Overview
Omidubicel is an investigational therapy for patients with high-risk hematologic malignancies.
Description
Successful blood and marrow transplantation (BMT) requires the infusion of a sufficient number of hematopoietic stem/progenitor cells (HSPCs), capable of both homing to the bone marrow and regenerating a full array of hematopoietic cell lineages with early and late repopulating ability in a timely fashion.
Omidubicel is a stem/progenitor cell-based product composed of ex vivo expanded allogeneic cells from one entire unit of umbilical cord blood. Omidubicel utilizes the small molecule nicotinamide (NAM), as an epigenetic approach to inhibit differentiation and to increase the migration, bone marrow (BM) homing and engraftment efficiency of hematopoietic progenitor cells (HPC) expanded in ex vivo cultures.
The overall study objectives are to provide access to omidubicel for transplantation in patients with hematological malignancies and to collect additional safety and efficacy data.
Eligibility
Inclusion Criteria:
- Patients must be at least 12 years of age
- Applicable disease criteria
- Patients must have one or two partially HLA-matched CBUs
- Back-up stem cell source
- Sufficient physiological reserves
- Females of childbearing potential agree to use appropriate method of contraception
- Signed written informed consent
Exclusion Criteria:
- Extensive bone marrow fibrosis
- Donor specific anti-HLA antibodies
- Pregnancy
- Medically unsuitable for transplant