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

Part of paid clinical trials in Torrance, California.

Sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Study ID
NCT03153319
Phase
PHASE1/PHASE2
Status
Active Not Recruiting

Conditions

  • Mucopolysaccharidosis I
  • Mucopolysaccharidosis II
  • Mucopolysaccharidosis VI

Eligibility Criteria

Sex
ALL
Age
5 Years - N/A
Healthy Volunteers
Not accepted

Interventions

Study Details

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.

Key Dates

Start date
Jun 5, 2017
Status verified
Jan 2026
Primary completion
Jan 31, 2027
Completion
Feb 28, 2027

Study Design

Enrollment
14 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Adalimumab
    20 mg subQ every other week (weight 15to \<30 kg) 40 mg subQ every other week (weight ≥30 kg). Non-responders will be escalated to weekly dosing.
  • Placebo Comparator: Placebo
    Saline placebo comparator
  • Experimental: Open-label adalimumab
    Open-label extension of adalimumab dose

Primary Outcome Measure

Pain - 16 weeks [ Time Frame: 16 weeks ]

Locations (1)

FacilityCityStateZIPSite coordinators
The Lundquist Institute at Harbor-UCLA Medical CenterTorranceCalifornia90502-

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By research site
The Lundquist Institute at Harbor-UCLA Medical Center· Torrance, CA

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