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

NCT04260698 | Completed Phase 3 Results FDA Drug

• 1 other identifier: GC P#07.01.020
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 6

Brief Summary

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

Conditions

Hematological Malignancies

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (15)

• 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

  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

  By Day 730 post-transplant

• Non-relapse Mortality

  By Day 180, Day 365 and Day 730 post-transplant

Study Arms (1)

omidubicel

EXPERIMENTAL

Received omidubicel

Biological: omidubicel

Interventions

omidubicel BIOLOGICAL

hematopoietic stem cell transplant

Also known as: NiCord

omidubicel

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• Extensive bone marrow fibrosis
• Donor specific anti-HLA antibodies
• Pregnancy
• Medically unsuitable for transplant

Sponsors & Collaborators

• Gamida Cell ltd: lead

Study Sites (6)

UCLA

Los Angeles, California, 90095, United States

Location

Stanford University Cancer Institute

Palo Alto, California, 94063, United States

Location

Loyola University, Cardinal Bernardin Cancer Center

Maywood, Illinois, 60153, United States

Location

University of Minnesota Masonic Cancer Center

Minneapolis, Minnesota, 55455, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

MeSH Terms

Conditions

Hematologic Neoplasms

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Results Point of Contact

• Title: Ronit Simantov, MD
• Organization: Gamida Cell

ronit@gamida-cell.com

+16465310676

Study Officials

• Mitchell Horwitz, MD

  Duke University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 5, 2020
• First Posted: February 7, 2020
• Study Start: July 8, 2020
• Primary Completion: May 8, 2025
• Study Completion: May 8, 2025
• Last Updated: June 22, 2026
• Results First Posted: June 22, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)