Palifermin for Stem Cell Transplant Outcomes

Recruiting at 2 trial locations

Overseen By Ashley E Carpenter

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: National Cancer Institute (NCI)

Disqualifiers: Active infection, CNS malignancy, HIV, others

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

Compensation: Varies

Number of Visits: 6

34 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, since participants will undergo chemotherapy and receive other drugs, it's possible that some medications may need to be adjusted. It's best to discuss your specific medications with the trial team.

What data supports the effectiveness of the drug Palifermin for improving stem cell transplant outcomes?

Palifermin has been shown to significantly reduce the severity of oral mucositis (painful mouth sores) in patients undergoing stem cell transplants, particularly in those with acute lymphoblastic leukemia. It also helps decrease hospital readmissions and the duration of hospital stays for patients with lymphoma undergoing similar treatments. ^{1 2 3 4 5}

Is Palifermin safe for use in humans?

Palifermin is generally considered safe for use in humans, with most adverse effects being mild and temporary, such as skin rash or swelling. It has been used to reduce oral mucositis in patients undergoing stem cell transplants, and studies have shown it does not significantly increase the risk of serious conditions like graft-versus-host disease. ^{1 3 5 6 7}

How does the drug Palifermin differ from other treatments for stem cell transplant outcomes?

Palifermin is unique because it is used as a short 3-day course before high-dose therapy to reduce toxicity and the need for supportive care after autologous blood stem-cell transplantation, particularly in patients with multiple myeloma. This approach is different from other treatments that may not specifically target the reduction of treatment-related toxicity in this way. ^{8 9 10 11 12}

What is the purpose of this trial?

Background: - In allogeneic stem cell transplantation (SCT), stem cells are taken from a donor and given to a recipient. Sometimes the recipient's immune system destroys the donors' cells. Or donor immune cells attack the recipient's tissues, called graft-versus-host disease (GVHD). This is less likely when the recipient and donor have similar human leukocyte antigens (HLA). Researchers want to see if the drug palifermin improves the results of allogeneic SCT from HLA-matched unrelated donors. Objective: - To see if high doses of palifermin before chemotherapy are safe, prevent chronic GVHD, and improve immune function after transplant. Eligibility: - Adults 18 years of age or older with blood or bone marrow cancer with no HLA-matched sibling donor, but with a HLA-matched unrelated donor. Description of Research Study: * Participants will be screened with medical history, physical exam, and blood and urine tests. They will have scans and heart and lung exams. * Before transplant, participants will: * Have many tests and exams. These include blood tests throughout the study and bone marrow biopsy. * Get a central line catheter if they do not have one. * Have 1-3 rounds of chemotherapy. * Have more tests to make sure they can have the transplant, including medical history, physical exam, blood tests, disease specific restaging. * Get palifermin by intravenous (IV) and conditioning chemotherapy to prepare for hematopoietic stem cell transplantation (HSCT). They will get other drugs; some they will take at least 6 months. * Participants will get the HSCT. * After transplant, participants will: * Be hospitalized at least 3-4 weeks. * Monitored at least weekly for the first 100 days. * Stay near District of Columbia (D.C). for approximately 100 days post-transplant. * After 100 days post-transplant - visit National Institutes of Health (NIH) 5 times the first 2 years, then yearly until 5 years post-transplant. * Additional tests/procedures may be performed to monitor safety, response to transplant, side effects.

Research Team

Alain Mina, M.D.

Principal Investigator

National Cancer Institute (NCI)

Eligibility Criteria

Adults over 18 with blood or bone marrow cancer, no HLA-matched sibling donor but a potential unrelated match, must understand and consent to the study. They should have good physical function, agree to use contraception if applicable, and live in the US. Excluded are HIV patients, pregnant women, those with certain psychiatric conditions or active infections not responding to treatment.

Inclusion Criteria

I don't have a family donor but may have a match in a donor registry.

I have been diagnosed with a blood cancer that meets certain criteria.

My acute lymphoblastic leukemia is in its first complete remission.

Exclusion Criteria

You live outside of the United States.

History of psychiatric disorder or any other condition which may compromise compliance with transplant protocol or expose patient to unnecessary risk as determined by principal investigator or lead associate investigator

I have an infection that isn't getting better with treatment.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Induction Chemotherapy

Participants receive disease-specific induction chemotherapy (EPOCH-F/R or FLAG) prior to transplant for disease control and immune depletion

Varies based on chemotherapy regimen

Palifermin Administration

Participants receive palifermin at escalating doses in a phase 1, open label design

1 day (Day -7)

Conditioning Chemotherapy

Participants receive cyclophosphamide and fludarabine as part of the conditioning regimen

4 days (Days -6 to -3)

Stem Cell Transplantation

Participants receive a peripheral blood stem cell product from an unrelated donor

1 day (Day 0)

Post-Transplant Hospitalization

Participants are hospitalized for monitoring and recovery post-transplant

3-4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment, including tests for GVHD at 60 days and 6 months

60 months

5 visits in the first 2 years, then yearly

Treatment Details

Interventions

• Palifermin

Trial Overview The trial is testing high doses of palifermin given before chemotherapy for safety and effectiveness in preventing chronic GVHD and improving immune function after an allogeneic stem cell transplant from HLA-matched unrelated donors.

Participant Groups

Group I: 2/Phase II Arm - Palifermin at the Recommended Phase 2 Dose Experimental Treatment 22 Interventions

Induction chemotherapy, then palifermin at the recommended phase 2 dose determined in Phase 1, then conditioning chemotherapy, then allogeneic stem cell transplant, then immunosuppression.

Group II: 1/Phase 1: Dose Escalation Arm - Palifermin Experimental Treatment 22 Interventions

Induction chemotherapy, then palifermin at escalating doses, then conditioning chemotherapy, then allogeneic stem cell transplant, then immunosuppression.

Who Is Running the Clinical Trial?

National Cancer Institute (NCI)

Lead Sponsor

Trials

14,080

Recruited

41,180,000+

Findings from Research

In a study of 17 patients undergoing high-dose chemotherapy for various cancers, the use of peripheral blood stem cells led to a significantly faster recovery of blood cell counts compared to traditional bone marrow infusion, indicating enhanced efficacy of this treatment approach.

The accelerated recovery resulted in fewer transfusions needed for platelets and red blood cells, reduced fever periods, and earlier hospital discharge, suggesting that peripheral stem cell transplantation could be a safer and more effective alternative to autologous bone marrow transplantation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Favorable effect of hematopoietic stem cells isolated from blood on hematologic recovery following high-dosage chemotherapy]. Richel, DJ., Baars, JW., Wijngaarden, MJ., et al.[2006]

References

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

The Impact of Palifermin Use on Hematopoietic Cell Transplant Outcomes in Children. [2018]

2. Poland pubmed.ncbi.nlm.nih.gov

Efficacy of palifermin on oral mucositis and acute GVHD after hematopoietic stem cell transplantation (HSCT) in hematology malignancy patients: a meta-analysis of trials. [2022]

3. England pubmed.ncbi.nlm.nih.gov

Palifermin for the reduction of acute GVHD: a randomized, double-blind, placebo-controlled trial. [2022]

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

Efficacy and effects of palifermin for the treatment of oral mucositis in patients affected by acute lymphoblastic leukemia. [2022]

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

Effect of Keratinocyte Growth Factor on Hospital Readmission and Regimen-Related Toxicities after Autologous Hematopoietic Cell Transplantation for Lymphoma. [2021]

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

Phase 1/2 randomized, placebo-control trial of palifermin to prevent graft-versus-host disease (GVHD) after allogeneic hematopoietic stem cell transplantation (HSCT). [2022]

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

Palifermin reduces incidence and severity of oral mucositis in allogeneic stem-cell transplant recipients. [2022]

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

A 3-day short course of palifermin before HDT reduces toxicity and need for supportive care after autologous blood stem-cell transplantation in patients with multiple myeloma. [2020]

9. England pubmed.ncbi.nlm.nih.gov

Comparison of marrow vs blood-derived stem cells for autografting in previously untreated multiple myeloma. [2019]

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

Peripheral blood stem cell (PBSC) mobilization and transplantation (PSCT) in patients with malignant lymphomas and solid tumors. [2017]

11. Italy pubmed.ncbi.nlm.nih.gov

Autologous blood stem cell transplantation as intensive consolidation therapy in hematological malignancies. [2004]

12. Netherlands pubmed.ncbi.nlm.nih.gov

[Favorable effect of hematopoietic stem cells isolated from blood on hematologic recovery following high-dosage chemotherapy]. [2006]