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Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

Primary Purpose

Mucopolysaccharidosis VI

Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Placebo
N-acetylgalactosamine 4-sulfatase
Sponsored by
BioMarin Pharmaceutical
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Mucopolysaccharidosis VI

Eligibility Criteria

7 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria 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 Exclusion Criteria 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

Sites / Locations

  • BioMarin Pharmaceutical Inc.

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Active Comparator

Arm Label

Placebo

rhASB

Arm Description

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Change From Baseline in 3-minute Stair Climb at 24 Weeks
Number of stairs climbed per minute in 3 minutes at 24 weeks minus number of stairs climbed per minute in 3 minutes at baseline
Change From Baseline in Urinary GAG (uGAG) at 24 Weeks
Glycosaminoglycan (GAG) level measured in urine

Full Information

First Posted
August 20, 2003
Last Updated
December 18, 2009
Sponsor
BioMarin Pharmaceutical
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1. Study Identification

Unique Protocol Identification Number
NCT00067470
Brief Title
Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI
Study Type
Interventional

2. Study Status

Record Verification Date
December 2009
Overall Recruitment Status
Completed
Study Start Date
September 2003 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
March 2004 (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
BioMarin Pharmaceutical

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis VI

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
Double
Allocation
Randomized
Enrollment
39 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Title
rhASB
Arm Type
Active Comparator
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Type
Drug
Intervention Name(s)
N-acetylgalactosamine 4-sulfatase
Other Intervention Name(s)
rhASB
Primary Outcome Measure Information:
Title
Change From Baseline in 12-minute Walk Test at 24 Weeks
Description
Number of meters walked in 12 minutes at week 24 minus number of meters walked in 12 minutes at baseline
Time Frame
baseline and 24 weeks
Secondary Outcome Measure Information:
Title
Change From Baseline in 3-minute Stair Climb at 24 Weeks
Description
Number of stairs climbed per minute in 3 minutes at 24 weeks minus number of stairs climbed per minute in 3 minutes at baseline
Time Frame
baseline and 24 weeks
Title
Change From Baseline in Urinary GAG (uGAG) at 24 Weeks
Description
Glycosaminoglycan (GAG) level measured in urine
Time Frame
baseline and 24 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
7 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria 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 Exclusion Criteria 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
Facility Information:
Facility Name
BioMarin Pharmaceutical Inc.
City
Novato
State/Province
California
ZIP/Postal Code
94949
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
33874971
Citation
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.
Results Reference
derived
Links:
URL
http://www.bmrn.com
Description
Related Info

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Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI

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