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Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI

NCT01521429 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 55

Last updated 2024-10-22

No results posted yet for this 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.

Conditions

  • Mucopolysaccharidoses

Sponsors & Collaborators

  • Rare Diseases Clinical Research Network

    collaborator NETWORK

  • National Institute of Neurological Disorders and Stroke (NINDS)

    collaborator NIH

  • National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

    collaborator NIH

  • UCSF Benioff Children's Hospital Oakland

    collaborator OTHER

  • National Center for Advancing Translational Sciences (NCATS)

    collaborator NIH

  • Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

    lead OTHER

Principal Investigators

  • Lynda E Polgreen, MD, MS · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

Eligibility

Min Age
5 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-08-31
Primary Completion
2019-07-31
Completion
2019-07-31

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01521429 on ClinicalTrials.gov