Expanded Access of Omidubicel, for Allogeneic Transplantation in Patients With Hematological Malignancies

Sponsor
Gamida Cell ltd (Industry)
Overall Status
Recruiting
CT.gov ID
NCT04260698
Collaborator
(none)
60
Enrollment
6
Locations
1
Arm
65.8
Anticipated Duration (Months)
10
Patients Per Site
0.2
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Omidubicel is an investigational therapy for patients with high-risk hematologic malignancies.

Condition or DiseaseIntervention/TreatmentPhase
  • Biological: omidubicel
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.

Omidubicel is a stem/progenitor cell-based product composed of ex vivo expanded allogeneic cells from one entire unit of umbilical cord blood. Omidubicel 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 overall study objectives are to provide access to omidubicel for transplantation in patients with hematological malignancies and to collect additional safety and efficacy data.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
60 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
An Open Label Expanded Access Study of Omidubicel, for Allogeneic Transplantation in Patients With Hematological Malignancies
Actual Study Start Date :
Jul 8, 2020
Anticipated Primary Completion Date :
Dec 31, 2023
Anticipated Study Completion Date :
Dec 31, 2025

Arms and Interventions

ArmIntervention/Treatment
Experimental: omidubicel

Omidubicel is a cryopreserved stem/progenitor cell based product comprised of: Ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF)) the non-cultured cell fraction of the same Cord Blood Unit (CBU) (Non-cultured Fraction (NF)) consisting of mature myeloid and lymphoid cells. Both fractions, i.e. CF and NF, will be kept frozen until they are thawed and infused on the day of transplantation.

Biological: omidubicel
hematopoietic stem cell transplant
Other Names:
  • NiCord
  • Outcome Measures

    Primary Outcome Measures

    1. To assess the time from transplant to neutrophil engraftment [by day 42 post-transplant inclusive]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    12 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    • Patients must be at least 12 years of age

    • Applicable disease criteria

    • Patients must have one or two partially HLA-matched CBUs

    • Back-up stem cell source

    • Sufficient physiological reserves

    • Females of childbearing potential agree to use appropriate method of contraception

    • Signed written informed consent

    Exclusion Criteria:
    • Extensive bone marrow fibrosis

    • Donor specific anti-HLA antibodies

    • Pregnancy

    • Medically unsuitable for transplant

    Contacts and Locations

    Locations

    SiteCityStateCountryPostal Code
    1UCLALos AngelesCaliforniaUnited States90095
    2Stanford University Cancer InstitutePalo AltoCaliforniaUnited States94063
    3Loyola University, Cardinal Bernardin Cancer CenterMaywoodIllinoisUnited States60153
    4University of Minnesota Masonic Cancer CenterMinneapolisMinnesotaUnited States55455
    5Duke University Medical CenterDurhamNorth CarolinaUnited States27710
    6Oregon Health & Science UniversityPortlandOregonUnited States97239

    Sponsors and Collaborators

    • Gamida Cell ltd

    Investigators

    • Principal Investigator: Mitchell Horwitz, MD, Duke University

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Gamida Cell ltd
    ClinicalTrials.gov Identifier:
    NCT04260698
    Other Study ID Numbers:
    • GC P#07.01.020
    First Posted:
    Feb 7, 2020
    Last Update Posted:
    Apr 1, 2022
    Last Verified:
    Mar 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Apr 1, 2022