Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI
Public ClinicalTrials.gov record NCT01521429. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Longitudinal Study of Bone and Endocrine Disease in Children with MPS I, II, and VI: a Multicenter Study of the Lysosomal Disease Network.
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT01521429
- Recruitment status
- Completed
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 55 participants
Conditions and interventions
Conditions
Interventions
Not listed
Eligibility (public fields only)
- Age range
- 5 Years to 35 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 31, 2009
- Primary completion
- Jul 30, 2019
- Completion
- Jul 30, 2019
- Last update posted
- Oct 21, 2024
2009 – 2019
United States locations
- U.S. sites
- 3
- U.S. states
- 2
- U.S. cities
- 3
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children's Hospital & Research Center Oakland | Oakland | California | 94609 | — |
| Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center | Torrance | California | 90502 | — |
| University of Minnesota | Minneapolis | Minnesota | 55454 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01521429, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 21, 2024 · Synced Sep 10, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT01521429 live on ClinicalTrials.gov.