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 range
5 Years
Sex
ALL
See this in plain English?
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;
* …
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
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