BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

NCT03639844 · Status: NO_LONGER_AVAILABLE · Type: EXPANDED_ACCESS

Last updated 2020-10-05

No results posted yet for this study

Summary

Providing access of BPX-501 gene modified T cells and rimiducid to pediatric patients who do not meet the eligibility criteria of the BP-U-004 study.

Conditions

  • Hurler Syndrome
  • Inherited Metabolic Disorder
  • Lysosomal Storage Disorder
  • Metachromatic Leukodystrophy
  • Inborn Errors of Metabolism

Interventions

BIOLOGICAL

rivogenlecleucel

BPX-501 T cells are genetically modified with a suicide safety switch. The cells are infused after T cell-depleted HSCT to potentially enhance immune reconstitution while reducing severity and duration of GVHD.

DRUG

rimiducid

Rimiducid induces activation of the Caspase 9 suicide gene in BPX-501 T cells inducing apoptosis of the modified T cells in case of GVHD

Sponsors & Collaborators

  • Bellicum Pharmaceuticals

    lead INDUSTRY

Principal Investigators

  • Bellicum Pharmaceuticals · Bellicum Pharmaceuticals, Inc.

Eligibility

Min Age
3 Months
Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Countries

  • United States

Study Locations

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Entities

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 NCT03639844 on ClinicalTrials.gov