500 Participants Needed

Cord Blood Transplant for Blood Cancers and Diseases

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Overseen ByJennifer S Wilder, R.N.
Age: Any Age
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Approved in 1 JurisdictionThis treatment is already approved in other countries

Trial Summary

What is the purpose of this trial?

Background: - Cord blood transplants can treat cancers and other diseases in children and adults. The U.S. Food and Drug Administration (FDA) requires cord blood to be collected and stored under certain safety standards. However, most available cord blood units were collected before the FDA set these standards. These units may not meet FDA standards, but they do meet similar standards set and followed by the National Marrow Donor Program (NMDP). Cord blood units that do not meet the new FDA standards may be used for transplants only as part of a research study. Doctors want to allow people who need transplants to receive cord blood that meets NMDP standards but may not meet FDA standards. Objectives: - To allow selected cord blood units that do not meet current FDA standards to be used for transplant. Eligibility: - Individuals who need cord blood units for transplant, and who best match cord blood units that are not FDA-licensed. Design: Participants will provide consent to receive cord blood that meets NMDP standards but may not meet FDA standards. Participants will remain on the study for observation for up to 1 year after transplant, or until they withdraw from the study for personal or medical reasons....

Will I have to stop taking my current medications?

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.

What data supports the effectiveness of the treatment Cord Blood Transplant for Blood Cancers and Diseases?

Research shows that omidubicel, a treatment derived from umbilical cord blood, leads to faster recovery of blood cells, fewer infections, and shorter hospital stays compared to standard cord blood transplants. It has also been associated with better long-term survival rates and improved quality of life for patients with blood cancers.12345

Is cord blood transplant generally safe for humans?

Cord blood transplant using omidubicel has shown to be generally safe in humans, with faster recovery and fewer infections compared to standard cord blood transplants. Long-term follow-up studies indicate stable blood cell production and immune function, with low rates of complications like secondary graft failure.13456

How is the cord blood transplant treatment Omidubicel different from other treatments for blood cancers and diseases?

Omidubicel is unique because it is a modified stem cell product derived from umbilical cord blood that has been expanded outside the body to increase the number of stem cells. This results in faster recovery of blood cells and fewer infections compared to traditional cord blood transplants, making it a promising option for patients without a matched donor.13478

Research Team

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Richard W Childs, M.D.

Principal Investigator

National Heart, Lung, and Blood Institute (NHLBI)

Eligibility Criteria

This trial is for pediatric and adult patients of any age with blood cancers or diseases affecting the bone marrow, like Myelodysplastic Syndrome or Aplastic Anemia. Participants must need a cord blood transplant and match with unlicensed cord blood units that meet NMDP standards but not current FDA standards.

Inclusion Criteria

I have a blood disorder that is inherited, acquired, or caused by treatment.
I am of any age.
Signed informed consent (and signed assent, if applicable) obtained prior to study enrollment

Exclusion Criteria

I am only receiving treatments approved by health authorities.
Patients whose selected unlicensed CBU(s) will be more than minimally manipulated
Cord blood transplant recipients at international transplant centers
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Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-transplant Conditioning

Pre-transplant conditioning and GVHD prophylaxis as per each transplant center's specification

2-3 weeks

Transplantation

Participants receive cord blood transplant with unlicensed CBU

1 day

Post-transplant Monitoring

Monitoring for neutrophil recovery, graft rejection, infection transmission, and GVHD

100 days to 1 year

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 year

Treatment Details

Interventions

  • Cord Blood Transplant
Trial OverviewThe study is testing the use of unlicensed preserved cord blood units for transplants in individuals who require them. These cord blood units comply with National Marrow Donor Program standards but may fall short of newer FDA requirements.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: 1Experimental Treatment1 Intervention
cord blood transplant with unlicensed CBU

Cord Blood Transplant is already approved in United States for the following indications:

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Approved in United States as Omisirge for:
  • Hematologic malignancies scheduled for umbilical cord transplantation following myeloablative conditioning

Find a Clinic Near You

Who Is Running the Clinical Trial?

National Heart, Lung, and Blood Institute (NHLBI)

Lead Sponsor

Trials
3,987
Recruited
47,860,000+

Findings from Research

Omidubicel, a modified stem cell graft from cord blood, was approved in April 2023 for treating adults and children aged 12 and older with hematological malignancies, aiming to improve infection rates and speed up neutrophil recovery after transplantation.
The approval of omidubicel marks a significant milestone in its development, highlighting its potential as a treatment option for patients undergoing cord blood transplantation following myeloablative conditioning.
Omidubicel: First Approval.Heo, YA.[2023]
In a phase 3 trial involving 108 patients, omidubicel-onlv, a modified allogeneic hematopoietic progenitor cell therapy, led to faster recovery of blood cell production and a shorter average hospital stay (41.2 days) compared to standard umbilical cord blood (UCB) therapy (50.8 days).
Patients receiving omidubicel-onlv experienced fewer complications, such as a lower rate of ICU admissions (10% vs. 23%) and fewer days spent in intensive care, indicating that this therapy not only improves recovery times but also reduces overall healthcare resource utilization.
Hospitalization and Healthcare Resource Utilization of Omidubicel-Onlv versus Umbilical Cord Blood Transplantation for Hematologic Malignancies: Secondary Analysis from a Pivotal Phase 3 Clinical Trial.Majhail, NS., Miller, B., Dean, R., et al.[2023]
Umbilical cord blood transplantation (CBT) is a crucial alternative for patients without matched donors, but traditional UCB use is limited by lower cell doses leading to delayed engraftment and higher infection risks.
Omidubicel, an ex vivo expanded cord blood product, has received FDA approval for its ability to enhance neutrophil recovery and reduce infection risks, showing promise for improving outcomes in CBT.
A new beginning: can omidubicel emerge as the next, viable alternative donor source?Gandhi, AP., Newell, LF., Maziarz, RT.[2023]

References

Omidubicel: First Approval. [2023]
Hospitalization and Healthcare Resource Utilization of Omidubicel-Onlv versus Umbilical Cord Blood Transplantation for Hematologic Malignancies: Secondary Analysis from a Pivotal Phase 3 Clinical Trial. [2023]
A new beginning: can omidubicel emerge as the next, viable alternative donor source? [2023]
Multicenter Long-Term Follow-Up of Allogeneic Hematopoietic Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials. [2023]
Health-Related Quality of Life Following Allogeneic Hematopoietic Cell Transplantation with Omidubicel versus Umbilical Cord Blood. [2023]
Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study. [2023]
Banking and transplantation of umbilical cord blood in Guangzhou, China. [2008]
Past, present, and future efforts to enhance the efficacy of cord blood hematopoietic cell transplantation. [2020]