A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Morquio A Syndrome Clinical Trial

Official title:
A Multicenter, Multinational, Open-Label, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Status Terminated

Clinical Trial Summary

This multicenter, open-label extension study is designed to assess long-term efficacy and safety of 2.0 milligrams (mg)/kilogram(kg)/week of BMN 110 in patients diagnosed with Mucopolysaccharidosis IVA (MPS IVA). Patients with MPS IVA, who enrolled in a prior BioMarin sponsored clinical study of BMN 110 (NCT00884949; Study Identification Number MOR-002), were eligible to enroll in this study (except patients who enrolled in NCT01275066; Study Identification Number MOR-004).

Clinical Trial Description

n/a

Study Design

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	NCT01242111
Study type	Interventional
Source	BioMarin Pharmaceutical
Contact
Status	Terminated
Phase	Phase 1/Phase 2
Start date	November 2010
Completion date	July 2014

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT02294877` - A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
Completed	`NCT01515956` - Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)	Phase 2
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
Recruiting	`NCT05284006` - Non-invasive Functional Assessment and Pathogenesis of Morquio A
Completed	`NCT02208661` - Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)
Completed	`NCT01415427` - Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)	Phase 3
Terminated	`NCT01697319` - Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation	Phase 2
Terminated	`NCT01609062` - Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome	Phase 2
Approved for marketing	`NCT01858103` - BMN 110 US Expanded Access Program	N/A