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Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI
Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Summary
Randomized, double-blind, placebo-controlled, parallel-group, single-center study followed by open-label phase, to evaluate the effects of adalimumab compared to placebo on the change from baseline in joint and skeletal disease in children and adults with mucopolysaccharidosis (MPS) I, II or VI.
Official title: Phase 1/2 Study of the Effect of Adalimumab on Physical Function and Musculoskeletal Disease in Mucopolysaccharidosis Types I, II, and VI
Key Details
Gender
All
Age Range
5 Years - Any
Study Type
INTERVENTIONAL
Enrollment
14
Start Date
2017-06-05
Completion Date
2027-02
Last Updated
2026-02-02
Healthy Volunteers
No
Interventions
Adalimumab Injection [Humira]
Investigational Drug
Saline Solution for Injection
Placebo Comparator
Locations (1)
The Lundquist Institute at Harbor-UCLA Medical Center
Torrance, California, United States