Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard Umbilical Cord Blood in Patients With Leukemia, Lymphoma, and Myelodysplastic Syndrome (MDS)
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| ClinicalTrials.gov Identifier: NCT02730299 |
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Recruitment Status :
Active, not recruiting
First Posted : April 6, 2016
Last Update Posted : April 14, 2020
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| 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® (omidubicel) Other: Cord Blood Unit | Phase 3 |
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 Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 124 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 |
| Actual Primary Completion Date : | December 2019 |
| Estimated Study Completion Date : | December 2020 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: NiCord® (omidubicel)
NiCord® is a cryopreserved stem/progenitor cell based product comprised of:
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 |
- The time to neutrophil engraftment in participants following transplantation. [ Time Frame: must occur on or before 42 days post transplant ]
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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 |
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
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
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| Study Chair: | Mitchell Horwitz, MD | Duke University |
| Responsible Party: | Gamida Cell ltd |
| ClinicalTrials.gov Identifier: | NCT02730299 |
| Other Study ID Numbers: |
GC P#05.01.020 |
| First Posted: | April 6, 2016 Key Record Dates |
| Last Update Posted: | April 14, 2020 |
| Last Verified: | April 2020 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
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Lymphoma Leukemia Preleukemia Precursor Cell Lymphoblastic Leukemia-Lymphoma Leukemia, Myeloid Leukemia, Myelogenous, Chronic, BCR-ABL Positive Leukemia, Myeloid, Acute Hematologic Neoplasms Myelodysplastic Syndromes Syndrome Neoplasms Neoplasms by Histologic Type |
Lymphoproliferative Disorders Lymphatic Diseases Immunoproliferative Disorders Immune System Diseases Disease Pathologic Processes Bone Marrow Diseases Hematologic Diseases Precancerous Conditions Leukemia, Lymphoid Myeloproliferative Disorders Neoplasms by Site |