A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

20

Participants

Timeline

Start Date

April 30, 2009

Primary Completion Date

February 28, 2011

Study Completion Date

March 31, 2011

Conditions
MPS IV A
Interventions
DRUG

BMN 110

"Subjects will receive a weekly 4- to 5-hour intravenous infusion of BMN 110 in 3 consecutive 12-week dosing intervals, using the following regimen:~* Weeks 1-12: 0.1 mg/kg/week~* Weeks 13-24: 1.0 mg/kg/week~* Weeks 25-36: 2.0 mg/kg/week~Subjects who complete the 36-week Dose-Escalation Period will have the option to continue drug treatment for an additional 36 to 48 weeks. Subjects continuing on treatment after the Dose-Escalation period will receive weekly 4- to 5-hour intravenous infusions of BMN 110 at a dose of 1.0 mg/kg/week."

Trial Locations (3)

Unknown

Birmingham

London

Manchester

Sponsors
All Listed Sponsors
lead

BioMarin Pharmaceutical

INDUSTRY