Umbilical Cord Blood Transplant for Blood Cancers

Recruiting in Palo Alto (17 mi)

Overseen by John Wagner, MD

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Academic

Recruiting

Sponsor: Masonic Cancer Center, University of Minnesota

Disqualifiers: Specified in treatment protocol

No Placebo Group

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

For the next 5-10 years or possibly longer, a high proportion of the Cord Blood Banks (CBB) inventory worldwide will be composed of unlicensed umbilical cord blood (UCB) units. While Food and Drug Administration (FDA)-licensed units will be prioritized, it will always be possible that an unlicensed unit will have known attributes, making it a better source of cells for the given indication. Because of the wide variety of current and potential indications as a source of cells for hematopoietic reconstitution or other form of cellular therapy, it is critical that the investigators have access to unlicensed UCB units.

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.

What data supports the effectiveness of the treatment for umbilical cord blood transplantation in blood cancers?

Umbilical cord blood (UCB) has been shown to be an effective source of stem cells for transplantation in patients with blood cancers, providing similar survival outcomes to other stem cell sources, especially in children. UCB is advantageous due to its availability and successful outcomes even with some donor-recipient mismatches, although improvements are needed for better recovery and reduced early transplant-related deaths.

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Is umbilical cord blood transplantation generally safe for humans?

Umbilical cord blood transplantation is considered safe and has a low incidence of chronic graft-versus-host disease (a condition where the donor's cells attack the recipient's body). It has been used successfully in adults with leukemia, although the low cell dose in a single unit can be a limitation.

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How is umbilical cord blood transplantation different from other treatments for blood cancers?

Umbilical cord blood transplantation is unique because it uses stem cells from umbilical cord blood, which can be a valuable option for patients without a matched donor. This treatment can involve using two units of cord blood to overcome the limitation of low cell numbers, potentially improving outcomes for patients with blood cancers.

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Eligibility Criteria

This trial is for patients needing a stem cell transplant to treat blood or lymphatic diseases and who have an unlicensed umbilical cord blood (UCB) unit that matches their needs. The UCB must be free of pathogens, with no equivalent licensed option available. Participants must consent to use the UCB as part of another approved treatment plan.

Inclusion Criteria

I (or my guardian) have agreed to use an experimental treatment as part of a clinical trial.

I am eligible for a treatment that requires stem cells from umbilical cord blood after a bone marrow preparation procedure.

I have a cord blood unit that matches and is free of blood-borne diseases, not from a licensed bank.

Exclusion Criteria

Exclusion criteria are specified in the treatment protocol according to indication.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-infusion Treatment

Participants receive intravenous hydration, acetaminophen, and diphenhydramine hydrochloride prior to UCB infusion

Approximately 30 minutes

Infusion

Infusion of minimally manipulated unlicensed UCB units with vital signs monitoring and management of infusion reactions

Within 24 hours

Post-transplant Care and Follow-up

Participants are monitored for safety and effectiveness, including incidence of serious infusion reactions and blood borne pathogen transmission

Up to 180 days

Participant Groups

The study tests the infusion of unlicensed UCB units in treating various conditions requiring hematopoietic stem cells from cord blood after intensive chemotherapy or radiation therapy, known as myeloablative or non-myeloablative conditioning.

Group I: Unlicensed Umbilical Cord Blood Infusion Experimental Treatment 1 Intervention

All patients will be registered in OnCore under this protocol as well as the specific treatment protocol. * Pre-infusion treatment using intravenous hydration, acetaminophen and diphenhydramine hydrochloride * Unlicensed Umbilical Cord Blood Infusion according to institutional guidelines. * Infusion of minimally manipulated unlicensed UCB units: * vital signs Monitoring during and after UCB infusion: * Management of infusion reactions * Post-transplant care and follow-up: will be done according to the disease specific treatment protocol and institutional guidelines.

Infusion of Cell Populations From Unlicensed Umbilical Cord Blood Units is already approved in United States, European Union for the following indications:

🇺🇸 Approved in United States as Umbilical Cord Blood Transplantation for:

• Hematopoietic reconstitution for inherited, acquired, or myeloablative treatment-related disorders
• Leukemia
• Hemoglobinopathies
• Fanconi’s anemia
• Immune or metabolic disorders

🇪🇺 Approved in European Union as Umbilical Cord Blood Transplantation for:

• Hematopoietic reconstitution for malignant and non-malignant disorders
• Acute leukemia
• Hemoglobinopathies
• Fanconi’s anemia
• Immune or metabolic disorders

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Masonic Cancer Center, University of Minnesota Minneapolis, MN

Who Is Running the Clinical Trial?

Masonic Cancer Center, University of Minnesota Lead Sponsor

References

1. England pubmed.ncbi.nlm.nih.gov

Expanded umbilical cord blood T cells used as donor lymphocyte infusions after umbilical cord blood transplantation. [2017] Umbilical cord blood (UCB) is an alternative graft source for hematopoietic stem cell transplantation and has been shown to give results comparable to transplantation with other stem cell sources. Donor lymphocyte infusion (DLI) is an effective treatment for relapsed malignancies after hematopoietic stem cell transplantation. However, DLI is not available after UCB transplantation.

2. England pubmed.ncbi.nlm.nih.gov

A cross-sectional study of umbilical cord blood donor profiles and their influence on umbilical cord blood collection in a Brazilian hospital. [2018] Umbilical cord blood (UCB) cells are a new alternative to bone marrow source for hematopoietic stem cell transplantation and their use has increased in the last decade. Thus efforts are being made to improve the umbilical cord blood unit's quality. Besides compatibility, other factors, such as the total nucleated cell (TNC) count and the percentage of CD34(+) cells in the product, are very important for a successful transplant outcome. Our aim was to describe our donor population and assess the best cord blood collection technique at Hospital Israelita Albert Einstein's cord blood bank (São Paulo, Brazil).

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

Awareness of cord blood collection and the impact on banking. [2017] Umbilical cord blood (UCB) is an important source of hematopoietic stem cells for transplantation especially in minority populations with limited chances of finding a histocompatible volunteer donor in the registry. UCB has the advantages of early availability, successful outcomes despite some histocompatibility mismatch, and low incidence of chronic graft-versus-host disease. Public cord blood banks that disseminate UCB products for transplant depend on voluntary donation at participating hospitals and obstetrical providers for collection.

4. Netherlands pubmed.ncbi.nlm.nih.gov

Enhanced engraftment of umbilical cord blood-derived stem cells in NOD/SCID mice by cotransplantation of a second unrelated cord blood unit. [2006] Umbilical cord blood (UCB) is considered as an attractive alternative source of hematopoietic stem cells for allogeneic stem cell transplantations in patients who lack human leukocyte antigen (HLA)-matched donors. However, the low cell dose adversely affects hematopoietic recovery and therefore limits application of UCB transplantation in adults. Transplantation of multiple UCB units could be a strategy to overcome cell dose limitations.

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

Transplant outcomes in acute leukemia. I. [2021] Umbilical cord blood (UCB) has gradually emerged over the last decade as an alternative source of hematopoietic cells for transplantation in children and adults with high-risk or advanced hematologic malignancies who do not have a suitably matched related or unrelated adult donor. This increase in use of UCB is due to favorable results in children, growing availability of UCB units with large cell doses, less stringent donor-recipient HLA matching, and rapid identification and acquisition of the unit. In children with acute leukemia, the data support similar leukemia-free survival after transplantation of human leukocyte antigen (HLA)-matched and one or two HLA-mismatched UCB and HLA-matched unrelated donor bone marrow. In adults with acute leukemia, some reports suggest a survival advantage after transplantation of matched unrelated bone marrow compared to UCB, while others report similar leukemia-free survival. Work is in progress to improve hematopoietic recovery and lower early transplant-related deaths, the two major limitations to a successful outcome after UCB transplant. The importance of HLA-matching and cell dose on outcomes after UCB transplantation support the need for an even greater investment in public cord blood banks. Simultaneously searching of accredited cord blood banks and bone marrow donor registries for patients without an HLA-matched sibling thought to benefit from hematopoietic stem cell transplantation (HSCT) is encouraged.

6. China pubmed.ncbi.nlm.nih.gov

Long-term outcomes in adults with leukemia treated with transplantation of two unrelated umbilical cord blood units. [2011] Wide application of umbilical cord blood transplantation (UCBT) in adult patients is limited by low cell-dose available in one umbilical cord blood (UCB) unit. The aim of this study was to investigate the safety and long-term outcomes of UCBT from unrelated donors in adult and adolescent patients with leukemia.

7. England pubmed.ncbi.nlm.nih.gov

Banking and transplantation of umbilical cord blood in Guangzhou, China. [2008] Umbilical cord blood (UCB) is an alternative source of hematopoietic stem cells (HSC) for transplantation of patients with hematologic malignancies or hereditary diseases.

8. Germany pubmed.ncbi.nlm.nih.gov

Double umbilical cord blood transplantation for children and adolescents. [2021] Umbilical cord blood transplantation (UCBT) with two units has been conducted with promising results in adults to overcome the limitation of low cell numbers. In an attempt to improve the outcomes, double UCBT was performed in children and adolescents. Sixty-one patients, including 44 acute leukemia, and 17 other hematologic diseases, received double UCBT. Donor-type engraftment achieved in 82% of patients. Except one patient with persistent mixed chimerism of two units, other 49 patients showed dominancy of one unit and only the CFU-GM was significant factor influencing dominancy. The event-free survival (EFS) of leukemia and other hematologic disease were 59% and 53%, respectively, and the EFS of acute leukemia patients who received transplant in first or second CR (68.6%) was significantly better than in those with advanced disease (22.2%) (P = 0.007). Among the factors influencing outcomes, low cell dose difference between two units (TNC difference/TNC of large unit

9. Denmark pubmed.ncbi.nlm.nih.gov

Safe and effective administration of BCG for bladder carcinoma in situ after umbilical cord blood stem cell transplantation. [2018] Although intravesical Bacillus Calmette-Guérin (BCG) instillation is the standard treatment for carcinoma in situ of the bladder, it is generally contraindicated in immunocompromised patients. Here we report the first case, to our knowledge, of BCG treatment for a bladder cancer patient who had received umbilical cord blood stem cell transplantation (UCBSCT). BCG can be given safely and effectively in select cases where reconstitution of the immune system has been achieved at least 2 years after UCBSCT.

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

Post transplant cyclophosphamide based haplo-identical transplant versus umbilical cord blood transplant; a meta-analysis. [2020] Both haplo-identical transplant (haplo) and umbilical cord transplant (UC) are valuable graft options for patients without available matched relative. Previous studies showed inconsistent outcomes comparing Post transplant Cyclophosphamide based haplo (PTCy-haplo) and UC; therefore, we attempt to compare the studies by mean of meta-analysis.

11. Switzerland pubmed.ncbi.nlm.nih.gov

Prognostic role and HER2 expression of circulating tumor cells in peripheral blood of patients prior to radical cystectomy: a prospective study. [2022] Preliminary research has suggested the potential prognostic value of circulating tumor cells (CTC) in patients with advanced nonmetastatic urothelial carcinoma of the bladder (UCB).

12. Iran pubmed.ncbi.nlm.nih.gov

Intracavitary Chemotherapy after kidney-Sparing Therapy for Upper Tract Urothelial Carcinoma: A Meta-Analysis. [2023] Intracavitary chemotherapy is one of the current treatment options for kidney-sparing treatment of upper tract urothelial carcinoma (UTUC). The purpose of this meta-analysis was to assess the efficacy and safety of intracavitary perfusion.