Stem Cell Transplantation with NiCord® for Blood Cancers

No longer recruiting at 55 trial locations

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Gamida Cell ltd

Disqualifiers: Other malignancy, Infection, CNS disease, 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new stem cell treatment called NiCord® (Omidubicel) for individuals with certain blood cancers, such as leukemia and lymphoma, who require a stem cell transplant. The goal is to determine if NiCord® is more effective than standard cord blood transplants. Participants will receive either NiCord® or the standard treatment. Eligible candidates have specific blood cancers and lack a matching stem cell donor. As a Phase 3 trial, this study represents the final step before FDA approval, providing participants an opportunity to contribute to potentially groundbreaking treatment advancements.

Do I have to stop taking my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that NiCord® (also called Omidubicel) demonstrated promising safety results in earlier studies. In these trials, most patients tolerated the treatment well. Some side effects occurred, which is common with any stem cell transplant.

Importantly, the FDA has approved NiCord® for use in blood cancer patients requiring a transplant. This approval indicates that its safety is well understood and that it has met strict safety and effectiveness standards in past studies.

Overall, while some risks exist as with any medical treatment, evidence supports that NiCord® is a safe option for patients undergoing stem cell transplantation. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for blood cancers?

Unlike the standard treatments for blood cancers, which often include chemotherapy and traditional stem cell transplants, NiCord® (omidubicel) offers a unique approach. NiCord® is derived from expanded umbilical cord blood stem cells, specifically using ex vivo expanded CD34+ progenitor cells. This method potentially enhances the effectiveness of stem cell transplants by increasing the number of available cells, which can lead to faster engraftment and recovery. Researchers are excited about NiCord® because it represents a significant advancement in stem cell therapy, offering the promise of improved outcomes for patients who may not have suitable donors for conventional transplants.

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

Research shows that NiCord® (Omidubicel), one of the treatments studied in this trial, delivers promising results for patients with blood cancers like acute myeloid leukemia and acute lymphoblastic leukemia. Studies have found that Omidubicel can lead to successful and long-lasting stem cell transplants. It has proven to be safe and effective, aiding blood cell recovery faster than traditional methods. Specifically, Omidubicel has improved transplant success and patient survival rates. These findings suggest that NiCord® could be a valuable treatment option for individuals undergoing stem cell transplants for certain blood cancers. Participants in this trial may receive NiCord® or an unmanipulated cord blood unit as part of the study's treatment arms. ^{1 2 4 6 7}

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 with certain blood cancers like leukemia or lymphoma who need a stem cell transplant and don't have a fully matched donor. They should be in good enough health to undergo the procedure, not pregnant, without active infections or other cancers, and have a back-up stem cell source.

Inclusion Criteria

I have a backup source for stem cells.

My condition meets the specific disease criteria for the trial.

Signed written informed consent

Exclusion Criteria

I have had a stem cell transplant from a donor.

Pregnancy or lactation

I have a donor who is a perfect match for me.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Conditioning Therapy

Participants undergo conditioning therapy to prepare for transplantation

1-2 weeks

Transplantation

Participants receive either NiCord® or unmanipulated cord blood unit transplantation

1 day

1 visit (in-person)

Post-transplant Monitoring

Participants are monitored for neutrophil and platelet engraftment and infections

100 days

Follow-up

Participants are monitored for safety and effectiveness after transplantation

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Cord Blood Unit
• NiCord® (Omidubicel)

Trial Overview The study compares NiCord® (omidubicel), an advanced cord blood unit, with standard unmanipulated cord blood units for stem cell transplantation in patients with specific types of leukemia and lymphoma.

How Is the Trial Designed?

Group I: NiCord® (omidubicel) Experimental Treatment 1 Intervention

NiCord® is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (NiCord® cultured fraction (CF)) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) (NiCord® Non-cultured Fraction (NF)) consisting of mature myeloid and lymphoid cells. Both fractions, i.e. NiCord® CF and NiCord® NF, will be kept frozen until they are thawed and infused on the day of transplantation.

Group II: Unmanipulated CBU(s) Active Control 1 Intervention

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 ... Eligible cancer diagnoses included acute myeloid leukemia (AML), acute lymphoblastic leukemia, and other leukemias, excluding AML with t(8;21), ...

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

Outcomes of myeloablative allogeneic hematopoietic cell ... Eligible cancer diagnoses included acute myeloid leukemia (AML), acute lymphoblastic leukemia, and other leukemias, excluding AML with t(8;21), inv(16), or ...

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

Omidubicel for Hematopoietic Cell Transplants This pooled analysis provides long-term outcomes that support the safe, effective, and durable use of omidubicel transplantation. ... Cancer ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT02730299

NCT02730299 | Stem Cell Transplantation With NiCord® ... Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in ... Loyola University, Cardinal Bernardin Cancer Center. Kansas Locations.

5. healthtree.org healthtree.org/mds/community/articles/omidubicel-stem-cell-transplants-approval-mds

Omidubicel - FDA Approval For Stem Cell Transplant Request a data registry. Access the largest and most complete dataset available in blood cancers.

6. dukecancerinstitute.org dukecancerinstitute.org/blogs/dci-led-clinical-trial-leads-fda-approval-novel-cord-blood-therapy

DCI-Led Clinical Trial Leads to FDA Approval of Novel ... The Blood Cancer Center at DCI has already treated five patients with an omidubicel on a “compassionate use” basis. FDA approval means that many ...

7. onclive.com onclive.com/view/omidubicel-approval-represents-key-advance-for-patients-with-blood-cancers-requiring-transplant

Omidubicel Approval Represents Key Advance for Patients ... Omidubicel Approval Represents Key Advance for Patients With Blood Cancers Requiring Transplant ... ESMO 2025 Breast Cancer Data May ...