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

Recruitment Status
COMPLETED
(See Contacts and Locations)Verified May 2025 by Gamida Cell ltd
Sponsor
Gamida Cell ltd
Information Provided by (Responsible Party)
Gamida Cell ltd
Clinicaltrials.gov Identifier
NCT04260698
Other Study ID Numbers:
GC P#07.01.020
First Submitted
February 4, 2020
First Posted
February 6, 2020
Last Update Posted
July 1, 2025
Last Verified
May 2025

ClinicalTrials.gov processed this data on June 2025. Link to the current ClinicalTrials.gov record .

History of Changes

Study Details

Study 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.

Condition or DiseaseIntervention/Treatment
Hematological Malignancies
Biological: omidubicel

Study Design

Study TypeInterventional
Actual Enrollment36 participants
Design AllocationN/A
Interventional ModelSingle Group Assignment
MaskingNone (Open Label)
Primary PurposeTreatment
Official TitleAn Open Label Expanded Access Study of Omidubicel, for Allogeneic Transplantation in Patients With Hematological Malignancies
Study Start DateJuly 7, 2020
Actual Primary Completion DateMay 7, 2025
Actual Study Completion DateMay 7, 2025

Groups and Cohorts

Group/CohortIntervention/Treatment
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

Outcome Measures

Primary Outcome Measures
  1. To assess the time from transplant to neutrophil engraftment

Eligibility Criteria

Ages Eligible for Study(Child, Adult, Older Adult)
Sexes Eligible for StudyAll
Accepts Healthy VolunteersNo
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

Contacts and Locations

Sponsors and CollaboratorsGamida Cell ltd
Locations
UCLA | Los Angeles California, United States, 90095Stanford University Cancer Institute | Palo Alto California, United States, 94063Loyola University, Cardinal Bernardin Cancer Center | Maywood Illinois, United States, 60153University of Minnesota Masonic Cancer Center | Minneapolis Minnesota, United States, 55455Duke University Medical Center | Durham North Carolina, United States, 27710Oregon Health & Science University | Portland Oregon, United States, 97239
Investigators
Principal Investigator: Mitchell Horwitz, MD, Duke University