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NCT01521429 · ClinicalTrials.gov registry record
Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI
A clinical trial, sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center.
- Completed
- Registry status
- 55
- Enrollment target
NCT01521429: Completed study, sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center.
NCT01521429 is a clinical trial that has completed, run by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center. The registered enrollment target is 55 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01521429 has completed, sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center.
- COMPLETED
- Registry status
- 55 participants
- Enrollment target
Study Summary
Approximately 85% of individuals with Mucopolysaccharidosis (MPS) type I, II, or VI report weekly pain and 50-60% have significant limitations in their activities of daily living due to MPS related musculoskeletal disease despite treatment with enzyme replacement therapy (ERT). Thus there is a critical need to identify additional therapies to alleviate the burden of musculoskeletal disease in order to improve the health and quality of life of individuals with MPS. However, disease progression needs to be quantified to be able to determine efficacy of new therapies. This study is a multi-institutional, 5-year, longitudinal study of musculoskeletal disease in MPS. The objective is to quantitatively describe the progression of skeletal disease and identify biomarkers that either predict disease severity or could be used as therapeutic targets in individuals with MPS I, II, and VI. A database of standardized measurements of musculoskeletal disease in MPS will allow the field to efficiently move forward with therapeutic clinical trials in patients with MPS.
Primary Outcome
Measurement of bone density and body composition
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 55 participants |
| Start Date | 2009-08 |
| Est. Completion | 2019-07-31 |
What the finished NCT01521429 record still lists
NCT01521429 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 55 participants, a relatively small participant target.
The record links to 0 conditions, and to 0 interventions.
NCT01521429 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01521429 about?
NCT01521429 is a clinical study titled "Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI". Approximately 85% of individuals with Mucopolysaccharidosis (MPS) type I, II, or VI report weekly pain and 50-60% have significant limitations in their activities of daily living due to MPS related musculoskeletal disease despite treatment with enzyme replacement therapy (ERT). Thus there is a criti...
What is the current status of trial NCT01521429?
This trial is currently completed. The enrollment target is 55 participants. The study started on 2009-08. Estimated completion is 2019-07-31.
Who is sponsoring clinical trial NCT01521429?
This trial is sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center, which has 55 total clinical trials registered on ClinicalTrials.gov.
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