Back to resultsBPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study
Primary Purpose
Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder
Status
No longer available
Phase
Locations
United States
Study Type
Expanded Access
Intervention
rivogenlecleucel
rimiducid
Sponsored by
About this trial
This is an expanded access trial for Hurler Syndrome
Eligibility Criteria
Inclusion Criteria:
- 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
- Lack of suitable conventional donor (HLA identical sibling or HLA phenotypically identical relative evaluated using high resolution molecular typing).
- A minimum genotypic identical match of 5/10 is required.
- 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.
- Lansky/Karnofsky score > 50
- Signed written informed consent
3.2 Subject exclusion criteria
- 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
- Patient receiving an immunosuppressive treatment for GVHD treatment due to a previous allograft at the time of inclusion
- Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance < 30 ml / min)
- Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction < 40%)
- Current active infectious disease (including positive HIV serology or viral RNA)
- Serious concurrent uncontrolled medical disorder
- Pregnant or breast feeding female patient
Lack of parents'/guardian's informed consent.
-
Sites / Locations
- Children's Hospital Los Angeles
- Stanford University; Division of Pediatric Stem Cell Transplant & Regenerative Medicine
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT03639844
First Posted
August 17, 2018
Last Updated
October 1, 2020
Sponsor
Bellicum Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT03639844
Brief Title
BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study
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
Study Type
Expanded Access
2. Study Status
Record Verification Date
October 2020
Overall Recruitment Status
No longer available
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bellicum Pharmaceuticals
4. Oversight
5. Study Description
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy, Inborn Errors of Metabolism
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Biological
Intervention Name(s)
rivogenlecleucel
Other Intervention Name(s)
BPX-501 T cells
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
rimiducid
Other Intervention Name(s)
AP1903, Rimiducid for Injection, AP1903 for Injection
Intervention Description
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Months
Maximum Age & Unit of Time
21 Years
Eligibility Criteria
Inclusion Criteria:
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
Lack of suitable conventional donor (HLA identical sibling or HLA phenotypically identical relative evaluated using high resolution molecular typing).
A minimum genotypic identical match of 5/10 is required.
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.
Lansky/Karnofsky score > 50
Signed written informed consent
3.2 Subject exclusion criteria
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
Patient receiving an immunosuppressive treatment for GVHD treatment due to a previous allograft at the time of inclusion
Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal value), or of renal function (creatinine clearance < 30 ml / min)
Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction < 40%)
Current active infectious disease (including positive HIV serology or viral RNA)
Serious concurrent uncontrolled medical disorder
Pregnant or breast feeding female patient
Lack of parents'/guardian's informed consent.
-
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bellicum Pharmaceuticals
Organizational Affiliation
Bellicum Pharmaceuticals, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Stanford University; Division of Pediatric Stem Cell Transplant & Regenerative Medicine
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
12. IPD Sharing Statement
Learn more about this trial
BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study
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