Unlicensed Cord Blood Transplant for Blood/Immune System Disorders

Recruiting in Palo Alto (17 mi)

+63 other locations

Overseen by Patricia Shi, MD

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Recruiting

Sponsor: New York Blood Center

Disqualifiers: Licensed cord blood, Non-US centers, others

No Placebo Group

Prior Safety Data

Approved in 1 Jurisdiction

Trial Summary

What is the purpose of this trial?

This trial is testing the safety of giving patients special blood from umbilical cords. The study focuses on patients who receive these infusions to see if there are any serious side effects. The cord blood has special cells that can help make new blood cells in the body. Umbilical cord blood has been used in the treatment of various diseases for many years, including leukemia, lymphoma, and congenital immunodeficiency.

Will I have to stop taking my current medications?

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

What data supports the effectiveness of the treatment Unlicensed Cord Blood Units for blood or immune system disorders?

Research shows that umbilical cord blood (UCB) transplantation is a viable alternative for patients needing stem cell transplants, with over 6000 procedures performed worldwide. UCB is noted for its easy availability, low infection risk, and ability to be used even when donor matches are not perfect, making it a promising option for those without matched donors. ^{1 2 3 4 5}

Is unlicensed cord blood transplantation generally safe for humans?

In Japan, cord blood has been used for many stem cell transplants, and while adverse events have been reported, efforts are being made to improve safety reporting and reduce risks. Additionally, over 1000 successful transplants have been performed worldwide, indicating a level of safety, though some patients experienced complications. ^{5 6 7 8 9}

How is the Unlicensed Cord Blood Transplant treatment different from other treatments for blood or immune system disorders?

Unlicensed Cord Blood Transplant uses umbilical cord blood as a source of stem cells, which is unique because it allows for rapid availability and greater tolerance of genetic mismatches compared to traditional bone marrow transplants. This treatment is particularly beneficial for patients who cannot find a fully matched donor, especially those of mixed ethnicity, and it can be used safely and effectively under the FDA's Investigational New Drug program. ^{4 7 10 11 12}

Research Team

Patricia Shi, MD

Principal Investigator

NY Blood Center

Eligibility Criteria

This trial is open to people of any age and gender with disorders affecting the blood-making system, whether inherited, acquired or due to intensive treatment. Participants must receive at least one unlicensed cord blood unit (CBU) made by NCBP. It's not for those treated outside the US, receiving manipulated CBUs post-thaw, or exclusively licensed/unlicensed CBUs from other banks.

Inclusion Criteria

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

My age or gender does not limit my participation.

My cord blood transplant uses a product made by the NCBP.

Exclusion Criteria

I am currently receiving treatment with licensed cord blood products.

I am receiving a cord blood treatment that involves special processing.

Patients who are receiving unlicensed cord blood products from other banks

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive the investigational cord blood units and are monitored for infusion-related reactions

1 week

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including engraftment evaluation

6 months

Treatment Details

Interventions

• Unlicensed CBU (Cord Blood Units)

Trial Overview The study is testing the safety of infusing unlicensed investigational cord blood units (CBUs) manufactured by NCBP. The focus is on documenting all problems related to the infusion process to assess potential risks.

Participant Groups

Group I: unlicensed CBU Experimental Treatment 1 Intervention

The Principal Investigators will be the transplant physicians at participating US transplant centers

Unlicensed CBU is already approved in United States for the following indications:

🇺🇸 Approved in United States as Unlicensed IND Cord Blood Units for:

• Unrelated transplantation

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Nationwide Children's Hospital Columbus, OH

Abramson Cancer Center of the University of Pennsylvania Medical Center Philadelphia, PA

Texas Children's Cancer Center Houston, TX

Children's Hospital of Michigan Detroit, MI

More Trial Locations

Who Is Running the Clinical Trial?

New York Blood Center

Lead Sponsor

Trials

25

Patients Recruited

28,400+

Findings from Research

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]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Umbilical cord blood transplantation: a new alternative option. Tse, W., Laughlin, MJ.[2016]

Increasing the inventory of stored umbilical cord blood units from 50,000 to 100,000 can cost between $44,000 to $86,000 per life-year gained, indicating a significant potential benefit for transplant candidates.

The study suggests that expanding the cord blood inventory is cost-effective, supporting the establishment of a national cord blood bank to improve matching rates for transplant candidates.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Use of cost-effectiveness analysis to determine inventory size for a national cord blood bank. Howard, DH., Meltzer, D., Kollman, C., et al.[2018]

A study of 5,267 umbilical cord blood units (CBUs) identified key characteristics that correlate with higher quality, including shorter processing times, younger gestational ages, and larger collection volumes.

To improve the quality of banked cord blood, it is recommended to prioritize collections from larger babies born before 38 weeks of gestation and ensure rapid transport to processing facilities.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Optimizing donor selection for public cord blood banking: influence of maternal, infant, and collection characteristics on cord blood unit quality. Page, KM., Mendizabal, A., Betz-Stablein, B., et al.[2023]

In a study of 677 cord blood transplants for acute leukemia, the banking techniques used did not significantly affect clinical outcomes such as engraftment and graft-versus-host disease, indicating that current methods are effective.

However, there was variability in cell viability assessments among different banks, highlighting the need for improved standardization in cord blood unit quality controls.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Impact of cord blood banking technologies on clinical outcome: a Eurocord/Cord Blood Committee (CTIWP), European Society for Blood and Marrow Transplantation and NetCord retrospective analysis. Saccardi, R., Tucunduva, L., Ruggeri, A., et al.[2022]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adverse events caused by cord blood infusion in Japan during a 5-year period. Hashimoto, S., Kato, K., Kai, S., et al.[2023]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cord blood unit access and selection: 2010 and beyond: best practices and emerging trends in cord blood unit selection. Boo, M., Ballen, K., Maiers, M.[2018]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Worldwide survey on key indicators for public cord blood banking technologies: By the World Marrow Donor Association Cord Blood Working Group. Jöris, M., Paulson, K., Foley, L., et al.[2023]

Human umbilical cord blood (CB) has been successfully used in over 1000 stem cell transplantations, primarily in children due to limitations in volume and cell numbers, highlighting its efficacy as a treatment option.

In a study of 50 patients receiving unrelated CB transplants, an overall engraftment rate of 83% was observed, with a one-year survival estimate of 42%, indicating the potential effectiveness of CB transplants despite high rates of HLA mismatching.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Haematopoietic transplant potential of unrelated and related cord blood: the first six years of the EUROCORD/NETCORD Bank Germany. Kögler, G., Somville, T., Göbel, U., et al.[2023]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

HLA frequency in candidates to transplant without compatible cord blood at the National Center of Blood Transfusion (Mexico). Rojo-Medina, J., Bello-López, JM.[2018]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Double umbilical cord blood transplantation. Majhail, NS., Brunstein, CG., Wagner, JE.[2007]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Unlicensed Umbilical Cord Blood Units Provide a Safe and Effective Graft Source for a Diverse Population: A Study of 2456 Umbilical Cord Blood Recipients. Ballen, K., Logan, BR., Chitphakdithai, P., et al.[2021]

References

1. United States 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. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Umbilical cord blood transplantation: a new alternative option. [2016]

3. United States pubmed.ncbi.nlm.nih.gov

Use of cost-effectiveness analysis to determine inventory size for a national cord blood bank. [2018]

4. United States pubmed.ncbi.nlm.nih.gov

Optimizing donor selection for public cord blood banking: influence of maternal, infant, and collection characteristics on cord blood unit quality. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

Impact of cord blood banking technologies on clinical outcome: a Eurocord/Cord Blood Committee (CTIWP), European Society for Blood and Marrow Transplantation and NetCord retrospective analysis. [2022]

6. England pubmed.ncbi.nlm.nih.gov

Adverse events caused by cord blood infusion in Japan during a 5-year period. [2023]

7. United States pubmed.ncbi.nlm.nih.gov

Cord blood unit access and selection: 2010 and beyond: best practices and emerging trends in cord blood unit selection. [2018]

8. England pubmed.ncbi.nlm.nih.gov

Worldwide survey on key indicators for public cord blood banking technologies: By the World Marrow Donor Association Cord Blood Working Group. [2023]

9. Germany pubmed.ncbi.nlm.nih.gov

Haematopoietic transplant potential of unrelated and related cord blood: the first six years of the EUROCORD/NETCORD Bank Germany. [2023]

10. England pubmed.ncbi.nlm.nih.gov

HLA frequency in candidates to transplant without compatible cord blood at the National Center of Blood Transfusion (Mexico). [2018]

11. England pubmed.ncbi.nlm.nih.gov

Double umbilical cord blood transplantation. [2007]

12. United States pubmed.ncbi.nlm.nih.gov

Unlicensed Umbilical Cord Blood Units Provide a Safe and Effective Graft Source for a Diverse Population: A Study of 2456 Umbilical Cord Blood Recipients. [2021]