Expanded Access Protocol Using Alpha/Beta T and CD19+ Depleted PBSC
Leukemia, Bone Marrow Failure Syndrome, Immunodeficiencies
About this trial
This is an expanded access trial for Leukemia
Eligibility Criteria
PATIENT AND DONOR ELIGIBILITY
Patients who lack an HLA matched sibling and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT) but do not meet criteria for current open institutional protocols using ClinMACs device for β T/CD19+ depletion.
Patients with the following transplantable diseases:
Non-malignant diseases:
- Metabolic storage diseases correctable by HSCT
- Bone marrow failure syndromes
- Immunodeficiencies/immune dysregulation syndromes
- Sickle cell disease or thalassemia
- Other diseases treated with HSCT
Malignant diseases:
- Acute leukemias
- Chronic leukemias
- Lymphomas
- Myelodyplastic syndrome
Organ function criteria:
It is important to note that the conditioning prescribed to the patient will be determined based on the disease and organ status and will be regimens considered standard. Appropriate combinations of chemotherapy, immunotherapy and/or radiation will be determined on an individual basis.
Patient eligibility will be assessed as per our institutional standard operating procedures:
- Lansky or Karnofsky performance >60
- Renal function: will be determined based on serum creatinine as per our Institutional SOP
- Hepatic: Transaminases will be assessed as per current institutional SOP
- Cardiac: Cardiac function will be assessed as per institutional SOP
- Bilirubin <2.5x normal (unless elevation due to Gilberts disease) as per Institutional SOP
- No active untreated infection
- Signed informed consent
- No fully HLA matched sibling donor available.
- Females of childbearing potential must have negative pregnancy test.
Donor Eligibility Patients must have an identified living donor
- Donor selection will comply with 21 Code of Federal Regulations (CFR) 1271*
- Unrelated donor that meets the matching criteria of the NMDP: Unrelated donors that may be up to a one antigen mismatch at A, B or DRB1. donor
- Related donor mismatched at one to five antigens (haploidentical)
- Donor suitable for mobilization of peripheral stem cells and apheresis and fulfills infectious disease criteria as per our institutional SOP, including HIV, Hepatitis B (HepB), Hepatitis C (HepC) polymerase chain reaction (PCR) negative.
- CHOP bone marrow transplant (BMT) procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases. Our donor collection program is Foundation for the Accreditation of Cellular Therapy (FACT) accredited.
- Unrelated donor identified through the National Marrow Donor Program (NMDP) and fulfills the NMDP criteria for donation. Unrelated donor willing and able to undergo mobilization of peripheral stem cells and apheresis
- The donors selected for this investigational new drug (IND) will either be unrelated donors identified through the National Marrow Donor Program (NMDP) or related donors. Regarding the unrelated donors; NMDP procedures for determining donor eligibility include donor screening and testing for relevant communicable disease agents and diseases.
Exclusion criteria:
- Uncontrolled bacterial, viral or fungal infections
- Fully HLA matched sibling donor
- Donor unable to donate peripheral stem cells
- Pregnant Females
Sites / Locations
- Children's Hospital of Philadelphia