NCT00067470

Brief Summary

The purpose of the study is to evaluate the ability of rhASB versus placebo to enhance endurance in patients with Mucopolysaccharidosis VI (MPS VI), as evidenced by an increase in the number of meters walked in the 12 minute walk test at Week 24 compared with baseline.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
39

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Sep 2003

Shorter than P25 for phase_3

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

First Submitted

Initial submission to the registry

August 20, 2003

Completed
5 days until next milestone

First Posted

Study publicly available on registry

August 25, 2003

Completed
7 days until next milestone

Study Start

First participant enrolled

September 1, 2003

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2004

Completed
5.5 years until next milestone

Results Posted

Study results publicly available

August 17, 2009

Completed
Last Updated

December 24, 2009

Status Verified

December 1, 2009

First QC Date

August 20, 2003

Results QC Date

February 4, 2009

Last Update Submit

December 18, 2009

Conditions

Mucopolysaccharidosis VI

Outcome Measures

Primary Outcomes (1)

  • Change From Baseline in 12-minute Walk Test at 24 Weeks

    Number of meters walked in 12 minutes at week 24 minus number of meters walked in 12 minutes at baseline

    baseline and 24 weeks

Secondary Outcomes (2)

  • Change From Baseline in 3-minute Stair Climb at 24 Weeks

    baseline and 24 weeks

  • Change From Baseline in Urinary GAG (uGAG) at 24 Weeks

    baseline and 24 weeks

Study Arms (2)

Placebo

PLACEBO COMPARATOR
Drug: Placebo

rhASB

ACTIVE COMPARATOR
Drug: N-acetylgalactosamine 4-sulfatase

Interventions

PlaceboDRUG
Placebo
N-acetylgalactosamine 4-sulfataseDRUG
Also known as: rhASB
rhASB

Eligibility Criteria

Age7 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Patient consent
  • Patient must be seven years of age or older
  • Patient must have documented biochemical or genetic proof of MPS VI
  • The patient must be able to walk independently at least 5 meters and no more than 270 meters in the first 6 minutes, or no more then 400 meters total in 12 minutes, in the screening 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)
  • Patient refuses or is unable to complete all screening evaluations
  • 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
  • The patient has clinically significant spinal cord compression
  • The patient has known hypersensitivity to rhASB or to components of the active or placebo test solutions

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

BioMarin Pharmaceutical Inc.

Novato, California, 94949, United States

Location

Related Publications (1)

  • Tandon PK, Kakkis ED. The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases. Orphanet J Rare Dis. 2021 Apr 19;16(1):183. doi: 10.1186/s13023-021-01805-5.

    PMID: 33874971DERIVED

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

Results Point of Contact

Title
Medical Information Services
Organization
BioMarin Pharmaceutical Inc.
medinfo@bmrn.com
800-983-4587

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
DOUBLE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

August 20, 2003

First Posted

August 25, 2003

Study Start

September 1, 2003

Study Completion

March 1, 2004

Last Updated

December 24, 2009

Results First Posted

August 17, 2009

Record last verified: 2009-12

Locations

US(1)