💬In plain words
What the trial is, and whether it could suit you
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
Sign in to get the eligibility rules as simple questions to take to your doctor.
📋 Eligibility rules (as published)
Inclusion Criteria:
* Diagnosis of Mucopolysaccharidosis type I Hurler-Scheie or Scheie syndrome.
* Age ≥ 10 years at time of study registration.
* Creatinine clearance, calculated or measured directly, that is \>60ml/min/1.73m2.
* Ejection fraction ≥ 40% by echocardiogram.
* Must commit to traveling to the study site for the necessary follow-up evaluations.
* Must agree to stay \<45-minute drive from the study site for a minimum of 5 days after cell infusion.
Exclusion Criteria:
* Known familial inherited cancer syndrome. Suspected cases will be investigated, per the physicians discretion, using relevant genetic tests to determine presence of germline mutations.
* History of B cell related cancer, EBV lymphoproliferative disease or autoimmune disorders.
* Evidence of active graft-vs-host disease.
* Underwent a previous hematopoietic stem cell transplant (HSCT).
* Requirement for systemic immune suppression.
* Requirement for continuous supplemental oxygen.
* Any medical condition likely to interfere with assessment of safety or efficacy of the study treatment.
* In the investigator's judgement, the subject is unlikely to complete all protocol-required study visits or procedures, including follow up visits, or comply with the study requirements for participation.
Other protocol defined inclusion/exclusion criteria may apply.
💊
What it tests
- Autologous Plasmablasts (B cells)
UCSF Benioff Children's Hospital Oakland
Oakland, United States
University of Minnesota
Minneapolis, United States
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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