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Monoclonal Antibodies

Adalimumab for Mucopolysaccharidosis

Phase 1 & 2
Waitlist Available
Led By Lynda Polgreen, MD
Research Sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Weight ≥15 kg
Significant bodily pain reported by the CHQ-PF50 or SF-36 (> 1 SD more severe [below] than the general population mean)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 52 weeks
Awards & highlights

Study Summary

This trial is studying how well adalimumab works compared to placebo in treating patients with MPS I, II, or VI.

Who is the study for?
This trial is for children and adults over 5 years old with Hurler Syndrome, Hunter Syndrome, or Maroteaux-Lamy Syndrome who have either been treated with ERT for at least a year or not treated at all. Participants must have joint motion limitations in at least 3 joints, weigh more than 15 kg, and experience significant pain.Check my eligibility
What is being tested?
The study tests the safety and effectiveness of Adalimumab injections compared to saline solution (placebo) on joint and skeletal disease in patients with MPS I, II or VI. It's randomized and double-blind: participants don't know if they're getting the real drug or placebo.See study design
What are the potential side effects?
Adalimumab may cause allergic reactions, increased risk of infections including TB, potential heart issues like congestive heart failure, liver problems such as Hepatitis B reactivation, nervous system disorders like multiple sclerosis or Guillain-Barre syndrome.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I weigh at least 15 kilograms.
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I experience more pain than most people do.
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I have been diagnosed with MPS I, II, or VI.
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I have (or have not) been on enzyme replacement therapy for at least a year.
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I have 3 or more joints with limited movement.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~52 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 52 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Adalimumab trough
Pain - 16 weeks
Secondary outcome measures
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability
Joint range-of-motion - 16 weeks
Joint range-of-motion - 52 weeks
+1 more

Trial Design

3Treatment groups
Experimental Treatment
Placebo Group
Group I: Open-label adalimumabExperimental Treatment1 Intervention
Open-label extension of adalimumab dose
Group II: AdalimumabExperimental Treatment1 Intervention
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.
Group III: PlaceboPlacebo Group1 Intervention
Saline placebo comparator

Find a Location

Who is running the clinical trial?

Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical CenterLead Sponsor
103 Previous Clinical Trials
46,459 Total Patients Enrolled
Lynda Polgreen, MDPrincipal InvestigatorThe Lundquist Institute at Harbor-UCLA Medical Center

Media Library

Adalimumab (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT03153319 — Phase 1 & 2
Hunter Syndrome Research Study Groups: Adalimumab, Open-label adalimumab, Placebo
Hunter Syndrome Clinical Trial 2023: Adalimumab Highlights & Side Effects. Trial Name: NCT03153319 — Phase 1 & 2
Adalimumab (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03153319 — Phase 1 & 2
~2 spots leftby Dec 2025
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