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

Launched by BELLICUM PHARMACEUTICALS · Aug 17, 2018

Keywords

ClinConnect Summary

This is an expanded access protocol of BPX-501 T cells infused after T cell-depleted HSCT in pediatric patients with non-malignant hematologic disorders eligible for treatment on the BP-U-004 study.

The purpose of this protocol is to provide access to the CaspaCIDe system combination product (BPX-501 gene modified T cells and rimiducid) to patients on a case by case basis who do not meet the BP-U-004 protocol eligibility criteria. BPX-501 infusion can enhance immune reconstitution with the potential for reducing the severity and duration of severe acute GVHD.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Males or females
• • 2. Age \< 21 years and \> 3 months
• • 3. Life expectancy \> 10 weeks
• • 4. Patients deemed eligible for allogeneic stem cell transplantation.
• 5. Non-malignant disorders including:
• • 1. inherited metabolic disorders such as adrenal leukodystrophy;
• • 2. lysosomal storage disorders such as Hurler syndrome or metachromatic leukodystrophy
• • 3. other inborn errors of metabolism
• • 6. Lack of suitable conventional donor (HLA identical sibling or HLA phenotypically identical relative evaluated using high resolution molecular typing).
• • 7. A minimum genotypic identical match of 5/10 is required.
• • 8. The donor and recipient must be identical, as determined by high resolution typing, at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, and HLA- DRB1.
• • 9. Lansky/Karnofsky score \> 50
• • 10. Signed written informed consent
• • 3.2 Subject exclusion criteria
• • 1. Age \< 3 months or \>21 years
• 2. Patients with non-malignant disorders eligible for treatment on the BP-U-004 study:
• • 1. primary immune deficiencies,
• • 2. severe aplastic anemia not responding to immune suppressive therapy,
• • 3. osteopetrosis,
• • 4. selected cases of hemoglobinopathies and
• • 5. congenital/hereditary cytopenia, including Fanconi Anemia before any clonal malignant evolution (MDS, AML)
• • 3. Greater than Grade II acute GVHD or chronic extensive GVHD due to a previous allograft at the time of inclusion
• • 4. Patient receiving an immunosuppressive treatment for GVHD treatment due to a previous allograft at the time of inclusion
• • 5. Dysfunction of liver (ALT/AST \> 5 times normal value, or bilirubin \> 3 times normal value), or of renal function (creatinine clearance \< 30 ml / min)
• • 6. Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction \< 40%)
• • 7. Current active infectious disease (including positive HIV serology or viral RNA)
• • 8. Serious concurrent uncontrolled medical disorder
• • 9. Pregnant or breast feeding female patient
• • 10. Lack of parents'/guardian's informed consent.
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