Expanded Access of Omidubicel, for Allogeneic Transplantation in Patients With Hematological Malignancies
Study Details
Study Description
Brief Summary
Omidubicel is an investigational therapy for patients with high-risk hematologic malignancies.
Condition or Disease | Intervention/Treatment | Phase |
---|---|---|
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Phase 3 |
Detailed 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.
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
---|---|
Experimental: omidubicel 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
hematopoietic stem cell transplant
Other Names:
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Outcome Measures
Primary Outcome Measures
- To assess the time from transplant to neutrophil engraftment [by day 42 post-transplant inclusive]
Eligibility Criteria
Criteria
Inclusion Criteria:
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Patients must be at least 12 years of age
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Applicable disease criteria
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Patients must have one or two partially HLA-matched CBUs
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Back-up stem cell source
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Sufficient physiological reserves
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Females of childbearing potential agree to use appropriate method of contraception
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Signed written informed consent
Exclusion Criteria:
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Extensive bone marrow fibrosis
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Donor specific anti-HLA antibodies
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Pregnancy
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Medically unsuitable for transplant
Contacts and Locations
Locations
Site | City | State | Country | Postal Code | |
---|---|---|---|---|---|
1 | UCLA | Los Angeles | California | United States | 90095 |
2 | Stanford University Cancer Institute | Palo Alto | California | United States | 94063 |
3 | Loyola University, Cardinal Bernardin Cancer Center | Maywood | Illinois | United States | 60153 |
4 | University of Minnesota Masonic Cancer Center | Minneapolis | Minnesota | United States | 55455 |
5 | Duke University Medical Center | Durham | North Carolina | United States | 27710 |
6 | Oregon Health & Science University | Portland | Oregon | United States | 97239 |
Sponsors and Collaborators
- Gamida Cell ltd
Investigators
- Principal Investigator: Mitchell Horwitz, MD, Duke University
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- GC P#07.01.020