NO_LONGER_AVAILABLENot Applicable

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

United States

Plain-language 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.

Age range3 Months – 21 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Males or females
✓. Age \< 21 years and \> 3 months
✓. Life expectancy \> 10 weeks
✓. Patients deemed eligible for allogeneic stem cell transplantation.
✓. Non-malignant disorders including:
✓. inherited metabolic disorders such as adrenal leukodystrophy;
✓. lysosomal storage disorders such as Hurler syndrome or metachromatic leukodystrophy
✓. other inborn errors of metabolism

✕. Age \< 3 months or \>21 years
✕. Patients with non-malignant disorders eligible for treatment on the BP-U-004 study:
✕. primary immune deficiencies,
✕. severe aplastic anemia not responding to immune suppressive therapy,
✕. osteopetrosis,
✕. selected cases of hemoglobinopathies and
✕. congenital/hereditary cytopenia, including Fanconi Anemia before any clonal malignant evolution (MDS, AML)
✕. Greater than Grade II acute GVHD or chronic extensive GVHD due to a previous allograft at the time of inclusion

NCT IDNCT03639844

SponsorBellicum Pharmaceuticals

Sponsor typeINDUSTRY

Study typeEXPANDED_ACCESS