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

Phase 3

Active, not recruiting

• Conditions: Hematological Malignancies
• Interventions: Biological: omidubicel

• Registration Number: NCT04260698
• Lead Sponsor: Gamida Cell ltd

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 Timeline

• Study First Submitted: 2020-02-05
• Study First Submitted QC: 2020-02-05
• Study First Posted: 2020-02-07
• Study Start: 2020-07-08
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-22
• Last Update Posted: 2024-12-27
• Primary Completion: 2025-05-31
• Study Completion: 2025-05-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 36

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

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Exclusion Criteria

• Extensive bone marrow fibrosis
• Donor specific anti-HLA antibodies
• Pregnancy
• Medically unsuitable for transplant

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
omidubicel	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.

Primary Outcome Measures

Name	Time	Method
To assess the time from transplant to neutrophil engraftment	by day 42 post-transplant inclusive

Trial Locations

Locations (6)

UCLA; 🇺🇸 Los Angeles, California, United States

Loyola University, Cardinal Bernardin Cancer Center; 🇺🇸 Maywood, Illinois, United States

University of Minnesota Masonic Cancer Center; 🇺🇸 Minneapolis, Minnesota, United States

Duke University Medical Center; 🇺🇸 Durham, North Carolina, United States

Oregon Health & Science University; 🇺🇸 Portland, Oregon, United States

Stanford University Cancer Institute; 🇺🇸 Palo Alto, California, United States