Study Completion

June 1, 2006

Last Updated

August 25, 2009

Record last verified: 2009-08

Locations

US(1)