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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04260698
Recruitment Status : Not yet recruiting
First Posted : February 7, 2020
Last Update Posted : February 12, 2020
Information provided by (Responsible Party):
Gamida Cell ltd

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

Condition or disease Intervention/treatment Phase
Hematological Malignancies Biological: omidubicel 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label Expanded Access Study of Omidubicel, for Allogeneic Transplantation in Patients With Hematological Malignancies
Estimated Study Start Date : June 15, 2020
Estimated Primary Completion Date : December 31, 2022
Estimated Study Completion Date : December 31, 2024

Arm Intervention/treatment
Experimental: omidubicel

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.

Biological: omidubicel
hematopoietic stem cell transplant
Other Name: NiCord

Primary Outcome Measures :
  1. To assess the time from transplant to neutrophil engraftment [ Time Frame: by day 42 post-transplant inclusive ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04260698

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Contact: Kelly Myers +97226595631

Sponsors and Collaborators
Gamida Cell ltd
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Principal Investigator: Mitchell Horwitz, MD Duke University
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Responsible Party: Gamida Cell ltd Identifier: NCT04260698    
Other Study ID Numbers: GC P#07.01.020
First Posted: February 7, 2020    Key Record Dates
Last Update Posted: February 12, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hematologic Neoplasms
Neoplasms by Site
Hematologic Diseases