Summary

Eligibility
for people ages 5 years and up (full criteria)
Location
at UCLA
Dates
study started
estimated completion
Principal Investigator
Lynda Polgreen, MD (ucla)

Description

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 or II.

Official Title

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

Details

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 or II. Children and adults diagnosed with MPS I or II, 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.

Keywords

Mucopolysaccharidosis I Mucopolysaccharidosis II Mucopolysaccharidoses Adalimumab Adalimumab Injection [Humira] Saline Solution for Injection Open-label adalimumab

Eligibility

You can join if…

Open to people ages 5 years and up

  • Male or female ≥5 years of age;
  • Diagnosis of MPS I or II;
  • 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.

You CAN'T join if...

  • 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.

Location

  • Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center accepting new patients
    Torrance California 90502 United States

Lead Scientist at UC Health

  • Lynda Polgreen, MD (ucla)
    Assistant Professor-in-Residence, Pediatrics. Authored (or co-authored) 44 research publications.

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
ID
NCT03153319
Phase
Phase 1/2
Study Type
Interventional
Last Updated