MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT
study id #: NCT04284254
condition: Mucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome), Mucopolysaccharidosis Type IH, MPS IH, Hurler Syndrome
status: Not yet recruitingpurpose:
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.
intervention: Autologous Plasmablasts
last updated: February 26, 2022
start date: December 2021
estimated completion: June 2023
last updated: July 8, 2021
phase of development: Phase 1/Phase 2
size / enrollment: 36
- Maximum Tolerated Dose (MTD)
Maximum tolerated dose (MTD) of autologous plasmablasts engineered to express large amounts of α-L-iduronidase (IDUA) using a Sleeping Beauty transposon approach
- 1 Year
Growth Velocity (cm/year)
Growth velocity in centimeters/year over a one-year period through determinations of sitting and standing height at baseline and post infusion
- 1 Year
Safety and Tolerability after Infusion: Incidence of Adverse Events
Incidence of Adverse Events
- 1 Year
- Z-score Growth Rate
- Donor Engraftment
Baseline, 6 months and 1 Year
- Levels of circulating antibodies (IgG, IgM, IgA and IgE)
• Eligible Sexes: all
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: 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.
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