Expanded Access of Omidubicel, for Allogeneic Transplantation in Patients With Hematological Malignancies

Gamida Cell

Status and phase: Completed; Phase 3

Conditions: Hematological Malignancies

Treatments: Biological: omidubicel

Study type: Interventional

Funder types: Industry

Identifiers

NCT04260698

GC P#07.01.020

Details and patient eligibility

About

Omidubicel is an investigational therapy for patients with high-risk hematologic malignancies.

Full 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.

Enrollment: 36 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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36 participants in 1 patient group

omidubicel

Experimental group

Description:

Omidubicel is a cryopreserved stem/progenitor cell based product comprised of: 1. Ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF)) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) (Non-cultured Fraction (NF)) consisting of mature myeloid and lymphoid cells. Both fractions, i.e. CF and NF, will be kept frozen until they are thawed and infused on the day of transplantation.

Treatment:

Biological: omidubicel