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

Sponsor

Bellicum Pharmaceuticals (Industry)

Overall Status

No longer available

CT.gov ID

NCT03639844

Collaborator

(none)

Locations

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
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.

Study Design

Study Type:

Expanded Access

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

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

3 Months to 21 Years

Sexes Eligible for Study:

All

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Hospital Los Angeles	Los Angeles	California	United States	90027
2	Stanford University; Division of Pediatric Stem Cell Transplant & Regenerative Medicine	Palo Alto	California	United States	94304