Unlicensed Cord Blood Transplant for Blood/Immune System Disorders

Recruiting at 67 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety of using unlicensed cord blood units (CBU) for treating blood and immune system disorders. Researchers assess the safety of these cord blood infusions and document any issues that arise during the process. The trial targets individuals with blood or immune system problems, whether inherited, developed over time, or caused by treatments like chemotherapy. Participants should receive cord blood from specific sources and not altered or manipulated units. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

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

Is there any evidence suggesting that the unlicensed cord blood units are likely to be safe for humans?

Research has shown that using unlicensed umbilical cord blood units (CBUs) is generally safe for those needing blood or immune system transplants. One study examined 2,456 individuals who received these cord blood units and found them to be a safe option for various patients.

Reports indicate that these cord blood units can be safely used in both children and adults undergoing procedures to replace damaged or diseased bone marrow. While all medical treatments carry some risks, studies have shown that unlicensed cord blood units are well-tolerated by patients.

Overall, the evidence is promising, but discussing any concerns with doctors before joining a clinical trial remains important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for blood and immune system disorders, such as chemotherapy or licensed cord blood units, the investigational therapy involves using unlicensed cord blood units (CBU). This approach is unique because it taps into a wider range of cord blood samples, potentially increasing the availability of matches for patients in need. Researchers are excited about this treatment because it could offer a new avenue for patients who struggle to find suitable donors in the existing registry, potentially improving outcomes and expanding access to life-saving transplants.

What evidence suggests that unlicensed cord blood units might be an effective treatment for blood/immune system disorders?

Studies have shown that unlicensed umbilical cord blood can be a safe and effective transplant option. A large study with 2,456 participants found that these cord blood units work well for both adults and children with blood disorders. They have been successfully used for conditions like blood cancers and genetic disorders affecting blood cells. Research indicates that these cord blood units help rebuild the blood and immune system, offering a potential cure for many. They have proven effective across various patient groups, making them a promising choice for those needing a transplant. Participants in this trial will receive unlicensed cord blood units as part of the study. ^{1 2 5 6 7}

Who Is on the Research Team?

Patricia Shi, MD

Principal Investigator

NY Blood Center

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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 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]

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]

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 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]

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]

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]

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]

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]

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]

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7198366/

Unlicensed umbilical cord blood units provide a safe and ... Unlicensed umbilical cord blood units provide a safe and effective graft source for a diverse population: a study of 2456 umbilical cord blood recipients.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT01656603

Safety Study of Unlicensed IND Cord Blood Units ... This study will evaluate the safety of infusion of the investigational cord blood units by carefully documenting all infusion-related problems. Detailed ...

3. astctjournal.org astctjournal.org/article/S1083-8791(19)30783-9/fulltext

Unlicensed Umbilical Cord Blood Units Provide a Safe and ... Unlicensed umbilical cord blood units can be safely and effectively used for hematopoietic cell transplantation (HCT) in adult and pediatric patients.

4. sciencedirect.com sciencedirect.com/science/article/pii/S1083879118311273

313 Use of Unlicensed Unrelated Umbilical Cord Blood ... Umbilical Cord Blood (UCB) transplant (UCBT) is a curative procedure for patients with hematologic malignancies and genetic disorders, and expands ...

5. withpower.com withpower.com/trial/phase-3-1-2012-8d994

Unlicensed Cord Blood Transplant for Blood/Immune System ... 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.

6. stjude.org stjude.org/care-treatment/clinical-trials/ncbp01-leukemia-blood-disease.html

Safety Study of Unlicensed, Investigational Cord Blood ... The purpose of this study is to evaluate the safety of administration of unlicensed, investigational frozen (cryopreserved) cord blood (CB) units.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT01351545

Study Details | NCT01351545 | A Multicenter Access and ... This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic ...