Hematological MalignanciesPhase 3NCT04260698
Expanded Access of Omidubicel, for Allogeneic Transplantation in Patients With Hematological Malignancies
A Phase 3 study testing omidubicel in people with Hematological Malignancies, run by Gamida Cell ltd at 6 sites in United States.
Trial summary
Omidubicel is an investigational therapy for patients with high-risk hematologic malignancies. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hematological Malignancies
- What is being tested?
- omidubicel
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 6 sitesUnited States
- Study dates
- 8 Jul 2020to 8 May 2025
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Extensive bone marrow fibrosis
- Donor specific anti-HLA antibodies
- Pregnancy
- Medically unsuitable for transplant
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Duke University Medical Center
Durham, North Carolina, 27710
UCLA
Los Angeles, California, 90095
Loyola University, Cardinal Bernardin Cancer Center
Maywood, Illinois, 60153
University of Minnesota Masonic Cancer Center
Minneapolis, Minnesota, 55455
Stanford University Cancer Institute
Palo Alto, California, 94063
Oregon Health & Science University
Portland, Oregon, 97239
About this study
Official title: 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.
Read the 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:
- 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.
- Conditions
- Hematological Malignancies
- Being tested
- omidubicel
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
omidubicelBiological
Also called NiCord
hematopoietic stem cell transplant
| Study group | Role | What they receive |
|---|---|---|
| omidubicel | Experimental | Received omidubicel Receives: Biological: omidubicel |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: by day 42 post-transplant inclusive
Secondary outcomes (15)
Cumulative Incidence of Platelet Engraftment >20,000 Cells/uL
Measured: 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.
Measured: By Day 730 post-transplant
Cumulative Incidence of Platelet Engraftment >50,000 Cells/uL
Measured: 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.
Measured: By Day 730 post-transplant
Non-relapse Mortality
Non-relapse mortality was defined as any death not preceded by relapse.
Measured: 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.
Measured: 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.
Measured: By Day 365 and Day 730 post-transplant
Donor Chimerism
Patients considered to have donor chimerism when they had at least 95% donor chimerism
Measured: By day 100 and Day 730 post-transplant
Secondary Graft Failure (SGF)
Measured: By Day 730 post-transplant
Disease Relapse
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: By Day 365 and Day 730 post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Feb 2020
- Study start8 Jul 2020
- Primary completion8 May 2025
- Study completion8 May 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hematological MalignanciesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gamida Cell ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mitchell Horwitz, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04260698
- Sponsor's protocol ID
- GC P#07.01.020
- Status last verified
- May 2026
- First submitted
- 5 Feb 2020
- Last updated
- 22 Jun 2026
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Expanded Access Study of Omidubicel, for Allogeneic Transplantation in Patients With Hematological Malignancies
- ClinicalTrials.gov ID
- NCT04260698
- Acronym / sponsor ID
- GC P#07.01.020
- Conditions
- Hematological Malignancies
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Gamida Cell ltdIndustry (pharma, biotech, devices)
- Study start
- 8 Jul 2020 (actual)
- Primary completion
- 8 May 2025 (actual)
- Study completion
- 8 May 2025 (actual)
- First posted
- 7 Feb 2020 (actual)
- Results
- Posted 22 Jun 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Omidubicel is an investigational therapy for patients with high-risk hematologic malignancies.
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:
- 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| omidubicel | Experimental | Biological: omidubicelReceived omidubicel |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (4 listed)
- Extensive bone marrow fibrosis
- Donor specific anti-HLA antibodies
- Pregnancy
- Medically unsuitable for transplant
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| omidubicelAlso: NiCordhematopoietic stem cell transplant | Biological | omidubicel |
Primary outcome
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.
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.
15 secondary outcomes
Cumulative Incidence of Platelet Engraftment >20,000 Cells/uL
Time frame: By Day 42 and Day 180 post-transplant
Time to Platelet Engraftment >20,000 Cells/uL
Time frame: By Day 730 post-transplant
Cumulative Incidence of Platelet Engraftment >50,000 Cells/uL
Time frame: By Day 42 and Day 180 post-transplant
Time to Platelet Engraftment >50,000 Cells/uL
Time frame: By Day 730 post-transplant
Non-relapse Mortality
Time frame: By Day 180, Day 365 and Day 730 post-transplant
Overall Survival (OS)
Time frame: By Day 180, Day 365 and Day 730 post-transplant
Disease Free Survival (DFS)
Time frame: By Day 365 and Day 730 post-transplant
Donor Chimerism
Time frame: By day 100 and Day 730 post-transplant
Secondary Graft Failure (SGF)
Time frame: By Day 730 post-transplant
Disease Relapse
Time frame: By Day 365 and Day 730 post-transplant
Cumulative Incidence of Acute GvHD Grade II-IV
Time frame: By Day 100 post-transplant
Cumulative Incidence of aGvHD Grade III-IV
Time frame: By Day 100 post-transplant
Cumulative Incidence of Chronic GvHD
Time frame: By Day 180 and Day 730 post-transplant
Chronic GvHD-free Relapse-free Survival (cGRFS)
Time frame: By Day 365 and Day 730 post-transplant
GvHD-free Relapse-free Survival (GRFS)
Time frame: By Day 365 and Day 730 post-transplant
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jun 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mitchell Horwitz, MD principal investigator
Duke University
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing omidubicel in people with Hematological Malignancies, run by Gamida Cell ltd at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 22 Jun 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time From Transplant to Neutrophil Engraftment (time frame: by day 42 post-transplant inclusive). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2026.