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

MPS IV A Clinical Trial

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)

Status Completed

Clinical Trial 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).

Clinical Trial Description

n/a

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT00884949
Study type	Interventional
Source	BioMarin Pharmaceutical
Contact
Status	Completed
Phase	Phase 1/Phase 2
Start date	April 2009
Completion date	March 2011

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See also
Status	Clinical Trial	Phase
Terminated	`NCT00787995` - A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)	N/A
Active, not recruiting	`NCT03632213` - Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI	Phase 2
Terminated	`NCT01242111` - A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)	Phase 1/Phase 2
Completed	`NCT01275066` - A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)	Phase 3