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

NO_LONGER_AVAILABLE

Conditions studied: Hurler Syndrome, Inherited Metabolic Disorder, Lysosomal Storage Disorder, Metachromatic Leukodystrophy, Inborn Errors of Metabolism

In brief

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.

Key facts

Study ID

NCT03639844

Run by

Bellicum Pharmaceuticals

Last updated by the study team

2020-10-05

Who can join

Age: 0 and older, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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
• 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%)

Where it is running

• Children's Hospital Los Angeles — Los Angeles, California, United States
• Stanford University; Division of Pediatric Stem Cell Transplant & Regenerative Medicine — Palo Alto, California, United States

Full record on ClinicalTrials.gov

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