Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in Patients With Leukemia, Lymphoma, and MDS
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
1 other identifier
interventional
125
10 countries
52
Brief 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 myeloid leukemia or lymphoma, all with required disease features rendering them eligible for allogeneic transplantation.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Dec 2016
Longer than P75 for phase_3
52 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 28, 2016
CompletedFirst Posted
Study publicly available on registry
April 6, 2016
CompletedStudy Start
First participant enrolled
December 16, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 29, 2021
CompletedResults Posted
Study results publicly available
October 25, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
February 7, 2025
CompletedFebruary 25, 2026
February 1, 2026
4.4 years
March 28, 2016
March 28, 2022
February 22, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
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.
post-transplant up to 42 days
Secondary Outcomes (3)
First Grade 2/3 Bacterial or Invasive Fungal Infections by 100 Days Following Transplantation
100 days post-transplant
Days Alive and Out of Hospital in the First 100 Days Post-transplantation
100 days post-transplantation
Number of Participants With Platelet Engraftment by 42 Days Post-transplantation
42 days post-transplantation
Other Outcomes (4)
LTF Chimerism
By 5 years post-transplant
LTF Overall Survival
By 5 years post-transplant
LTF Disease Progression/Relapse
By 5 years post-transplant
- +1 more other outcomes
Study Arms (2)
NiCord® (omidubicel)
EXPERIMENTALNiCord® 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 COMPARATORInterventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Gamida Cell ltdlead
Study Sites (52)
UCLA
Los Angeles, California, 90095, United States
City of Hope
Los Angeles, California, 91010, United States
Stanford University Cancer Institute
Palo Alto, California, United States
UC San Diego Moores Cancer Center
San Diego, California, 92093, United States
Children's Hospital Colorado
Denver, Colorado, 80045, United States
Northwestern University
Evanston, Illinois, 60208, United States
Loyola University, Cardinal Bernardin Cancer Center
Maywood, Illinois, 60153, United States
University of Kansas Cancer Center
Westwood, Kansas, 66205, United States
The University of Maryland Medicine Center
Baltimore, Maryland, 21201, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215, United States
Henry Ford Medical Center
Detroit, Michigan, United States
University of Minnesota Masonic Cancer Center
Minneapolis, Minnesota, 55455, United States
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, 08901, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
Cleveland Clinic Children's
Cleveland, Ohio, 44195, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
West Cancer Clinic
Germantown, Tennessee, 38138, United States
Children's Medical Center of Dallas
Dallas, Texas, 75235, United States
University of Virginia Cancer Center
Charlottesville, Virginia, 22903, United States
Instituto Nacional de Câncer José Alencar Gomes da Silva - INCA
Rio de Janeiro, Rio de Janeiro, 20230-130, Brazil
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo Pediatrics
São Paulo, São Paulo, 05403-010, Brazil
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
São Paulo, São Paulo, 05403-010, Brazil
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo
São Paulo, São Paulo, 14048-900, Brazil
Hospital Israelita Albert Einstein
São Paulo, Brazil
Robert Debré
Paris, 75019, France
Rambam
Haifa, Israel
Hadassah Medical Center
Jerusalem, Israel
Rabin Medical Center, Beilinson Hospital
Petah Tikva, Israel
Dana-Dwek Children's Hospital, Tel-Aviv Sourasky Medical Center
Tel Aviv, Israel
Tel-Aviv Sourasky Medical Center
Tel Aviv, Israel
Chaim Sheba Medical Center, The Edmond and Lily Safra Children's hospital
Tel Litwinsky, Israel
Careggi University Hospital
Florence, 50134, Italy
Ospedale Pediatrico Bambino Gesù
Rome, 00165, Italy
University Medical Center Utrecht
Utrecht, 3503 AB, Netherlands
Prinses Maxima Centrum voor Kinderoncologie B.V.
Utrecht, 3584 CS, Netherlands
Instituto Português de Oncologia de Lisboa Francisco Gentil
Lisbon, 1099-023, Portugal
National University Cancer Institute
Singapore, 119074, Singapore
Singapore General Hospital
Singapore, 169608, Singapore
Hospital Universitari Vall d'Hebron pediatrics
Barcelona, 08035, Spain
University Hospital Vall d'Hebron
Barcelona, 08035, Spain
ICO Bellvitge
Barcelona, 08908, Spain
Sant Joan de Deu
Barcelona, 08950, Spain
Hospital de la Santa Creu i Sant Pau
Barcelona, Spain
Hospital Universitario Virgen del Rocío
Seville, Spain
Hospital Universitario La Fe
Valencia, 46009, Spain
Hospital Universitario y Politécnico La Fe (pediatric)
Valencia, Spain
The Royal Marsden NHS Foundation Trust
Sutton, Surrey, SM2 5PT, United Kingdom
Queen Elizabeth Hospital
Birmingham, B15 2GW, United Kingdom
St James Hospital
Leeds, LS9 7TF, United Kingdom
Manchester University NHS Foundation Trust
Manchester, M13 9WL, United Kingdom
Related Publications (4)
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.
PMID: 37703995DERIVEDSzabolcs 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.
PMID: 37120136DERIVEDHorwitz 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.
PMID: 34157093DERIVEDDel 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.
PMID: 33811823DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Limitations and Caveats
The analyses of the long term follow-up sub-study were descriptive only and the study was not powered to detect statistical differences between the treatment groups.
Results Point of Contact
- Title
- Associate Director, Clinical Operations
- Organization
- Gamida Cell
Study Officials
- STUDY CHAIR
Mitchell Horwitz, MD
Duke University
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 28, 2016
First Posted
April 6, 2016
Study Start
December 16, 2016
Primary Completion
April 29, 2021
Study Completion
February 7, 2025
Last Updated
February 25, 2026
Results First Posted
October 25, 2022
Record last verified: 2026-02
Data Sharing
- IPD Sharing
- Will not share