Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in Patients With Leukemia, Lymphoma, and MDS
Primary Purpose
Hematological Malignancies, Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML)
Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
NiCord® (omidubicel)
Cord Blood Unit
Sponsored by
About this trial
This is an interventional treatment trial for Hematological Malignancies
Eligibility 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
Sites / Locations
- UCLA
- City of Hope
- Stanford University Cancer Institute
- UC San Diego Moores Cancer Center
- Children's Hospital Colorado
- Northwestern University
- Loyola University, Cardinal Bernardin Cancer Center
- University of Kansas Cancer Center
- The University of Maryland Medicine Center
- Boston Children's Hospital
- Dana-Farber Cancer Institute
- Henry Ford Medical Center
- University of Minnesota Masonic Cancer Center
- Rutgers Cancer Institute of New Jersey
- Duke University Medical Center
- Cleveland Clinic Children's
- Oregon Health & Science University
- UPMC Children's Hospital of Pittsburgh
- West Cancer Clinic
- Children's Medical Center of Dallas
- University of Virginia Cancer Center
- Instituto Nacional de Câncer José Alencar Gomes da Silva - INCA
- Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo Pediatrics
- Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
- Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo
- Hospital Israelita Albert Einstein
- Robert Debré
- Rambam
- Hadassah Medical Center
- Rabin Medical Center, Beilinson Hospital
- Dana-Dwek Children's Hospital, Tel-Aviv Sourasky Medical Center
- Tel-Aviv Sourasky Medical Center
- Chaim Sheba Medical Center, The Edmond and Lily Safra Children's hospital
- Careggi University Hospital
- Ospedale Pediatrico Bambino Gesù
- University Medical Center Utrecht
- Prinses Maxima Centrum voor Kinderoncologie B.V.
- Instituto Português de Oncologia de Lisboa Francisco Gentil
- National University Cancer Institute
- Singapore General Hospital
- Hospital Universitari Vall d'Hebron pediatrics
- University Hospital Vall d'Hebron
- ICO Bellvitge
- Sant Joan de Deu
- Hospital de la Santa Creu i Sant Pau
- Hospital Universitario Virgen del Rocío
- Hospital Universitario La Fe
- Hospital Universitario y Politécnico La Fe (pediatric)
- The Royal Marsden NHS Foundation Trust
- Queen Elizabeth Hospital
- St James Hospital
- Manchester University NHS Foundation Trust
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Active Comparator
Arm Label
NiCord® (omidubicel)
Unmanipulated CBU(s)
Arm Description
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.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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
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
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
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02730299
Brief Title
Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in Patients With Leukemia, Lymphoma, and MDS
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
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
December 16, 2016 (Actual)
Primary Completion Date
April 29, 2021 (Actual)
Study Completion Date
February 18, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Gamida Cell ltd
4. Oversight
Data Monitoring Committee
Yes
5. 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hematological Malignancies, Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML), Chronic Myelogenous Leukemia (CML), Myelodysplastic Syndrome (MDS), Lymphoma, Acute Leukemia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
125 (Actual)
8. Arms, Groups, and Interventions
Arm Title
NiCord® (omidubicel)
Arm Type
Experimental
Arm Description
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.
Arm Title
Unmanipulated CBU(s)
Arm Type
Active Comparator
Intervention Type
Drug
Intervention Name(s)
NiCord® (omidubicel)
Intervention Type
Other
Intervention Name(s)
Cord Blood Unit
Intervention Description
Cord blood unit
Primary Outcome Measure Information:
Title
Time to Neutrophil Engraftment
Description
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.
Time Frame
post-transplant up to 42 days
Secondary Outcome Measure Information:
Title
First Grade 2/3 Bacterial or Invasive Fungal Infections by 100 Days Following Transplantation
Description
First Bacterial Infection Grades 2-3 or Invasive Fungal Infection by 100 Days following Transplantation for the Intent to Treat Population
Time Frame
100 days post-transplant
Title
Days Alive and Out of Hospital in the First 100 Days Post-transplantation
Description
Days alive and out of hospital in the first 100 Days post-transplantation for the Intent to Treat Population
Time Frame
100 days post-transplantation
Title
Number of Participants With Platelet Engraftment by 42 Days Post-transplantation
Description
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
Time Frame
42 days post-transplantation
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mitchell Horwitz, MD
Organizational Affiliation
Duke University
Official's Role
Study Chair
Facility Information:
Facility Name
UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
City of Hope
City
Los Angeles
State/Province
California
ZIP/Postal Code
91010
Country
United States
Facility Name
Stanford University Cancer Institute
City
Palo Alto
State/Province
California
Country
United States
Facility Name
UC San Diego Moores Cancer Center
City
San Diego
State/Province
California
ZIP/Postal Code
92093
Country
United States
Facility Name
Children's Hospital Colorado
City
Denver
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Northwestern University
City
Evanston
State/Province
Illinois
ZIP/Postal Code
60208
Country
United States
Facility Name
Loyola University, Cardinal Bernardin Cancer Center
City
Maywood
State/Province
Illinois
ZIP/Postal Code
60153
Country
United States
Facility Name
University of Kansas Cancer Center
City
Westwood
State/Province
Kansas
ZIP/Postal Code
66205
Country
United States
Facility Name
The University of Maryland Medicine Center
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21201
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Dana-Farber Cancer Institute
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Facility Name
Henry Ford Medical Center
City
Detroit
State/Province
Michigan
Country
United States
Facility Name
University of Minnesota Masonic Cancer Center
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Rutgers Cancer Institute of New Jersey
City
New Brunswick
State/Province
New Jersey
ZIP/Postal Code
08901
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Cleveland Clinic Children's
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
UPMC Children's Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
West Cancer Clinic
City
Germantown
State/Province
Tennessee
ZIP/Postal Code
38138
Country
United States
Facility Name
Children's Medical Center of Dallas
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Facility Name
University of Virginia Cancer Center
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22903
Country
United States
Facility Name
Instituto Nacional de Câncer José Alencar Gomes da Silva - INCA
City
Rio De Janeiro
ZIP/Postal Code
20230-130
Country
Brazil
Facility Name
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo Pediatrics
City
São Paulo
ZIP/Postal Code
05403-010
Country
Brazil
Facility Name
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
City
São Paulo
ZIP/Postal Code
05403-010
Country
Brazil
Facility Name
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo
City
São Paulo
ZIP/Postal Code
14048-900
Country
Brazil
Facility Name
Hospital Israelita Albert Einstein
City
São Paulo
Country
Brazil
Facility Name
Robert Debré
City
Paris
ZIP/Postal Code
75019
Country
France
Facility Name
Rambam
City
Haifa
Country
Israel
Facility Name
Hadassah Medical Center
City
Jerusalem
Country
Israel
Facility Name
Rabin Medical Center, Beilinson Hospital
City
Petach Tikva
Country
Israel
Facility Name
Dana-Dwek Children's Hospital, Tel-Aviv Sourasky Medical Center
City
Tel Aviv
Country
Israel
Facility Name
Tel-Aviv Sourasky Medical Center
City
Tel Aviv
Country
Israel
Facility Name
Chaim Sheba Medical Center, The Edmond and Lily Safra Children's hospital
City
Tel HaShomer
Country
Israel
Facility Name
Careggi University Hospital
City
Florence
ZIP/Postal Code
50134
Country
Italy
Facility Name
Ospedale Pediatrico Bambino Gesù
City
Rome
ZIP/Postal Code
00165
Country
Italy
Facility Name
University Medical Center Utrecht
City
Utrecht
ZIP/Postal Code
3503 AB
Country
Netherlands
Facility Name
Prinses Maxima Centrum voor Kinderoncologie B.V.
City
Utrecht
ZIP/Postal Code
3584 CS
Country
Netherlands
Facility Name
Instituto Português de Oncologia de Lisboa Francisco Gentil
City
Lisbon
ZIP/Postal Code
1099-023
Country
Portugal
Facility Name
National University Cancer Institute
City
Singapore
ZIP/Postal Code
119074
Country
Singapore
Facility Name
Singapore General Hospital
City
Singapore
ZIP/Postal Code
169608
Country
Singapore
Facility Name
Hospital Universitari Vall d'Hebron pediatrics
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
University Hospital Vall d'Hebron
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
ICO Bellvitge
City
Barcelona
ZIP/Postal Code
08908
Country
Spain
Facility Name
Sant Joan de Deu
City
Barcelona
ZIP/Postal Code
08950
Country
Spain
Facility Name
Hospital de la Santa Creu i Sant Pau
City
Barcelona
Country
Spain
Facility Name
Hospital Universitario Virgen del Rocío
City
Sevilla
Country
Spain
Facility Name
Hospital Universitario La Fe
City
Valencia
ZIP/Postal Code
46009
Country
Spain
Facility Name
Hospital Universitario y Politécnico La Fe (pediatric)
City
Valencia
Country
Spain
Facility Name
The Royal Marsden NHS Foundation Trust
City
Sutton
State/Province
Surrey
ZIP/Postal Code
SM2 5PT
Country
United Kingdom
Facility Name
Queen Elizabeth Hospital
City
Birmingham
ZIP/Postal Code
B15 2GW
Country
United Kingdom
Facility Name
St James Hospital
City
Leeds
ZIP/Postal Code
LS9 7TF
Country
United Kingdom
Facility Name
Manchester University NHS Foundation Trust
City
Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
34157093
Citation
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.
Results Reference
derived
PubMed Identifier
33811823
Citation
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.
Results Reference
derived
Learn more about this trial
Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in Patients With Leukemia, Lymphoma, and MDS
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