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Transplantation of Ex Vivo Expanded, UCB-derived, Stem & Progenitor Cells vs. Unmanipulated UCB for HM Patients

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ClinicalTrials.gov Identifier: NCT02730299
Recruitment Status : Recruiting
First Posted : April 6, 2016
Last Update Posted : November 14, 2018
Sponsor:
Information provided by (Responsible Party):
Gamida Cell ltd

Study Description
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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
Hematological Malignancies Acute Lymphoblastic Leukemia (ALL) Acute Myelogenous Leukemia (AML) Chronic Myelogenous Leukemia (CML) Myelodysplastic Syndrome (MDS) Lymphoma Acute Leukemia Drug: NiCord® Other: Cord Blood Unit 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
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 120 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : November 2016
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2020

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: NiCord

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.

 Drug: NiCord®
Active Comparator: Unmanipulated CBU(s) Other: Cord Blood Unit
Cord blood unit


Outcome Measures
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Primary Outcome Measures :
  1. The time to neutrophil engraftment in participants following transplantation. [ Time Frame: must occur on or before 42 days post transplant ]

Eligibility Criteria
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Ages Eligible for Study:   12 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02730299


Contacts
Contact: Kelly Myers +972-2-6595631 clinicaltrials@gamida-cell.com

Locations
United States, California
UCLA Recruiting
Los Angeles, California, United States, 90095
Contact: Gary Schiller, MD       GSchiller@mednet.ucla.edu   
Principal Investigator: Gary Schiller, MD         
City of Hope Recruiting
Los Angeles, California, United States, 91010
Principal Investigator: Nicole Karras, MD         
United States, Illinois
Loyola University, Cardinal Bernardin Cancer Center Recruiting
Maywood, Illinois, United States, 60153
Contact: Patrick Stiff, MD    708-327-3216    pstiff@lumc.edu   
Principal Investigator: Patrick Stiff, MD         
United States, Kansas
University of Kansas Cancer Center Recruiting
Westwood, Kansas, United States, 66205
Contact: Joseph McGuirk, MD    913-945-6591    jmcguirk@kumc.edu   
Principal Investigator: Joseph McGuirk, MD         
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Corey Cutler, MD    617-851-2852    cscutler@partners.org   
Principal Investigator: Corey Cutler, MD         
Dana-Farber Cancer Institute Recruiting
Boston, Massachusetts, United States, 02215
Contact: Corey Cutler, MD    617-851-2852    cscutler@partners.org   
Principal Investigator: Corey Cutler, MD         
United States, Minnesota
University of Minnesota Masonic Cancer Center Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Claudio Brunstein, MD    612-625-3918    bruns072@umn.edu   
Principal Investigator: Claudio Brunstein, MD         
United States, North Carolina
Duke University Medical Center Recruiting
Durham, North Carolina, United States, 27710
Contact: Mitchell Horwitz, MD    919-668-1045    mitchell.horwitz@duke.edu   
Principal Investigator: Mitchell Horwitz, MD         
United States, Ohio
Cleveland Clinic Children's Recruiting
Cleveland, Ohio, United States, 44195
Contact: Rabi Hanna, MD    216-444-0663    Hannar2@ccf.org   
Principal Investigator: Rabi Hanna, MD         
United States, Oregon
Oregon Health & Science University Recruiting
Portland, Oregon, United States, 97239
Contact: Richard Maziarz, MD    503-494-4606    maziarzr@ohsu.edu   
Principal Investigator: Richard Maziarz, MD         
Israel
Rambam Recruiting
Haifa, Israel
Contact: Tsila Zuckerman, MD    +972-4-777-3248    t_zuckerman@rambam.health.gov.il   
Hadassah Medical Center Recruiting
Jerusalem, Israel
Contact: Batia Avni, MD         
Netherlands
University Medical Center Utrecht Recruiting
Utrecht, Netherlands, 3503 AB
Contact: Jaap Jan Boelens, MD    +31-88-7554003    J.J.Boelens@umcutrecht.nl   
Principal Investigator: Jaap Jan Boelens, MD         
Prinses Maxima Centrum voor Kinderoncologie B.V. Recruiting
Utrecht, Netherlands, 3584 CS
Contact: Jaap Jan Boelens, MD, PhD    +31 88 972 7272    J.J.Boelens@prinsesmaximacentrum.nl   
Principal Investigator: Jaap Jan Boelens, MD, PhD         
Singapore
National University Cancer Institute Recruiting
Singapore, Singapore, 119074
Contact: Liang Piu Koh, MD    (65) 6779 5555    liang_piu_koh@nuhs.edu.sg   
Principal Investigator: Liang Piu Koh, MD         
Singapore General Hospital Recruiting
Singapore, Singapore, 169608
Contact: William Hwang Ying Khee, MD    6222 3322    william.hwang.y.k@singhealth.com.sg   
Principal Investigator: William Hwang Ying Khee, MD         
Spain
University Hospital Vall d'Hebron Recruiting
Barcelona, Spain, 08035
Contact: David Valcárcel, MD, PhD    +34-934 893 000 ext 6417    dvalcarcel.vhebron@me.com   
Principal Investigator: David Valcárcel, MD, PhD         
Hospital de la Santa Creu i Sant Pau Recruiting
Barcelona, Spain
Contact: Luisa Sisinni, MD    +34 932 91 90 00      
Hospital Universitario La Fe Recruiting
Valencia, Spain, 46009
Contact: Guillermo Sanz, MD    +34 (96) 386-2709    sanz_gui@gva.es   
Principal Investigator: Guillermo Sanz, MD         
Sponsors and Collaborators
Gamida Cell ltd
More Information
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Responsible Party: Gamida Cell ltd
ClinicalTrials.gov Identifier: NCT02730299     History of Changes
Other Study ID Numbers: GC P#05.01.020
First Posted: April 6, 2016    Key Record Dates
Last Update Posted: November 14, 2018
Last Verified: November 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Leukemia
Neoplasms
Myelodysplastic Syndromes
Preleukemia
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Leukemia, Lymphoid
Leukemia, Myeloid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Leukemia, Myeloid, Acute
 Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Myeloproliferative Disorders