Status:
COMPLETED
Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI
Lead Sponsor:
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Collaborating Sponsors:
Rare Diseases Clinical Research Network
National Institute of Neurological Disorders and Stroke (NINDS)
Conditions:
Mucopolysaccharidoses
Eligibility:
All Genders
5-35 years
Brief 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 mu...
Detailed Description
Although children with MPS I, II, and VI who are treated with hematopoietic cell transplantation (HCT) and/or enzyme replacement therapy (ERT) are now living into adulthood with good cognitive develop...
Eligibility Criteria
Inclusion
- Diagnosis of MPS I, II, or VI
- Ability to travel to study center for evaluations.
- Age ≥ 5 years and \< 35 years: age at entry into study must be ≥5 years and ≤33 years to ensure a minimum of 2 study visits.
Exclusion
- Pregnancy (will be determined at each study visit)
- Participation in any other study within the past 12 months which would result in increasing the child's radiation exposure above 500 mrem for the calendar year.
- Participants who cannot comply with study procedures or have other factors that would inhibit their participation as determined by the PI's discretion.
Key Trial Info
Start Date :
August 1 2009
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
July 31 2019
Estimated Enrollment :
55 Patients enrolled
Trial Details
Trial ID
NCT01521429
Start Date
August 1 2009
End Date
July 31 2019
Last Update
October 22 2024
Active Locations (3)
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1
Children's Hospital & Research Center Oakland
Oakland, California, United States, 94609
2
Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center
Torrance, California, United States, 90502
3
University of Minnesota
Minneapolis, Minnesota, United States, 55454