Unlicensed Cord Blood Transplant for Blood/Immune System Disorders

Recruiting at 63 trial locations

Overseen By Emeline Masson Frenet

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: New York Blood Center

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

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

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

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

Compensation: Varies

Number of Visits: 1

Duration: 1 week

9999 Participants Needed

Trial Summary

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}

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.

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

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

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

Who Is Running the Clinical Trial?

New York Blood Center

Lead Sponsor

Trials

25

Recruited

28,400+

Findings from Research

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 (UCB) is increasingly recognized as a viable alternative source of hematopoietic stem cells for patients, especially adults lacking HLA-matched donors, with over 6000 successful transplant procedures performed worldwide.

UCB offers several advantages, including easy procurement, low risk of infection transmission, and the ability to tolerate HLA mismatches, making it a promising option for both transplantation and regenerative medicine.

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]

A study analyzing 140 reported adverse events from cord blood infusions in Japan found that hypertension was the most common side effect, while serious adverse events like anaphylaxis occurred at a low frequency of 0.23%.

The research highlighted the need for standardized reporting systems for adverse events in cord blood transplantation, as existing classification criteria were unable to classify a significant number of cases, which could hinder efforts to improve safety and reduce risks.

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]

In 2010, over 1000 transplants used umbilical cord blood as a graft source, highlighting its growing importance in unrelated allogeneic hematopoietic cell transplantation.

The FDA is implementing regulations requiring that all unrelated-donor cord blood units used in transplants must be licensed or covered under an FDA-accepted IND, emphasizing the need for transplant physicians to understand these new guidelines and best practices for selecting suitable cord blood units.

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]

A survey of public cord blood banks worldwide revealed that most are adhering to standard technical procedures for cord blood unit collection and processing, which is crucial for ensuring the quality of units available for stem cell transplantation.

However, the survey identified areas needing improvement, such as better accreditation, increased standardization in testing, and establishing clear thresholds for total nucleated cells in cord blood units to enhance accessibility for therapeutic use.

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]

A study of 364 candidates for umbilical cord blood transplantation in Mexico revealed a wide genetic diversity in HLA haplotypes, which complicates the search for compatible donors, especially for patients of mixed ethnicity.

The most common HLA haplotypes identified included specific combinations of HLA-A, HLA-B, and HLA-DRB1 alleles, highlighting the need for a broader donor search beyond local populations to improve transplantation outcomes.

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]

Unrelated umbilical cord blood (UCB) is a promising alternative for allogeneic hematopoietic cell transplantation, offering benefits like rapid availability and lower risk of severe graft-versus-host disease compared to unrelated donor bone marrow.

Using two unrelated UCB units for transplantation is a safe and effective strategy that addresses the limitation of cell dose in single UCB units, potentially improving patient outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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

Umbilical cord blood transplantation (UCBT) is a safe and effective treatment for patients with hematologic malignancies and genetic disorders, expanding access for non-Caucasian patients who may not find a fully matched donor, as shown in a study of 2456 patients from 2011 to 2016.

The study found that both licensed and unlicensed UCB units provided similar rates of engraftment and survival in children with malignancies, indicating that unlicensed UCB is a viable and safe option for transplantation.

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]