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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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 Synd

Brief Summary: 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).

Detailed Summary:
Sponsor: BioMarin Pharmaceutical

Current Primary Outcome: Subject Incidence of Treatment Emergent AEs [ Time Frame: Entire Study, through week 84 ]

Original Primary Outcome: Safety (Vital signs; Physical examination; Electrocardiogram [ECG]; Echocardiogram; Cervical spine x-ray; Clinical laboratory tests; Immunogenicity tests; Adverse event assessment; Concomitant medications) [ Time Frame: Screening, Baseline, Weeks 1-72 ]

Current Secondary Outcome:

• 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.

Original Secondary Outcome:

• Pharmacokinetic (PK) parameters [ Time Frame: Weeks 1, 12, 24, 36 ]
• Pharmacodynamic (PD) parameters [ Time Frame: Screening, Baseline, Weeks 4, 8, 10, 11, 12, 16, 20, 22, 23, 24, 28, 32, 34, 35, 36, 48, 60, 72 ]
• Clinical measures [ Time Frame: Baseline, Weeks 6, 12, 18, 24, 30, 36, 48, 60, 72 ]

Information By: BioMarin Pharmaceutical

Dates:
Date Received: April 10, 2009
Date Started: April 2009
Date Completion:
Last Updated: May 28, 2014
Last Verified: May 2014