Cord Blood Transplant for Blood Cancers and Diseases

Overseen By Jennifer S Wilder, R.N.

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

Disqualifiers: Licensed CBUs, International centers, Other IND protocol, others

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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

500 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the use of cord blood transplants for people with blood cancers and other diseases. The main goal is to determine if cord blood units meeting National Marrow Donor Program safety standards can be safely used, even if they don't meet newer FDA standards. This trial may suit individuals needing a cord blood transplant who match with these specific units. Researchers will monitor participants for up to one year after their transplant. As a Phase 1 and Phase 2 trial, this research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.

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.

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

Research has shown that umbilical cord blood transplants have been safely used for many years. Studies indicate that about 49.5% of people survive two years after a cord blood transplant, a promising result that compares well to other transplant options.

Cord blood is unique because it contains many young cells that are less developed than those in adult blood, aiding recovery after a transplant. Although the cord blood units in this trial do not meet the latest FDA standards, they do meet the standards of the National Marrow Donor Program, which are similar.

Using cord blood is generally well-tolerated and has successfully treated blood cancers and diseases for decades. While no treatment is without risks, the long history of using cord blood supports confidence in its safety. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatment?

Cord blood transplant is unique because it uses stem cells from umbilical cord blood to treat blood cancers and diseases. Unlike traditional bone marrow transplants, which require a close match between donor and recipient, cord blood transplants can be performed with less stringent matching, potentially widening the donor pool. Researchers are excited about this treatment because cord blood contains a higher concentration of hematopoietic stem cells, which may lead to faster recovery and a reduced risk of complications. Additionally, cord blood is readily available and can be stored for future use, offering a more accessible option for patients in need.

What evidence suggests that cord blood transplant is effective for blood cancers and diseases?

Research has shown that cord blood transplants, which participants in this trial will receive, can effectively treat blood cancers. Studies have found that cord blood from unrelated donors can cure many children with leukemia. Rich in stem cells, cord blood serves as the body's building blocks for producing blood cells. These stem cells can replace damaged ones and help rebuild a healthy immune system. Survival rates for patients receiving cord blood transplants often match those of other transplant methods, making cord blood a valuable option for those needing a transplant. ^{2 4 6 7 8}

Who Is on the Research Team?

Richard W Childs, M.D.

Principal Investigator

National Heart, Lung, and Blood Institute (NHLBI)

Are You a Good Fit for This Trial?

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 am of any age.

I have a blood disorder that is inherited, acquired, or caused by treatment.

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

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

• Cord Blood Transplant

Trial Overview

The 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.

How Is the Trial Designed?

Group I: 1 Experimental Treatment 1 Intervention

cord blood transplant with unlicensed CBU

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

Approved in United States as Omisirge for:

• Hematologic malignancies scheduled for umbilical cord transplantation following myeloablative conditioning

Who Is Running the Clinical Trial?

National Heart, Lung, and Blood Institute (NHLBI)

Lead Sponsor

Trials

3,987

Recruited

47,860,000+

Published Research Related to This Trial

Omidubicel, an ex vivo-expanded umbilical cord blood therapy, shows promising long-term outcomes with a 3-year overall survival rate of 62.5% and disease-free survival rate of 54.0% in 105 patients with hematologic malignancies or sickle cell disease, based on a pooled analysis of 5 multicenter trials.

The therapy demonstrated stable blood cell production and immune function over a median follow-up of 22 months, with normal immune cell counts maintained for up to 8 years, indicating its potential for durable engraftment and safety in long-term use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Multicenter Long-Term Follow-Up of Allogeneic Hematopoietic Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials. Lin, C., Schwarzbach, A., Sanz, J., et al.[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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A new beginning: can omidubicel emerge as the next, viable alternative donor source? Gandhi, AP., Newell, LF., Maziarz, RT.[2023]

Cord blood (CB) has been successfully used in over 35,000 clinical transplants for various disorders, demonstrating its viability as a source of hematopoietic stem cells (HSCs) and progenitor cells compared to bone marrow and mobilized peripheral blood.

Recent advancements aim to enhance the efficacy of CB transplants by increasing HSC numbers, expanding functional HSCs outside the body, and improving their ability to engraft, highlighting the need for further clinical trials to test these innovative approaches.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Past, present, and future efforts to enhance the efficacy of cord blood hematopoietic cell transplantation. Huang, X., Guo, B., Capitano, M., et al.[2020]

A total of 8623 umbilical cord blood (UCB) units were collected in a provincial bank in Guangzhou, China, with 4147 units (48.1%) stored for potential transplantation, demonstrating effective donor screening and processing protocols.

The study reported high cell viability (97.8%) and successful engraftment times for transplanted patients, with children showing faster neutrophil engraftment (17 days) compared to adults (20 days), indicating the efficacy of UCB as a source of hematopoietic stem cells for transplantation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Banking and transplantation of umbilical cord blood in Guangzhou, China. Wu, JY., Liao, C., Xu, ZP., et al.[2008]

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Omidubicel: First Approval. Heo, YA.[2023]

In a phase 3 trial involving 125 patients aged 13 to 65 with hematologic malignancies, omidubicel led to significantly faster neutrophil engraftment (12 days) compared to standard umbilical cord blood transplantation (22 days), indicating improved efficacy.

Patients receiving omidubicel also experienced quicker platelet recovery, fewer severe infections, and spent more time out of the hospital in the first 100 days post-transplant, suggesting it may reduce early transplant-related complications.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Omidubicel vs standard myeloablative umbilical cord blood transplantation: results of a phase 3 randomized study. Horwitz, ME., Stiff, PJ., Cutler, C., et al.[2023]

In a study involving 108 patients undergoing allogeneic hematopoietic cell transplantation, omidubicel demonstrated significant improvements in health-related quality of life (HRQL) compared to standard umbilical cord blood transplantation, with better scores in physical and functional well-being observed as early as 42 days post-transplant.

The benefits of omidubicel on HRQL were not only statistically significant but also clinically meaningful, with improvements persisting for up to one year, indicating its potential for enhancing patient well-being after transplantation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Health-Related Quality of Life Following Allogeneic Hematopoietic Cell Transplantation with Omidubicel versus Umbilical Cord Blood. Lin, C., Sajeev, G., Stiff, PJ., et al.[2023]

Citations

1.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC2581998/

Results of the Cord Blood Transplantation Study (COBLT)

Unrelated donor cord blood transplantation from partially HLA-mismatched units can cure many children with leukemias.

2.

ashpublications.org

ashpublications.org/blood/article/90/12/4665/236483/Placental-and-or-Umbilical-Cord-Blood-An

Placental and/or Umbilical Cord Blood: An Alternative Source ...

Both term and preterm umbilical cord blood contains a significantly higher number of early and committed progenitor cells when compared with adult peripheral ...

3.

sciencedirect.com

sciencedirect.com/science/article/abs/pii/S0188440924000948

Umbilical Cord Blood Hematopoietic Cells: From Biology to ...

Umbilical cord blood (UCB) is a rich source of hematopoietic stem and progenitor cells that are biologically superior to their adult counterparts.

4.

nejm.org

nejm.org/doi/full/10.1056/NEJMoa1405584

One-Unit versus Two-Unit Cord-Blood Transplantation for ...

We found that among children and adolescents with hematologic cancer, survival rates were similar after single-unit and double-unit cord-blood transplantation.

5.

beonbrand.getbynder.com

beonbrand.getbynder.com/m/ac65b09fe645d887/original/Placental-and-Umbilical-Cord-Blood-as-a-Source-of-Stem-Cells.pdf

Placental-and-Umbilical-Cord-Blood-as-a-Source-of-Stem- ...

Mo (2016) reported on outcomes after umbilical cord blood and haploidentical hematopoietic cell transplantation (haplo-HCT) in 129 children ...

6.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC4250219/

Concise Review: Umbilical Cord Blood Transplantation

The probability of 2-year survival was 49.5% [13], that faired favorably compared with previous reports. A larger study from the Center for International Blood ...

7.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC7567024/

Umbilical Cord Blood Transplants: Current Status and ...

Umbilical cord blood (UCB) stem cells have been successfully used for hematopoietic cell transplant (HCT) since the first report in 1989 (1).

8.

provider.healthybluemo.com

provider.healthybluemo.com/medpolicies/healthybluemo/active/mp_pw_a045960.html

TRANS.00016 Umbilical Cord Blood Progenitor Cell Collection, ...

This document addresses umbilical cord blood banking, which is a process of collecting hematopoietic progenitor stem cells from the umbilical cord and ...