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

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ClinicalTrials.gov Identifier: NCT03639844
Expanded Access Status : No longer available
First Posted : August 21, 2018
Last Update Posted : October 5, 2020
Information provided by (Responsible Party):
Bellicum Pharmaceuticals

Brief 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.

Condition or disease Intervention/treatment
Hurler Syndrome Inherited Metabolic Disorder Lysosomal Storage Disorder Metachromatic Leukodystrophy Inborn Errors of Metabolism Biological: rivogenlecleucel Drug: rimiducid

Detailed Description:

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.

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Study Type : Expanded Access
Expanded Access Type : Individual Patients
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Expanded Access Protocol for CaspaCIDe T Cells From An HLA-Partially Matched Related Donor After Negative Selection of TCR αβ+T Cells In Pediatric Patients Affected by Hematological and Other Disorders

Intervention Details:
  • 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.
    Other Name: BPX-501 T cells
  • 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
    Other Names:
    • AP1903
    • Rimiducid for Injection
    • AP1903 for Injection

Information from the National Library of Medicine

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Ages Eligible for Study:   3 Months to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All

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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03639844

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United States, California
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Stanford University; Division of Pediatric Stem Cell Transplant & Regenerative Medicine
Palo Alto, California, United States, 94304
Sponsors and Collaborators
Bellicum Pharmaceuticals
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Study Director: Bellicum Pharmaceuticals Bellicum Pharmaceuticals, Inc.
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Responsible Party: Bellicum Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03639844    
Other Study ID Numbers: BP-C-004
First Posted: August 21, 2018    Key Record Dates
Last Update Posted: October 5, 2020
Last Verified: October 2020
Additional relevant MeSH terms:
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Leukodystrophy, Metachromatic
Metabolism, Inborn Errors
Mucopolysaccharidosis I
Lysosomal Storage Diseases
Metabolic Diseases
Pathologic Processes
Genetic Diseases, Inborn
Hereditary Central Nervous System Demyelinating Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Lysosomal Storage Diseases, Nervous System
Demyelinating Diseases
Lipid Metabolism, Inborn Errors
Lipid Metabolism Disorders
Carbohydrate Metabolism, Inborn Errors
Connective Tissue Diseases