Morquio A Syndrome Clinical Trial
Official title:
A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease over time.
n/a
Observational Model: Case-Only
| 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 | |
| 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 | |
| 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 |