Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI
Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis Type I focused on measuring Mucopolysaccharidosis, Inflammation, Tumor necrosis factor - alpha inhibitor
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of MPS I, II or VI;
- Treatment with ERT for ≥1 year or no ERT for ≥1 year;
- Weight ≥15 kg;
- Bodily pain reported by the CHQ-PF50 or SF-36 > 1 SD below 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 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.
Sites / Locations
- Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center
Arms of the Study
Arm 1
Arm 2
Experimental
Experimental
Adalimumab first, then Placebo
Placebo first, then Adalimumab
Participants first received Adalimumab 20 mg subQ every other week (weight 15 to <30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks. Participants then received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks.
Participants first received Placebo saline subQ (volume matching Adalimumab 20 mg or 40 mg subQ) every other week for 16 weeks. Participants then received Adalimumab 20 mg subQ every other week (weight 15 to <30 kg) or 40 mg subQ every other week (weight ≥30 kg) for 16 weeks.