Trials / Withdrawn
WithdrawnNCT04284254
MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT
Sleeping Beauty Transposon-Engineered Plasmablasts for Expression and Delivery of Alpha-L-iduronidase in Patients With Hurler Syndrome That Have Previously Undergone Allogeneic Transplantation
- Status
- Withdrawn
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 0 (actual)
- Sponsor
- Masonic Cancer Center, University of Minnesota · Academic / Other
- Sex
- All
- Age
- 3 Years – 8 Years
- Healthy volunteers
- Not accepted
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.
Conditions
- Mucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome)
- Mucopolysaccharidosis Type IH
- MPS IH, Hurler Syndrome
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Autologous Plasmablasts | Autologous Plasmablasts engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty transposon system. Phase 1: * Dose Level 1: 5 x 10e7 cells/kg on Day 0 * Dose Level 2: 1 x 10e8 cells/kg on Day 0 * Dose Level 3: 1 x 10e8 cells/kg x 2 doses on Day 0 and Day 30 +/-3 days. Phase 2: \- Maximum Tolerated Dose (MTD) established in Phase I |
Timeline
- Start date
- 2022-12-01
- Primary completion
- 2023-06-01
- Completion
- 2023-06-01
- First posted
- 2020-02-25
- Last updated
- 2022-10-07
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04284254. Inclusion in this directory is not an endorsement.