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

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

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

Study Overview

• Status: Completed
• Conditions: Hematological Malignancies
• Intervention / Treatment: Biological: omidubicel

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 consisting of mature myeloid and lymphoid cells as follows:

1. Ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF)), containing a minimum of 8.0 × 10^8 total viable cells of which a minimum of 8.7% is CD34+ cells and a minimum of 9.2 × 10^7 CD34+ cells, and
2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) (Non-cultured Fraction (NF)), containing a minimum of 4.0 × 10^8 total viable cells with a minimum of 2.4 × 10^7 CD3+ cells

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 Type: Interventional
• Enrollment (Actual): 36
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90095
      • UCLA
    • Palo Alto, California, United States, 94063
      • Stanford University Cancer Institute
  • Illinois
    • Maywood, Illinois, United States, 60153
      • Loyola University, Cardinal Bernardin Cancer Center
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota Masonic Cancer Center
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health & Science University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: omidubicel; Received omidubicel	Biological: omidubicel; hematopoietic stem cell transplant; Other Names: NiCord

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time From Transplant to Neutrophil Engraftment	Neutrophil engraftment was defined as achieving an absolute neutrophil count (ANC) greater than or equal to 0.5 x 10^9/L on 3 consecutive measurements by Day 42 post-transplant inclusive. The first day of the three measurements was designated the day of neutrophil engraftment.	by day 42 post-transplant inclusive
Cumulative Incidence of Neutrophil Engraftment	Death, second transplant, and relapse were competing risks at the time they occur if they occur prior to neutrophil engraftment, and no transplant was a competing risk at Day 0. If the patient failed to achieve neutrophil engraftment, they were considered to have a competing risk at Day 43.	by day 42 post-transplant inclusive

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cumulative Incidence of Platelet Engraftment >20,000 Cells/uL		By Day 42 and Day 180 post-transplant
Time to Platelet Engraftment >20,000 Cells/uL	Time to platelet engraftment >20,000 cells/ul was defined as the number of days from transplant to the first day of a minimum of 3 consecutive measurements on different days in which the platelet count is 20,000 cells/ul or higher with no platelet transfusion within the previous 7 days (count day of engraftment as one of the preceding 7 days) was calculated. The first day of the three measurements was designated the day of platelet engraftment.	By Day 730 post-transplant
Cumulative Incidence of Platelet Engraftment >50,000 Cells/uL		By Day 42 and Day 180 post-transplant
Time to Platelet Engraftment >50,000 Cells/uL	Time to platelet engraftment >50,000 cells/ul was defined as the number of days from transplant to the first day of a minimum of 3 consecutive measurements on different days in which the platelet count is 50,000 cells/ul or higher with no platelet transfusion within the previous 7 days (count day of engraftment as one of the preceding 7 days) was calculated. The first day of the three measurements was designated the day of platelet engraftment.	By Day 730 post-transplant
Non-relapse Mortality	Non-relapse mortality was defined as any death not preceded by relapse.	By Day 180, Day 365 and Day 730 post-transplant
Overall Survival (OS)	OS probability was defined as the probability of participants remaining alive at specified time points following transplantation, estimated using Kaplan-Meier methods.	By Day 180, Day 365 and Day 730 post-transplant
Disease Free Survival (DFS)	Disease-free survival was defined as the survival without disease relapse or death from any cause, whichever came first.	By Day 365 and Day 730 post-transplant
Donor Chimerism	Patients considered to have donor chimerism when they had at least 95% donor chimerism	By day 100 and Day 730 post-transplant
Secondary Graft Failure (SGF)		By Day 730 post-transplant
Disease Relapse		By Day 365 and Day 730 post-transplant
Cumulative Incidence of Acute GvHD Grade II-IV	Death, failure to achieve neutrophil engraftment, secondary graft failure, and relapse were considered competing events.	By Day 100 post-transplant
Cumulative Incidence of aGvHD Grade III-IV	Death, failure to achieve neutrophil engraftment, secondary graft failure, and relapse were considered competing events.	By Day 100 post-transplant
Cumulative Incidence of Chronic GvHD	Death, failure to achieve neutrophil engraftment, secondary graft failure, and relapse were considered competing events.	By Day 180 and Day 730 post-transplant
Chronic GvHD-free Relapse-free Survival (cGRFS)	Chronic graft versus host disease-free, relapse-free survival (cGRFS) was defined as chronic GvHD, relapse, or death by any cause.	By Day 365 and Day 730 post-transplant
GvHD-free Relapse-free Survival (GRFS)	Graft versus host disease-free, relapse-free survival (GRFS) was defined as acute GvHD Grade III-IV, chronic GvHD, relapse, or death by any cause	By Day 365 and Day 730 post-transplant

Collaborators and Investigators

• Sponsor: Gamida Cell ltd
• Investigators: Principal Investigator: Mitchell Horwitz, MD, Duke University

Study record dates

Study Major Dates

• Study Start (Actual): July 8, 2020
• Primary Completion (Actual): May 8, 2025
• Study Completion (Actual): May 8, 2025

Study Registration Dates

• First Submitted: February 5, 2020
• First Submitted That Met QC Criteria: February 5, 2020
• First Posted (Actual): February 7, 2020

Study Record Updates

• Last Update Posted (Actual): June 22, 2026
• Last Update Submitted That Met QC Criteria: May 24, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Hematologic Neoplasms
• Other Study ID Numbers: GC P#07.01.020

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No