Study of rhASB in Patients With Mucopolysaccharidosis VI

BioMarin Pharmaceutical

Status and phase: Completed; Phase 3

Conditions: Mucopolysaccharidosis VI

Treatments: Drug: N-acetylgalactosamine 4-sulfatase; Drug: Placebo/rhASB

Study type: Interventional

Funder types: Industry

Identifiers

NCT00104234

ASB-03-06

Details and patient eligibility

About

The purpose of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with Mucopolysaccharidosis VI.

Enrollment: 39 patients

Sex: All

Ages: 7+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patient consent
• Patient must have successfully completed study ASB-03-05, including having received at least 20 of 24 scheduled weekly infusions and having missed no more then two consecutive infusions
• If female of childbearing potential, patient must have a negative pregnancy test

Exclusion criteria

• Pregnant or lactating patient
• Patient has received an investigational drug within 30 days prior to study enrollment
• Patient is unwilling or unable to travel to the primary site for periodic assessments

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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39 participants in 2 patient groups

rhASB/rhASB

Other group

Description:

N-acetylgalactosamine 4-sulfatase

Treatment:

Drug: N-acetylgalactosamine 4-sulfatase

Placebo/rhASB

Other group

Treatment:

Drug: Placebo/rhASB