A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
This multicenter, open-label study is designed to assess safety, dose-response using pharmacokinetic (PK) and pharmacodynamic (PD) measures, and clinical efficacy of BMN 110 in subjects between 5 and 18 years of age, diagnosed with Mucopolysaccharidosis IVA (MPS IVA).
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase 1/2, Multicenter, Open-label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)|
- Subject Incidence of Treatment Emergent AEs [ Time Frame: Entire Study, through week 84 ] [ Designated as safety issue: Yes ]
The primary objective of the study was to evaluate the safety of weekly infusions of BMN 110 administered in escalating doses to subjects with MPS IVA.
The safety variable incidence of TEAE is summarized.
- Change From Baseline in 6MWT [ Time Frame: Baseline to Weeks 12, 24, 36, 48, 72 ] [ Designated as safety issue: No ]Change from baseline in meters in 6-minute Walk Test. As a measure of endurance, a 6-minute walk test (6MWT) was performed according to the American Thoracic Society Guidelines. Patients were instructed to walk as far as possible in 6 minutes.
- Change From Baseline in 3MSCT [ Time Frame: Baseline to Weeks 12, 24, 36, 48, 72 ] [ Designated as safety issue: No ]Change from baseline in the 3-minute Stair Climb Test. Patients walked up stairs that have a railing, which could be used for support, for 3 minutes, with the number of stairs climbed recorded. The test result was the number of steps climbed per minute.
- Percent Change From Baseline in uKS [ Time Frame: Baseline to Weeks 12, 24, 36, 72 ] [ Designated as safety issue: No ]Percent Change from baseline in Normalized Urine KS. The percent change was calculated (Week X value - baseline value)/baseline value *100%
- Percent Change From Baseline in MVV [ Time Frame: Baseline to Weeks 12, 24, 36, 72 ] [ Designated as safety issue: No ]Percent Change from baseline in Maximum Voluntary Ventilation.
- Percent Change From Baseline in FVC [ Time Frame: Baseline to Weeks 12, 24, 36, 72 ] [ Designated as safety issue: No ]Percent Change from baseline in Forced Vital Capacity.
|Study Start Date:||April 2009|
|Study Completion Date:||March 2011|
|Primary Completion Date:||February 2011 (Final data collection date for primary outcome measure)|
Experimental: BMN 110
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:
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00884949
|Birmingham, United Kingdom|
|London, United Kingdom|
|Manchester, United Kingdom|
|Study Director:||Celeste Decker, MD||BioMarin Pharmceutical Inc.|