Omidubicel for Blood Cancers

No longer recruiting at 5 trial locations

Overseen By Kelly Myers

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Gamida Cell ltd

Disqualifiers: Bone marrow fibrosis, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 1

Duration: 1 day

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called omidubicel for individuals with high-risk blood cancers. Omidubicel is a new therapy that uses special blood cells from umbilical cords to aid patients needing a stem cell transplant. The trial aims to determine if omidubicel can better support patients during recovery. Suitable candidates for this trial include those diagnosed with serious blood cancers who have access to partially matched cord blood units for transplant. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications.

Is there any evidence suggesting that Omidubicel is likely to be safe for humans?

Research shows that the FDA has approved omidubicel for some patients with blood cancers, indicating it has undergone safety testing. In earlier studies, omidubicel helped the immune system recover faster and resulted in fewer infections compared to traditional treatments. However, a serious safety concern exists: omidubicel carries a warning for potentially life-threatening reactions during infusion and a condition called graft-versus-host disease, where donor cells attack the patient's body. While it can be effective, discussing these potential risks with a healthcare provider is crucial before deciding to join a trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for blood cancers, which often involve chemotherapy and stem cell transplants from bone marrow or peripheral blood, Omidubicel is unique because it uses a novel approach with umbilical cord blood-derived cells. This treatment combines ex vivo expanded hematopoietic CD34+ progenitor cells with mature myeloid and lymphoid cells, offering a potentially more effective way to boost the immune system and produce blood cells. Researchers are excited about Omidubicel because it could lead to quicker recovery times and reduce the risk of infections compared to traditional stem cell transplants.

What evidence suggests that Omidubicel might be an effective treatment for blood cancers?

Research has shown that omidubicel, the treatment under study in this trial, is a promising option for serious blood cancers. One study found that patients who received omidubicel recovered their white blood cells faster, which is crucial for fighting infections after a stem cell transplant. Another study found that these patients experienced fewer infections compared to those who received standard treatments. Additionally, long-term data suggest that patients with blood cancers who received omidubicel lived longer. These findings support omidubicel as an effective treatment option for these serious conditions. ^{3 4 6 7 8}

Who Is on the Research Team?

Mitchell Horwitz, MD

Principal Investigator

Duke University

Are You a Good Fit for This Trial?

This trial is for patients aged 12 or older with high-risk blood cancers who have at least one partially HLA-matched cord blood unit available. They must be physically able to undergo a transplant and not pregnant. A backup stem cell source is required, and women of childbearing age must agree to use contraception.

Inclusion Criteria

I am at least 12 years old.

I have a backup source for stem cells.

My body is strong enough to handle treatment.

Exclusion Criteria

My bone marrow is significantly scarred.

I am not a candidate for organ transplant due to my health.

I have antibodies against donor tissues.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive an infusion of omidubicel, a stem/progenitor cell-based product, for transplantation

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after transplantation

12 weeks

Open-label extension

Participants may continue to be monitored for long-term safety and efficacy

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• Omidubicel

Trial Overview The trial is testing omidubicel, an experimental therapy designed to improve outcomes in allogeneic transplantation for individuals with serious hematologic malignancies (blood cancers).

How Is the Trial Designed?

Group I: omidubicel Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Gamida Cell ltd

Lead Sponsor

Trials

9

Recruited

290+

Citations

1. ashpublications.org ashpublications.org/bloodneoplasia/article-split/2/3/100104/536734/Outcomes-of-myeloablative-allogeneic-hematopoietic

Outcomes of myeloablative allogeneic hematopoietic cell ... In a phase 3 randomized trial for high-risk hematologic malignancies, omidubicel had faster neutrophil engraftment and fewer infectious ...

2. astctjournal.org astctjournal.org/article/S2666-6367(25)01138-8/fulltext

Results of a Multicenter Expanded Access Program ... outcomes in patients with hematologic malignancies following transplantation with omidubicel. Between August 2020 and May 2023, 29 patients ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10960759/

Projected Impact of Omidubicel-onlv on Racial/Ethnic ... In a study of 601 consecutive adults with hematologic malignancies (HM) over a 6-year period at Memorial Sloan-Kettering Cancer Center, Europeans were more ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S2950328025000391

Outcomes of myeloablative allogeneic hematopoietic cell ... In an international phase 3 clinical trial, 125 patients with hematologic malignancies were randomized to myeloablative allogeneic HCT using omidubicel or ...

5. targetedonc.com targetedonc.com/view/ten-year-omidubicel-data-improves-survival-in-hematologic-malignancies-with-hsct

Ten-Year Omidubicel Data Improves Survival in ... Ten-Year Omidubicel Data Improves Survival in Hematologic Malignancies ... Outcomes and Preserving Fertility in Premenopausal HR+ Breast Cancer.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11549910/

Omidubicel for Hematopoietic Cell Transplants From a safety perspective, omidubicel has a boxed warning due to the risk of life-threatening infusion reactions, graft-versus-host disease, ...

7. omisirge.com omisirge.com/

OMISIRGE® (omidubicel-onlv) | Allogeneic Hematopoietic ... Omisirge® (omidubicel-onlv) is the first FDA-approved allogeneic progenitor cell therapy for patients aged 12 and up with hematologic malignancies in need ...

8. onclive.com onclive.com/view/fda-approval-insights-omidubicel-in-blood-cancers-requiring-transplant

FDA Approval Insights: Omidubicel in Blood Cancers ... Dr Horwitz discusses the FDA approval of omidubicel in patients with blood cancers, key efficacy and safety data from the pivotal phase 3 trial, ...