MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT

• Status: Not yet recruiting
• Trial end date: 2023-06-01
• Target enrollment: 0
• Participant gender: All

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 plasmablasts engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty transposon system.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Masonic Cancer Center, University of Minnesota

Criteria

Inclusion Criteria:

• 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 Criteria:

• 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.