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

University of Minnesota (UMN)

Status and phase

Withdrawn

Phase 2

Phase 1

Conditions

Mucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome)

MPS IH, Hurler Syndrome

Mucopolysaccharidosis Type IH

Treatments

Drug: Autologous Plasmablasts

Study type

Interventional

Funder types

Other

Identifiers

NCT04284254

MT2018-18 (Other Identifier)

2018LS094

Details and patient eligibility

About

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.

Sex

All

Ages

3 to 8 years old

Volunteers

No Healthy Volunteers

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.