A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
|ClinicalTrials.gov Identifier: NCT00884949|
Recruitment Status : Completed
First Posted : April 21, 2009
Results First Posted : June 30, 2014
Last Update Posted : June 30, 2014
|Condition or disease||Intervention/treatment||Phase|
|MPS IV A||Drug: BMN 110||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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)|
|Study Start Date :||April 2009|
|Primary Completion Date :||February 2011|
|Study Completion Date :||March 2011|
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.
- Subject Incidence of Treatment Emergent AEs [ Time Frame: Entire Study, through week 84 ]
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 ]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 ]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 ]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 ]Percent Change from baseline in Maximum Voluntary Ventilation.
- Percent Change From Baseline in FVC [ Time Frame: Baseline to Weeks 12, 24, 36, 72 ]Percent Change from baseline in Forced Vital Capacity.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00884949
|Birmingham, United Kingdom|
|London, United Kingdom|
|Manchester, United Kingdom|
|Study Director:||Celeste Decker, MD||BioMarin Pharmceutical Inc.|