MPS IVA Clinical Trial
Official title:
Gait Analysis in Patients With MPS IVA Treated With Enzyme Replacement Therapy
The purpose of this study is to learn about gait (walking) characteristics in MPS IVA and possible changes in gait with enzyme replacement therapy.
The purpose of this 3-year study is to conduct a longitudinal investigation using motion
analysis of gait to objectively and quantitatively assess gait characteristics of MPS IVA
patients. Data documenting gait characteristics and abnormalities will be gathered in
patients initiating enzyme replacement therapy.
Inclusion criteria Individuals with MPS IVA diagnosed by enzyme or molecular studies
Exclusion Criteria Individual with MPS IVA previously enrolled in BMN-110 Individual is
non-ambulatory The individual is unable to complete longitudinal study visits at least yearly
;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT05845749 -
Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI
|
Phase 1/Phase 2 | |
| Completed |
NCT02294877 -
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
|
||
| Completed |
NCT01961518 -
Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome
|
N/A | |
| Completed |
NCT01515956 -
Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
|
Phase 2 | |
| Recruiting |
NCT05284006 -
Non-invasive Functional Assessment and Pathogenesis of Morquio A
|
||
| Completed |
NCT01415427 -
Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
|
Phase 3 | |
| Recruiting |
NCT04532047 -
In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases
|
Phase 1 | |
| 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 |