Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard Umbilical Cord Blood in Patients With Leukemia, Lymphoma, and Myelodysplastic Syndrome (MDS)
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
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
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: NiCord® (omidubicel) NiCord® is a cryopreserved stem/progenitor cell based product comprised of: ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (NiCord® cultured fraction (CF)) 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. |
Drug: NiCord® (omidubicel)
|
| Active Comparator: Unmanipulated CBU(s)
|
Other: Cord Blood Unit
Cord blood unit
|
Outcome Measures
Primary Outcome Measures
- The time to neutrophil engraftment in participants following transplantation. [must occur on or before 42 days post transplant]
Eligibility Criteria
Criteria
Inclusion Criteria:
-
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
Exclusion Criteria:
-
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | UCLA | Los Angeles | California | United States | 90095 |
| 2 | City of Hope | Los Angeles | California | United States | 91010 |
| 3 | Stanford University Cancer Institute | Palo Alto | California | United States | |
| 4 | UC San Diego Moores Cancer Center | San Diego | California | United States | 92093 |
| 5 | Children's Hospital Colorado | Denver | Colorado | United States | 80045 |
| 6 | Northwestern University | Evanston | Illinois | United States | 60208 |
| 7 | Loyola University, Cardinal Bernardin Cancer Center | Maywood | Illinois | United States | 60153 |
| 8 | University of Kansas Cancer Center | Westwood | Kansas | United States | 66205 |
| 9 | The University of Maryland Medicine Center | Baltimore | Maryland | United States | 21201 |
| 10 | Boston Children's Hospital | Boston | Massachusetts | United States | 02115 |
| 11 | Dana-Farber Cancer Institute | Boston | Massachusetts | United States | 02215 |
| 12 | Henry Ford Medical Center | Detroit | Michigan | United States | |
| 13 | University of Minnesota Masonic Cancer Center | Minneapolis | Minnesota | United States | 55455 |
| 14 | Rutgers Cancer Institute of New Jersey | New Brunswick | New Jersey | United States | 08901 |
| 15 | Duke University Medical Center | Durham | North Carolina | United States | 27710 |
| 16 | Cleveland Clinic Children's | Cleveland | Ohio | United States | 44195 |
| 17 | Oregon Health & Science University | Portland | Oregon | United States | 97239 |
| 18 | UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | United States | 15224 |
| 19 | West Cancer Clinic | Germantown | Tennessee | United States | 38138 |
| 20 | Children's Medical Center of Dallas | Dallas | Texas | United States | 75235 |
| 21 | University of Virginia Cancer Center | Charlottesville | Virginia | United States | 22903 |
| 22 | Instituto Nacional de Câncer José Alencar Gomes da Silva - INCA | Rio De Janeiro | Brazil | 20230-130 | |
| 23 | Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo Pediatrics | São Paulo | Brazil | 05403-010 | |
| 24 | Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo | São Paulo | Brazil | 05403-010 | |
| 25 | Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo | São Paulo | Brazil | 14048-900 | |
| 26 | Hospital Israelita Albert Einstein | São Paulo | Brazil | ||
| 27 | Robert Debré | Paris | France | 75019 | |
| 28 | Rambam | Haifa | Israel | ||
| 29 | Hadassah Medical Center | Jerusalem | Israel | ||
| 30 | Rabin Medical Center, Beilinson Hospital | Petach Tikva | Israel | ||
| 31 | Dana-Dwek Children's Hospital, Tel-Aviv Sourasky Medical Center | Tel Aviv | Israel | ||
| 32 | Tel-Aviv Sourasky Medical Center | Tel Aviv | Israel | ||
| 33 | Chaim Sheba Medical Center, The Edmond and Lily Safra Children's hospital | Tel HaShomer | Israel | ||
| 34 | Careggi University Hospital | Florence | Italy | 50134 | |
| 35 | Ospedale Pediatrico Bambino Gesù | Rome | Italy | 00165 | |
| 36 | University Medical Center Utrecht | Utrecht | Netherlands | 3503 AB | |
| 37 | Prinses Maxima Centrum voor Kinderoncologie B.V. | Utrecht | Netherlands | 3584 CS | |
| 38 | Instituto Português de Oncologia de Lisboa Francisco Gentil | Lisbon | Portugal | 1099-023 | |
| 39 | National University Cancer Institute | Singapore | Singapore | 119074 | |
| 40 | Singapore General Hospital | Singapore | Singapore | 169608 | |
| 41 | Hospital Universitari Vall d'Hebron pediatrics | Barcelona | Spain | 08035 | |
| 42 | University Hospital Vall d'Hebron | Barcelona | Spain | 08035 | |
| 43 | ICO Bellvitge | Barcelona | Spain | 08908 | |
| 44 | Sant Joan de Deu | Barcelona | Spain | 08950 | |
| 45 | Hospital de la Santa Creu i Sant Pau | Barcelona | Spain | ||
| 46 | Hospital Universitario Virgen del Rocío | Sevilla | Spain | ||
| 47 | Hospital Universitario La Fe | Valencia | Spain | 46009 | |
| 48 | Hospital Universitario y Politécnico La Fe (pediatric) | Valencia | Spain | ||
| 49 | The Royal Marsden NHS Foundation Trust | Sutton | Surrey | United Kingdom | SM2 5PT |
| 50 | Queen Elizabeth Hospital | Birmingham | United Kingdom | B15 2GW | |
| 51 | St James Hospital | Leeds | United Kingdom | LS9 7TF | |
| 52 | Manchester University NHS Foundation Trust | Manchester | United Kingdom | M13 9WL |
Sponsors and Collaborators
- Gamida Cell ltd
Investigators
- Study Chair: Mitchell Horwitz, MD, Duke University
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- GC P#05.01.020