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

• Status: Recruiting
• Trial end date: 2022-10-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Los Angeles Biomedical Research Institute; Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
• Treatments: Adalimumab

Criteria

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);

• ≥ 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:

• 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.