Cord Blood Units for Blood Cancers

Recruiting at 139 trial locations

Overseen By Elizabeth Fosberg, B.A.

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Center for International Blood and Marrow Transplant Research

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

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

Compensation: Varies

Number of Visits: Unknown

99999 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests unlicensed cryopreserved cord blood units (CBUs) to determine their effectiveness in treating blood-related diseases. These diseases may be inherited, acquired, or result from intense treatments that impact blood formation. The trial includes both children and adults who have not found relief with other licensed CBUs. Participants will receive these CBUs to assess any improvement in their condition. This trial may suit those with serious blood disorders. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to benefit from potentially effective treatment.

Do I need to stop my current medications to join the trial?

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 prior data suggests that these unlicensed cryopreserved cord blood units are safe for pediatric and adult patients with hematologic malignancies?

Research has shown that unlicensed frozen cord blood units (CBUs) have undergone safety testing in several studies. These studies carefully track any problems during treatment to assess patient tolerance. Although specific data from these studies is not provided here, the emphasis on safety ensures that any side effects or issues receive close monitoring. Since these CBUs are not fully approved, ongoing efforts aim to ensure their safety for individuals with blood-related conditions. In summary, while the treatment remains under study, a clear process exists to monitor safety and manage potential risks. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about using unlicensed cryopreserved cord blood units (CBUs) because they offer a unique approach for patients needing stem cell transplants. Unlike traditional bone marrow or peripheral blood stem cell transplants, CBUs are more readily available and can be stored until needed, which means patients can potentially receive transplants more quickly. Additionally, CBUs don't have to match the recipient as closely as other sources, providing more options for people who struggle to find a perfect match. This could make lifesaving transplants accessible to a broader range of patients, offering new hope for those with blood disorders or certain cancers.

What evidence suggests that unlicensed cryopreserved cord blood units are effective for hematologic malignancies?

Research has shown that using stored cord blood, which remains unlicensed, is safe and effective for treating blood-related diseases. In this trial, participants will receive unlicensed cryopreserved cord blood units (CBUs) for specific indications. Studies have found that these CBUs can be successfully used for transplants in both children and adults. Specifically, a large study with 2,456 patients reported positive results, demonstrating that these units are a reliable source for blood cell transplants. This treatment is particularly beneficial for patients with leukemia and similar conditions. Overall, cord blood units provide a valuable option for those needing blood-forming cell transplants. ^{1 6 7 8 9}

Who Is on the Research Team?

Heather Stefanski, MD, PhD

Principal Investigator

NMDP/CIBMTR

Are You a Good Fit for This Trial?

Inclusion Criteria

Disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment

Signed informed consent (and signed assent, if applicable) obtained prior to study enrollment

Pediatric and adult patients of any age

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Transplantation

Participants receive unlicensed cryopreserved cord blood units (CBUs) for transplantation

Immediate

Engraftment Monitoring

Monitoring of neutrophil recovery and other engraftment parameters post-transplant

8 weeks

Follow-up

Participants are monitored for safety, infection transmission, infusion reactions, and GVHD

1 year

What Are the Treatments Tested in This Trial?

Interventions

• Unlicensed Cryopreserved Cord Blood Units

How Is the Trial Designed?

Group I: Unlicensed CBU Experimental Treatment 1 Intervention

The cohort includes recipients of any age receiving unlicensed cryopreserved cord blood units (CBUs) for designated indications.

Who Is Running the Clinical Trial?

Center for International Blood and Marrow Transplant Research

Lead Sponsor

Trials

40

Recruited

200,190,000+

National Marrow Donor Program

Collaborator

Trials

63

Recruited

202,000+

Citations

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

Unlicensed umbilical cord blood units provide a safe and ... Use of unlicensed UCB units is safe, effective and provides an important graft source for a diverse population. Keywords: Cord Blood Transplantation, Leukemia, ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT01861093

Safety Study of Unlicensed IND Cord Blood Units ... The primary aim of this study is to examine the safety of administration of the unlicensed investigational NCBP hematopoietic progenitor cell-cord blood ...

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

Unlicensed Umbilical Cord Blood Units Provide a Safe and ... This report, one of the largest of its kind, examines outcomes for 2456 patients of diverse race/ethnicity (50% children and 36% of adults were non-Caucasian) ...

4. cibmtr.org cibmtr.org/Files/RCI-BMT/10-CBA-Study/10-CBA-Protocol_v10.5_v25Jul2025_02Oct2025.pdf

Protocol 10-CBA Unlicensed Cryopreserved Cord Blood Units (CBUs) for. Transplantation in Pediatric and Adult Patients with. Hematologic Malignancies and Other Indications.

5. sciencedirect.com sciencedirect.com/science/article/pii/S1083879119307839

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.

6. fredhutch.org fredhutch.org/en/research/clinical-trials/trial-details.fh_trial_id_9021.a-multicenter-safety-study-of-unlicensed-investigational-cryopreserved-cord-blood-units-cbu-s-manufactured-by-the-national-cord-blood-program-ncbp.html

Clinical Trial Details A Multicenter Safety Study of Unlicensed, Investigational Cryopreserved Cord Blood Units (CBU's) Manufactured by the National Cord Blood Program ...

7. research-studies.allinforhealth.info research-studies.allinforhealth.info/us/en/listing/824/a-multicenter-safety-study/

A multicenter safety study of unlicensed investigational ... The purpose of this study is to evaluate the safety of administration of the investigational cord blood units by carefully documenting all ...

8. jcto.weill.cornell.edu jcto.weill.cornell.edu/open_clinical_trials/a-multicenter-safety-study-of-unlicensed-investigational-cryopreserved-cord-blood-units-cbus-manufactured-by-the-national-cord-blood-program-ncbp-and-provided-for-unrelated-hematopoietic-stem-cell-transplantation-of-pediatric-and

A multicenter safety study of unlicensed, investigational ... A multicenter safety study of unlicensed, investigational cryopreserved cord blood units (CBUs) manufactured by the National Cord Blood Program (NCBP) and ...

9. studypages.com studypages.com/s/a-multicenter-access-and-distribution-protocol-for-unlicensed-cryopreserved-cord-blood-units-cbus-248923/

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