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

NCT03153319 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2026-02-02

No results posted yet for this study

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.

Conditions

  • Mucopolysaccharidosis I
  • Mucopolysaccharidosis II
  • Mucopolysaccharidosis VI

Interventions

DRUG

Adalimumab Injection [Humira]

Investigational Drug

DRUG

Saline Solution for Injection

Placebo Comparator

Sponsors & Collaborators

  • McGill University Health Centre/Research Institute of the McGill University Health Centre

    collaborator OTHER

  • Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

    lead OTHER

Principal Investigators

  • Lynda Polgreen, MD · The Lundquist Institute at Harbor-UCLA Medical Center

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-06-05
Primary Completion
2027-01-31
Completion
2027-02-28
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03153319 on ClinicalTrials.gov