Cell therapy for MPS I to improve immune function
Part of Genetic & congenital, Hormones & metabolism, Skin clinical trials.
This Phase 1 study tests an engineered cell therapy made to help the body in people with MPS I (Hurler-Scheie or Scheie). It is early-stage research, so the main goal is to learn about safety and the best way to treat.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have been diagnosed with MPS I Hurler-Scheie or Scheie syndrome
- You are at least 10 years old when you enroll
- Your kidneys are working well enough (creatinine clearance over 60 ml/min/1.73m²)
- Your heart pumping is okay (ejection fraction of 40% or higher on an ultrasound/echo)
- You can travel to the study site for follow-up visits
- After the cell infusion, you can stay within a 45-minute drive for at least 5 days
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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