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Completed

NCT Number: NCT04260698

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

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

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Key information

Conditions

Hematological Malignancies Hematologic Diseases Hematologic Neoplasms Hemic and Lymphatic Diseases Neoplasms Neoplasms by Site

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

UCLA, Los Angeles, California, United States

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About this study

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:

  • 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
  • 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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

omidubicel

Biological

hematopoietic stem cell transplant

Other names: NiCord

Primary outcomes

  1. Time From Transplant to Neutrophil Engraftment

    Time frame: by day 42 post-transplant inclusive

    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.

  2. Cumulative Incidence of Neutrophil Engraftment

    Time frame: by day 42 post-transplant inclusive

    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.

Secondary outcomes

  1. Cumulative Incidence of Platelet Engraftment >20,000 Cells/uL

    Time frame: By Day 42 and Day 180 post-transplant

  2. Time to Platelet Engraftment >20,000 Cells/uL

    Time frame: By Day 730 post-transplant

    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.

  3. Cumulative Incidence of Platelet Engraftment >50,000 Cells/uL

    Time frame: By Day 42 and Day 180 post-transplant

  4. Time to Platelet Engraftment >50,000 Cells/uL

    Time frame: By Day 730 post-transplant

    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.

  5. Non-relapse Mortality

    Time frame: By Day 180, Day 365 and Day 730 post-transplant

    Non-relapse mortality was defined as any death not preceded by relapse.

  6. Overall Survival (OS)

    Time frame: By Day 180, Day 365 and Day 730 post-transplant

    OS probability was defined as the probability of participants remaining alive at specified time points following transplantation, estimated using Kaplan-Meier methods.

  7. Disease Free Survival (DFS)

    Time frame: By Day 365 and Day 730 post-transplant

    Disease-free survival was defined as the survival without disease relapse or death from any cause, whichever came first.

  8. Donor Chimerism

    Time frame: By day 100 and Day 730 post-transplant

    Patients considered to have donor chimerism when they had at least 95% donor chimerism

  9. Secondary Graft Failure (SGF)

    Time frame: By Day 730 post-transplant

  10. Disease Relapse

    Time frame: By Day 365 and Day 730 post-transplant

  11. Cumulative Incidence of Acute GvHD Grade II-IV

    Time frame: By Day 100 post-transplant

    Death, failure to achieve neutrophil engraftment, secondary graft failure, and relapse were considered competing events.

  12. Cumulative Incidence of aGvHD Grade III-IV

    Time frame: By Day 100 post-transplant

    Death, failure to achieve neutrophil engraftment, secondary graft failure, and relapse were considered competing events.

  13. Cumulative Incidence of Chronic GvHD

    Time frame: By Day 180 and Day 730 post-transplant

    Death, failure to achieve neutrophil engraftment, secondary graft failure, and relapse were considered competing events.

  14. Chronic GvHD-free Relapse-free Survival (cGRFS)

    Time frame: By Day 365 and Day 730 post-transplant

    Chronic graft versus host disease-free, relapse-free survival (cGRFS) was defined as chronic GvHD, relapse, or death by any cause.

  15. GvHD-free Relapse-free Survival (GRFS)

    Time frame: By Day 365 and Day 730 post-transplant

    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

Sponsors and collaborators

Lead sponsor

Gamida Cell ltd

Industry

Registry information

Official study title

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

Important dates

Study start
2020
Primary completion
2025
Study completion
2025
First posted
Feb 7, 2020
Registry last updated
Jun 22, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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