Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in Patients With Leukemia, Lymphoma, and MDS
NCT ID: NCT02730299
Last Updated: 2025-04-06
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE3
125 participants
INTERVENTIONAL
2016-12-16
2025-02-07
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Transplantation of NiCord®, Umbilical Cord Blood-derived Ex Vivo Expanded Cells, in Patients With HM
NCT01816230
Pilot Study Evaluating Safety & Efficacy of DCBT: NiCord® & UNM CBU to Patients With Hematological Malignancies
NCT01221857
Donor Umbilical Cord Blood Transplant With or Without Ex-vivo Expanded Cord Blood Progenitor Cells in Treating Patients With Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, or Myelodysplastic Syndromes
NCT01690520
Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome
NCT03173937
Infusion of Expanded Cord Blood Cells in Addition to Single Cord Blood Transplant in Treating Patients With Acute Leukemia, Chronic Myeloid Leukemia, or Myelodysplastic Syndromes
NCT03399773
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
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
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
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.
NiCord® (omidubicel)
Unmanipulated CBU(s)
Cord Blood Unit
Cord blood unit
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
NiCord® (omidubicel)
Cord Blood Unit
Cord blood unit
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion 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
Exclusion Criteria
* Prior allogeneic HSCT
* Other active malignancy
* Active or uncontrolled infection
* Active/symptoms of central nervous system (CNS) disease
* Pregnancy or lactation
12 Years
65 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Gamida Cell ltd
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Mitchell Horwitz, MD
Role: STUDY_CHAIR
Duke University
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
UCLA
Los Angeles, California, United States
City of Hope
Los Angeles, California, United States
Stanford University Cancer Institute
Palo Alto, California, United States
UC San Diego Moores Cancer Center
San Diego, California, United States
Children's Hospital Colorado
Denver, Colorado, United States
Northwestern University
Evanston, Illinois, United States
Loyola University, Cardinal Bernardin Cancer Center
Maywood, Illinois, United States
University of Kansas Cancer Center
Westwood, Kansas, United States
The University of Maryland Medicine Center
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, United States
Henry Ford Medical Center
Detroit, Michigan, United States
University of Minnesota Masonic Cancer Center
Minneapolis, Minnesota, United States
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, United States
Duke University Medical Center
Durham, North Carolina, United States
Cleveland Clinic Children's
Cleveland, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
West Cancer Clinic
Germantown, Tennessee, United States
Children's Medical Center of Dallas
Dallas, Texas, United States
University of Virginia Cancer Center
Charlottesville, Virginia, United States
Instituto Nacional de Câncer José Alencar Gomes da Silva - INCA
Rio de Janeiro, , Brazil
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo Pediatrics
São Paulo, , Brazil
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
São Paulo, , Brazil
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo
São Paulo, , Brazil
Hospital Israelita Albert Einstein
São Paulo, , Brazil
Robert Debré
Paris, , 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, , Italy
Ospedale Pediatrico Bambino Gesù
Rome, , Italy
University Medical Center Utrecht
Utrecht, , Netherlands
Prinses Maxima Centrum voor Kinderoncologie B.V.
Utrecht, , Netherlands
Instituto Português de Oncologia de Lisboa Francisco Gentil
Lisbon, , Portugal
National University Cancer Institute
Singapore, , Singapore
Singapore General Hospital
Singapore, , Singapore
Hospital Universitari Vall d'Hebron pediatrics
Barcelona, , Spain
University Hospital Vall d'Hebron
Barcelona, , Spain
ICO Bellvitge
Barcelona, , Spain
Sant Joan de Deu
Barcelona, , Spain
Hospital de la Santa Creu i Sant Pau
Barcelona, , Spain
Hospital Universitario Virgen del Rocío
Seville, , Spain
Hospital Universitario La Fe
Valencia, , Spain
Hospital Universitario y Politécnico La Fe (pediatric)
Valencia, , Spain
The Royal Marsden NHS Foundation Trust
Sutton, Surrey, United Kingdom
Queen Elizabeth Hospital
Birmingham, , United Kingdom
St James Hospital
Leeds, , United Kingdom
Manchester University NHS Foundation Trust
Manchester, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
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.
Szabolcs 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.
Horwitz 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.
Del 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.
Provided Documents
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
GC P#05.01.020
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.