Hematological MalignanciesPhase 3NCT02730299
Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in Patients With Leukemia, Lymphoma, and MDS
A Phase 3 study testing NiCord® (omidubicel) and Cord Blood Unit in people with Hematological Malignancies and Acute Lymphoblastic Leukemia (ALL), run by Gamida Cell ltd at 52 sites in 10 countries.
Trial summary
This study is an open-label, controlled, multicenter, international, Phase III, randomized study of transplantation of NiCord® versus transplantation of one or two unmanipulated, unrelated cord blood units in patients with acute lymphoblastic leukemia or acute myeloid leukemia, myelodysplastic syndrome, chronic… Read more about this study
- Who is it for?
- Ages 12 to 65 years
- Conditions the study is for
- Hematological Malignancies, Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML)
- What is being tested?
- NiCord® (omidubicel) and Cord Blood Unit
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 125 participants
- Where
- 52 sitesin 10 countries
- Study dates
- 16 Dec 2016to 7 Feb 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 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Applicable disease criteria
- Patients must have one or two partially HLA-matched CBUs
- Back-up stem cell source
- Adequate Karnofsky/Lansky Performance score
- Sufficient physiological reserves
- Signed written informed consent
Who cannot join
- HLA-matched donor able to donate
- Prior allogeneic HSCT
- Other active malignancy
- Active or uncontrolled infection
- Active/symptoms of central nervous system (CNS) disease
- Pregnancy or lactation
Trial locations and contacts
52 sites in 10 countries.
52 sites in 10 countries
United States21 sites
The University of Maryland Medicine Center
Baltimore, Maryland, 21201
Boston Children's Hospital
Boston, Massachusetts, 02115
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
University of Virginia Cancer Center
Charlottesville, Virginia, 22903
Cleveland Clinic Children's
Cleveland, Ohio, 44195
Children's Medical Center of Dallas
Dallas, Texas, 75235
Children's Hospital Colorado
Denver, Colorado, 80045
Henry Ford Medical Center
Detroit, Michigan
Duke University Medical Center
Durham, North Carolina, 27710
Northwestern University
Evanston, Illinois, 60208
West Cancer Clinic
Germantown, Tennessee, 38138
UCLA
Los Angeles, California, 90095
City of Hope
Los Angeles, California, 91010
Loyola University, Cardinal Bernardin Cancer Center
Maywood, Illinois, 60153
University of Minnesota Masonic Cancer Center
Minneapolis, Minnesota, 55455
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, 08901
Stanford University Cancer Institute
Palo Alto, California
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Oregon Health & Science University
Portland, Oregon, 97239
UC San Diego Moores Cancer Center
San Diego, California, 92093
University of Kansas Cancer Center
Westwood, Kansas, 66205
Spain8 sites
Hospital Universitari Vall d'Hebron pediatrics
Barcelona, 08035
University Hospital Vall d'Hebron
Barcelona, 08035
ICO Bellvitge
Barcelona, 08908
Sant Joan de Deu
Barcelona, 08950
Hospital de la Santa Creu i Sant Pau
Barcelona
Hospital Universitario Virgen del Rocío
Seville
Hospital Universitario La Fe
Valencia, 46009
Hospital Universitario y Politécnico La Fe (pediatric)
Valencia
Israel6 sites
Rambam
Haifa
Hadassah Medical Center
Jerusalem
Rabin Medical Center, Beilinson Hospital
Petah Tikva
Dana-Dwek Children's Hospital, Tel-Aviv Sourasky Medical Center
Tel Aviv
Tel-Aviv Sourasky Medical Center
Tel Aviv
Chaim Sheba Medical Center, The Edmond and Lily Safra Children's hospital
Tel Litwinsky
Brazil5 sites
Instituto Nacional de Câncer José Alencar Gomes da Silva - INCA
Rio de Janeiro, Rio de Janeiro, 20230-130
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo Pediatrics
São Paulo, São Paulo, 05403-010
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
São Paulo, São Paulo, 05403-010
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo
São Paulo, São Paulo, 14048-900
Hospital Israelita Albert Einstein
São Paulo
United Kingdom4 sites
Queen Elizabeth Hospital
Birmingham, B15 2GW
St James Hospital
Leeds, LS9 7TF
Manchester University NHS Foundation Trust
Manchester, M13 9WL
The Royal Marsden NHS Foundation Trust
Sutton, Surrey, SM2 5PT
Italy2 sites
Careggi University Hospital
Florence, 50134
Ospedale Pediatrico Bambino Gesù
Rome, 00165
Netherlands2 sites
University Medical Center Utrecht
Utrecht, 3503 AB
Prinses Maxima Centrum voor Kinderoncologie B.V.
Utrecht, 3584 CS
Singapore2 sites
National University Cancer Institute
Singapore, 119074
Singapore General Hospital
Singapore, 169608
Portugal1 site
Instituto Português de Oncologia de Lisboa Francisco Gentil
Lisbon, 1099-023
About this study
Official title: A Multicenter, Randomized, Phase III Registration Trial of Transplantation of NiCord®, Ex Vivo Expanded, UCB-derived, Stem and Progenitor Cells, vs. Unmanipulated UCB for Patients With Hematological Malignancies
This study is an open-label, controlled, multicenter, international, Phase III, randomized study of transplantation of NiCord® versus transplantation of one or two unmanipulated, unrelated cord blood units in patients with acute lymphoblastic leukemia or acute myeloid leukemia, myelodysplastic syndrome, chronic myeloid leukemia or lymphoma, all with required disease features rendering them eligible for allogeneic transplantation.
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.
A major drawback of Umbilical Cord Blood (UCB) is the low stem cell dose available for transplantation, compared to mobilized peripheral blood (PB) or bone marrow. This low stem cell dose can compromise the chances of engraftment and contributes to delayed kinetics of neutrophil and platelet recovery, as well as other transplant outcomes.
The aim of ex vivo expansion of cord blood is to provide a graft with sufficient numbers of cells that have rapid and robust in vivo neutrophil and platelet producing potential to enable successful transplantation.
NiCord® is a stem/progenitor cell-based product composed of ex vivo expanded allogeneic cells from one entire unit of UCB. NiCord® 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 chief aim of the study is to compare the safety and efficacy of NiCord® single ex-vivo expanded cord blood unit transplantation to unmanipulated cord blood unit transplantation in patients with hematological malignancies following conditioning therapy.
- Conditions
- Hematological Malignancies, Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML), Chronic Myelogenous Leukemia (CML), Myelodysplastic Syndrome (MDS), Lymphoma
- Being tested
- NiCord® (omidubicel), Cord Blood Unit
- Medical subject headings
- Hematologic Neoplasms, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Myelodysplastic Syndromes, Lymphoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NiCord® (omidubicel)Drug
Cord Blood UnitOther
Cord blood unit
| Study group | Role | What they receive |
|---|---|---|
| NiCord® (omidubicel) | Experimental | NiCord® is a cryopreserved stem/progenitor cell based product comprised of:
1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (NiCord® cultured fraction (CF))
2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) (NiCord® Non-cultured Fraction (NF)) consisting of mature myeloid and lymphoid cells.
Both fractions, i.e. NiCord® CF and NiCord® NF, will be kept frozen until they are thawed and infused on the day of transplantation. Receives: Drug: NiCord® (omidubicel) |
| Unmanipulated CBU(s) | Active comparator | Not described Receives: Other: Cord Blood Unit |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to Neutrophil Engraftment
The time to engraftment of neutrophils >500/μl was defined as per Center for International Blood and Marrow Transplant Research (CIBMTR) standards, requiring donor chimerism for neutrophil engraftment.
Measured: post-transplant up to 42 days
Secondary outcomes (3)
First Grade 2/3 Bacterial or Invasive Fungal Infections by 100 Days Following Transplantation
First Bacterial Infection Grades 2-3 or Invasive Fungal Infection by 100 Days following Transplantation for the Intent to Treat Population
Measured: 100 days post-transplant
Days Alive and Out of Hospital in the First 100 Days Post-transplantation
Days alive and out of hospital in the first 100 Days post-transplantation for the Intent to Treat Population
Measured: 100 days post-transplantation
Number of Participants With Platelet Engraftment by 42 Days Post-transplantation
Platelet engraftment defined as the first day of a minimum of three consecutive measurements on different days such that the patient has achieved a platelet count > 20 × 10^9/L with no platelet transfusions during the preceding seven days (counting day of engraftment as one of the preceding seven days) for the Intent to Treat Population
Measured: 42 days post-transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Apr 2016 (estimated)
- Study start16 Dec 2016
- Primary completion29 Apr 2021
- Study completion7 Feb 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.
- Publications (4)Papers the sponsor has linked to this study.
- 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 125 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gamida Cell ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mitchell Horwitz, MD, Study chair, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02730299
- Sponsor's protocol ID
- GC P#05.01.020
- Status last verified
- Feb 2026
- First submitted
- 28 Mar 2016
- Last updated
- 25 Feb 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Majhail NS, Miller B, Dean R, Manghani R, Shin H, Sivaraman S, Maziarz RT. Hospitalization and Healthcare Resource Utilization of Omidubicel-Onlv versus Umbilical Cord Blood Transplantation for Hematologic Malignancies: Secondary Analysis from a Pivotal Phase 3 Clinical Trial. Transplant Cell Ther. 2023 Dec;29(12):749.e1-749.e5. doi: 10.1016/j.jtct.2023.09.004. Epub 2023 Sep 12.
PubMed 37703995Szabolcs P, Mazor RD, Yackoubov D, Levy S, Stiff P, Rezvani A, Hanna R, Wagner J, Keating A, Lindemans CA, Karras N, McGuirk J, Hamerschlak N, Lopez-Torija I, Sanz G, Valcarcel D, Horwitz ME. Immune Reconstitution Profiling Suggests Antiviral Protection after Transplantation with Omidubicel: A Phase 3 Substudy. Transplant Cell Ther. 2023 Aug;29(8):517.e1-517.e12. doi: 10.1016/j.jtct.2023.04.018. Epub 2023 Apr 28.
PubMed 37120136Horwitz ME, Stiff PJ, Cutler C, Brunstein C, Hanna R, Maziarz RT, Rezvani AR, Karris NA, McGuirk J, Valcarcel D, Schiller GJ, Lindemans CA, Hwang WYK, Koh LP, Keating A, Khaled Y, Hamerschlak N, Frankfurt O, Peled T, Segalovich I, Blackwell B, Wease S, Freedman LS, Galamidi-Cohen E, Sanz G. Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study. Blood. 2021 Oct 21;138(16):1429-1440. doi: 10.1182/blood.2021011719.
PubMed 34157093Del Pozo Martin Y. 47th Annual Meeting of the EBMT. Lancet Haematol. 2021 May;8(5):e317-e318. doi: 10.1016/S2352-3026(21)00104-6. Epub 2021 Mar 31. No abstract available.
PubMed 33811823
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Phase III Registration Trial of Transplantation of NiCord®, Ex Vivo Expanded, UCB-derived, Stem and Progenitor Cells, vs. Unmanipulated UCB for Patients With Hematological Malignancies
- ClinicalTrials.gov ID
- NCT02730299
- Acronym / sponsor ID
- GC P#05.01.020
- Conditions
- Hematological Malignancies, Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML), Chronic Myelogenous Leukemia (CML), Myelodysplastic Syndrome (MDS), Lymphoma
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 125 participants (actual)
- Lead sponsor
- Gamida Cell ltdIndustry (pharma, biotech, devices)
- Study start
- 16 Dec 2016 (actual)
- Primary completion
- 29 Apr 2021 (actual)
- Study completion
- 7 Feb 2025 (actual)
- First posted
- 6 Apr 2016 (estimated)
- Results
- Posted 25 Oct 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is an open-label, controlled, multicenter, international, Phase III, randomized study of transplantation of NiCord® versus transplantation of one or two unmanipulated, unrelated cord blood units in patients with acute lymphoblastic leukemia or acute myeloid leukemia, myelodysplastic syndrome, chronic myeloid leukemia or lymphoma, all with required disease features rendering them eligible for allogeneic transplantation.
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.
A major drawback of Umbilical Cord Blood (UCB) is the low stem cell dose available for transplantation, compared to mobilized peripheral blood (PB) or bone marrow. This low stem cell dose can compromise the chances of engraftment and contributes to delayed kinetics of neutrophil and platelet recovery, as well as other transplant outcomes.
The aim of ex vivo expansion of cord blood is to provide a graft with sufficient numbers of cells that have rapid and robust in vivo neutrophil and platelet producing potential to enable successful transplantation.
NiCord® is a stem/progenitor cell-based product composed of ex vivo expanded allogeneic cells from one entire unit of UCB. NiCord® 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 chief aim of the study is to compare the safety and efficacy of NiCord® single ex-vivo expanded cord blood unit transplantation to unmanipulated cord blood unit transplantation in patients with hematological malignancies following conditioning therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NiCord® (omidubicel) | Experimental | Drug: NiCord® (omidubicel)NiCord® is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (NiCord® cultured fraction (CF)) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) (NiCord® Non-cultured Fraction (NF)) consisting of mature myeloid and lymphoid cells. Both fractions, i.e. NiCord® CF and NiCord® NF, will be kept frozen until they are thawed and infused on the day of transplantation. |
| Unmanipulated CBU(s) | Active comparator | Other: Cord Blood Unit |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Applicable disease criteria
- Patients must have one or two partially HLA-matched CBUs
- Back-up stem cell source
- Adequate Karnofsky/Lansky Performance score
- Sufficient physiological reserves
Key exclusion criteria (6 listed)
- HLA-matched donor able to donate
- Prior allogeneic HSCT
- Other active malignancy
- Active or uncontrolled infection
- Active/symptoms of central nervous system (CNS) disease
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 |
|---|---|---|
| NiCord® (omidubicel) | Drug | NiCord® (omidubicel) |
| Cord Blood UnitCord blood unit | Other | Unmanipulated CBU(s) |
Primary outcome
Time to Neutrophil Engraftment
Time frame: post-transplant up to 42 days
The time to engraftment of neutrophils >500/μl was defined as per Center for International Blood and Marrow Transplant Research (CIBMTR) standards, requiring donor chimerism for neutrophil engraftment.
3 secondary outcomes
First Grade 2/3 Bacterial or Invasive Fungal Infections by 100 Days Following Transplantation
Time frame: 100 days post-transplant
Days Alive and Out of Hospital in the First 100 Days Post-transplantation
Time frame: 100 days post-transplantation
Number of Participants With Platelet Engraftment by 42 Days Post-transplantation
Time frame: 42 days post-transplantation
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Feb 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37703995Majhail NS, Miller B, Dean R, Manghani R, Shin H, Sivaraman S, Maziarz RT. Hospitalization and Healthcare Resource Utilization of Omidubicel-Onlv versus Umbilical Cord Blood Transplantation for Hematologic Malignancies: Secondary Analysis from a Pivotal Phase 3 Clinical Trial. Transplant Cell Ther. 2023 Dec;29(12):749.e1-749.e5. doi: 10.1016/j.jtct.2023.09.004. Epub 2023 Sep 12.
- Derived from this study: PubMed 37120136Szabolcs P, Mazor RD, Yackoubov D, Levy S, Stiff P, Rezvani A, Hanna R, Wagner J, Keating A, Lindemans CA, Karras N, McGuirk J, Hamerschlak N, Lopez-Torija I, Sanz G, Valcarcel D, Horwitz ME. Immune Reconstitution Profiling Suggests Antiviral Protection after Transplantation with Omidubicel: A Phase 3 Substudy. Transplant Cell Ther. 2023 Aug;29(8):517.e1-517.e12. doi: 10.1016/j.jtct.2023.04.018. Epub 2023 Apr 28.
- Derived from this study: PubMed 34157093Horwitz ME, Stiff PJ, Cutler C, Brunstein C, Hanna R, Maziarz RT, Rezvani AR, Karris NA, McGuirk J, Valcarcel D, Schiller GJ, Lindemans CA, Hwang WYK, Koh LP, Keating A, Khaled Y, Hamerschlak N, Frankfurt O, Peled T, Segalovich I, Blackwell B, Wease S, Freedman LS, Galamidi-Cohen E, Sanz G. Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study. Blood. 2021 Oct 21;138(16):1429-1440. doi: 10.1182/blood.2021011719.
- Derived from this study: PubMed 33811823Del Pozo Martin Y. 47th Annual Meeting of the EBMT. Lancet Haematol. 2021 May;8(5):e317-e318. doi: 10.1016/S2352-3026(21)00104-6. Epub 2021 Mar 31. No abstract available.
- 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 study chair
Duke University
52 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing NiCord® (omidubicel) and Cord Blood Unit in people with Hematological Malignancies and Acute Lymphoblastic Leukemia (ALL), run by Gamida Cell ltd at 52 sites in 10 countries.
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 6 inclusion and 6 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 25 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 52 sites in United States, Spain, Israel, Brazil, United Kingdom and 5 more countries. 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 25 Oct 2022). 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 to Neutrophil Engraftment (time frame: post-transplant up to 42 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02730299, which also lists 4 linked publications.
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 25 Feb 2026; AzTrials last refreshed its copy on 26 Oct 2026.