CClinicalTrials.gg
Sign In
Active, not recruitingNCT03153319Updated Feb 2, 2026

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

A Phase 1/2 interventional study of Adalimumab Injection [Humira] and Saline Solution for Injection in Mucopolysaccharidosis I, Mucopolysaccharidosis II and Mucopolysaccharidosis VI, sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center. Active, not recruiting at 1 site in United States. Open to participants aged 5 Years and older. Per ClinicalTrials.gov, last updated 2026-02-02.

Sponsored by Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center · Phase 1/2, Interventional, and Treatment

Phase
Phase 1/2
Study type
Interventional
Enrollment
14
Allocation
Randomized
Ages
5 Years and older
Sex
All
01

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.

Read the 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.

02

Conditions studied

  • Mucopolysaccharidosis I
  • Mucopolysaccharidosis II
  • Mucopolysaccharidosis VI
03

Who can participate

Ages eligible
5 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Male or female ≥5 years of age;
  • Diagnosis of MPS I, II or VI;
  • Treatment with ERT for ≥1 year or no treatment with ERT for ≥1 year;
  • Weight ≥15 kg;
  • Significant bodily pain reported by the CHQ-PF50 or SF-36 (> 1 SD more severe [below] than the general population mean);
  • ≥ 3 joints with limitations in motion; and Patient or parent/legal guardian is able and willing to provide informed consent. For patients 7 to 17 years of age, assent must also be provided.

Exclusion criteria

Exclusion Criteria:

  • History of HCT less than 2 years prior to enrollment;
  • Immune suppression therapy less than 1 year prior to enrollment;
  • Active graft versus host disease;
  • Current diagnosis or history of lymphoma or other malignancy;
  • Current active infection;
  • History of serious opportunistic infection (e.g., bacterial [Legionella and Listeria]; tuberculosis [TB]; invasive fungal infections; or viral, parasitic, and other opportunistic infections);
  • Positive TB skin test, positive Quantiferon-TB Gold TB test, positive chest X-ray, or a recent exposure to TB
  • Congestive heart failure defined by an ejection fracture \<50% measured by ECHO;
  • Demyelinating disorders (e.g., central nervous system [CNS] disorders including multiple sclerosis and optic neuritis and peripheral nervous system disorders including Guillain-Barre syndrome);
  • Hematologic abnormalities (e.g., pancytopenia, aplastic anemia);
  • Hepatitis B infection (active or chronic carrier);
  • Latex sensitivity;
  • Pregnancy or breastfeeding;
  • Known or suspected allergy to adalimumab or related products;
  • Participation in simultaneous therapeutic study that involves an investigational study drug or agent within 4 weeks of study enrollment;
  • Requirement for live vaccine exposure that would be expected to occur during the time frame of the study; or
  • Any other social or medical condition that the Investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated or be detrimental to the study.
04

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
14 participants (estimated)

Study 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.

    Drug: Adalimumab Injection [Humira]

  • Placebo comparator
    Placebo

    Saline placebo comparator

    Drug: Saline Solution for Injection

  • Experimental
    Open-label adalimumab

    Open-label extension of adalimumab dose

    Drug: Adalimumab Injection [Humira]

Interventions

  • DrugAdalimumab Injection [Humira]

    Investigational Drug

  • DrugSaline Solution for Injection

    Placebo Comparator

05

What researchers measure

Primary outcomes

  1. 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

  2. Adalimumab trough

    Percentage of subjects who achieve a goal trough concentration of adalimumab with every other week dosing

    Time frame: 32 weeks

Secondary outcomes

  1. 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

  2. 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

  3. 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

  4. Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability

    Percentage of subjects who develop an AE and/or SAE

    Time frame: 52 weeks

06

Study locations

1 site
  • The Lundquist Institute at Harbor-UCLA Medical Center
    Torrance, California 90502, United States
07

Registry details

Key details

Study ID
NCT03153319
Lead sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Collaborators
McGill University Health Centre/Research Institute of the McGill University Health Centre
Responsible party
Lynda E Polgreen (Principal Investigator, Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center) — Principal investigator
First posted
May 15, 2017
Start date
Jun 5, 2017
Primary completion
Jan 2027 (estimated)
Completion
Feb 2027 (estimated)
Last update
Feb 2, 2026

Study contacts

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

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Jan 2026. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Mucopolysaccharidosis I→

Other studies from this sponsor

Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

Start the discussion