Status:
WITHDRAWN
MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT
Lead Sponsor:
Masonic Cancer Center, University of Minnesota
Conditions:
Mucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome)
Mucopolysaccharidosis Type IH
Eligibility:
All Genders
3-8 years
Phase:
PHASE1
PHASE2
Brief Summary
This is a single center, Phase 1/2 study in which patients with Hurler syndrome who have previously undergone allogeneic hematopoietic stem cell transplantation are treated with autologous plasmablast...
Eligibility Criteria
Inclusion
- Diagnosis of Mucopolysaccharidosis type IH (MPS IH, Hurler syndrome)
- Underwent a previous hematopoietic stem cell transplant \>1 year prior to study enrollment
- Age ≥3 years and ≤8 years at time of study registration
- ≥ 10 kilograms body weight
- Creatinine \<1.5 normal for gender and age.
- Ejection fraction ≥ 40% by echocardiogram
- Must commit to traveling to the University of Minnesota for the necessary followup evaluations
- Must agree to stay in the Twin Cities area (\<45-minute drive from the Masonic Children's Hospital) for a minimum of 5 days after each cell infusion
- Voluntary written parental consent prior to the performance of any study related procedures
Exclusion
- Prior enzyme replacement therapy within 4 months prior to enrolling on study
- History of B cell related cancer, EBV lymphoproliferative disease or autoimmune disorders
- Evidence of active graft vs. host disease
- 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.
Key Trial Info
Start Date :
December 1 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 1 2023
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT04284254
Start Date
December 1 2022
End Date
June 1 2023
Last Update
October 7 2022
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