MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT (NCT04284254) | Clinical Trial Compass
WithdrawnPhase 1/2
MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT
Stopped: Study went on hold because more pre-clinical work needs to be done per FDA feedback. However, the study never went off hold and the study team has decided to close this study without opening to enrollment.
0Started 2022-12
Plain-language 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.
Who can participate
Age range
3 Years – 8 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Maximum Tolerated Dose (MTD)
Timeframe: 1 Year
2
Growth Velocity (cm/year)
Timeframe: 1 Year
3
Safety and Tolerability after Infusion: Incidence of Adverse Events
Timeframe: 1 Year
Trial details
NCT IDNCT04284254
SponsorMasonic Cancer Center, University of Minnesota