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

Study Overview
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Brief Summary
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Omidubicel is an investigational therapy for patients with high-risk hematologic malignancies.

Detailed Description
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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.

Official Title
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An Open Label Expanded Access Study of Omidubicel, for Allogeneic Transplantation in Patients With Hematological Malignancies

Conditions
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Hematological Malignancies

Intervention / Treatment
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* Biological: omidubicel


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: omidubicel<br>Omidubicel is a cryopreserved stem/progenitor cell based product comprised of: Ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF)) 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. | Biological: omidubicel<br>* hematopoietic stem cell transplant<br>* Other names: NiCord;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To assess the time from transplant to neutrophil engraftment |  | by day 42 post-transplant inclusive |