Phase 3 N=39 Randomized Double-blind Treatment

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

Mucopolysaccharidosis VI

Bottom Line

View on ClinicalTrials.gov: NCT00067470 ↗

Enrolled (actual)

Serious AEs

18.0%

Results posted

Aug 2009

Primary outcome: Primary: Change From Baseline in 12-minute Walk Test at 24 Weeks — 109; 26 meters — p=0.025

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Placebo (Drug); N-acetylgalactosamine 4-sulfatase (Drug)
Age: Pediatric, Adult, Older Adult · 7+ yrs
Sex: All
Sponsor: BioMarin Pharmaceutical
Primary completion: —

Outcome Measures

Outcome	Result	p-value
PRIMARY Change From Baseline in 12-minute Walk Test at 24 Weeks	109; 26	0.025 sig
SECONDARY Change From Baseline in 3-minute Stair Climb at 24 Weeks	7.4; 2.7	0.053
SECONDARY Change From Baseline in Urinary GAG (uGAG) at 24 Weeks	346; 330; 85; 317	—

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.

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00067470). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.