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.
This study is a 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. Children and adults diagnosed with MPS I, II or VI, with significant joint restrictions and pain will be randomized to adalimumab treatment or placebo treatment for the first 16 weeks. This will be followed by a 32-week open label adalimumab treatment phase.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
14
Investigational Drug
Placebo Comparator
The Lundquist Institute at Harbor-UCLA Medical Center
Torrance, California, United States
Pain - 16 weeks
Mean difference in bodily pain measured by the Children's Health Questionnaire - Parent Form 50 (CHQ-PF50) or the Medical Outcomes Study - Short Form 36 (SF-36) in treatment versus placebo at 16 weeks
Time frame: 16 weeks
Adalimumab trough
Percentage of subjects who achieve a goal trough concentration of adalimumab with every other week dosing
Time frame: 32 weeks
Joint range-of-motion - 16 weeks
Percentage of subjects who achieve a 5 degree or more improvement in joint range-ot-motion in treatment versus placebo at 16 weeks.
Time frame: 16 weeks
Pain - 52 weeks
Mean difference in bodily pain measured by the CHQ-PF50 or the SF-36 at 52 weeks compared to baseline.
Time frame: 52 weeks
Joint range-of-motion - 52 weeks
Percentage of subjects who achieve a 5 degree or more improvement in joint range-ot-motion at 52 weeks compared to baseline.
Time frame: 52 weeks
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability
Percentage of subjects who develop an AE and/or SAE
Time frame: 52 weeks
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