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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04260698
Recruitment Status : Not yet recruiting
First Posted : February 7, 2020
Last Update Posted : February 12, 2020
Sponsor:
Information provided by (Responsible Party):
Gamida Cell ltd

Tracking Information
First Submitted Date  ICMJE February 5, 2020
First Posted Date  ICMJE February 7, 2020
Last Update Posted Date February 12, 2020
Estimated Study Start Date  ICMJE June 15, 2020
Estimated Primary Completion Date December 31, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 5, 2020)
To assess the time from transplant to neutrophil engraftment [ Time Frame: by day 42 post-transplant inclusive ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Expanded Access of Omidubicel, for Allogeneic Transplantation in Patients With Hematological Malignancies
Official Title  ICMJE An Open Label Expanded Access Study of Omidubicel, for Allogeneic Transplantation in Patients With Hematological Malignancies
Brief Summary Omidubicel is an investigational therapy for patients with high-risk hematologic malignancies.
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hematological Malignancies
Intervention  ICMJE Biological: omidubicel
hematopoietic stem cell transplant
Other Name: NiCord
Study Arms  ICMJE Experimental: omidubicel

Omidubicel is a cryopreserved stem/progenitor cell based product comprised of:

  1. Ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (cultured fraction (CF))
  2. 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.

Intervention: Biological: omidubicel
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: February 5, 2020)
60
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 31, 2024
Estimated Primary Completion Date December 31, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Kelly Myers +97226595631 kelly@gamida-cell.com
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04260698
Other Study ID Numbers  ICMJE GC P#07.01.020
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Gamida Cell ltd
Study Sponsor  ICMJE Gamida Cell ltd
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Mitchell Horwitz, MD Duke University
PRS Account Gamida Cell ltd
Verification Date February 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP