NCT03153319

Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI

Phase 1/2 Study of the Effect of Adalimumab on Physical Function and Musculoskeletal Disease in Mucopolysaccharidosis Types I, II, and VI

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 14 (estimated)
• Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center · Academic / Other
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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.

Conditions

• Mucopolysaccharidosis I
• Mucopolysaccharidosis II
• Mucopolysaccharidosis VI

Interventions

Type	Name	Description
DRUG	Adalimumab Injection [Humira]	Investigational Drug
DRUG	Saline Solution for Injection	Placebo Comparator

Timeline

• Start date: 2017-06-05
• Primary completion: 2027-01-01
• Completion: 2027-02-01
• First posted: 2017-05-15
• Last updated: 2026-02-02

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study