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

  • STATUS
    Recruiting
  • End date
    Oct 14, 2022
  • participants needed
    14
  • sponsor
    Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Updated on 17 February 2021
humira
mucopolysaccharidosis
mucopolysaccharidosis type i
adalimumab injection [humira]
bodily pain

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.

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 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.

Details
Condition Hunter's Syndrome, Hurler's Syndrome, Hunter Syndrome (MPS II), mucopolysaccharidosis type i
Treatment Adalimumab Injection [Humira], Saline Solution for Injection
Clinical Study IdentifierNCT03153319
SponsorLundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Last Modified on17 February 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

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)
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

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
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