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

NCT04284254 · Status: WITHDRAWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL

Last updated 2022-10-07

No results posted yet for this study

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

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

Sponsors & Collaborators

  • Masonic Cancer Center, University of Minnesota

    lead OTHER

Principal Investigators

  • Paul Orchard, MD · University of Minnesota, Department of Pediatrics

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
3 Years
Max Age
8 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-12-31
Primary Completion
2023-06-30
Completion
2023-06-30
FDA Drug
Yes

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04284254 on ClinicalTrials.gov