Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI (NCT03153319) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI
United States14 participantsStarted 2017-06-05
Plain-language 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.
Who can participate
Age range5 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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:
* 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;
* …
What they're measuring
1
Pain - 16 weeks
Timeframe: 16 weeks
2
Adalimumab trough
Timeframe: 32 weeks
Trial details
NCT IDNCT03153319
SponsorLundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center