Palifermin for Stem Cell Transplant Outcomes

No longer 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether the drug palifermin can enhance the outcome of stem cell transplants from HLA-matched unrelated donors. The main goals are to determine if palifermin is safe, reduces the risk of graft-versus-host disease (where donor cells attack the body), and improves immune function after the transplant. Individuals with blood or bone marrow cancer who lack an HLA-matched sibling donor but have a matched unrelated donor may be suitable candidates. Participants will receive chemotherapy and palifermin, undergo a stem cell transplant, and then be closely monitored. As a Phase 1/Phase 2 trial, this research aims to understand how the treatment works in people and measure its effectiveness in an initial group, offering participants a chance to contribute to groundbreaking medical advancements.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that palifermin is generally safe for patients undergoing hematopoietic stem cell transplantation (HSCT). Studies have found that palifermin reduces the need for nutritional supplements and pain relief after certain types of HSCT, indicating good tolerance. Another study tracked patients for up to 15 years and confirmed palifermin's long-term safety. Additionally, palifermin has been proven to reduce severe oral mucositis, or painful mouth sores, in patients with blood cancers undergoing HSCT. These findings suggest that palifermin can be a safe option for patients, although individual experiences may vary. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about palifermin for stem cell transplant outcomes because it has a unique role in reducing the side effects of chemotherapy. Unlike standard treatments that focus solely on eliminating cancer cells, palifermin is engineered to protect and repair the cells lining the mouth and throat, which are often damaged during the aggressive cancer treatments. This protective mechanism could lead to fewer complications and a quicker recovery for patients undergoing stem cell transplants, making the overall treatment process more tolerable. Additionally, palifermin is being tested at different doses to optimize its effectiveness, which could further enhance patient outcomes.

What evidence suggests that palifermin might be an effective treatment for improving stem cell transplant outcomes?

Research has shown that palifermin, which participants in this trial will receive, can reduce painful mouth sores, known as oral mucositis, in individuals undergoing stem cell transplants from donors. Studies have found that it lessens the severity and frequency of these sores. While its primary benefit is reducing mouth sores, its effectiveness in preventing long-term complications like chronic graft-versus-host disease (GVHD) or boosting the immune system post-transplant remains uncertain. Some research indicates it may not significantly affect transplant outcomes in younger patients, but it is generally considered safe. Palifermin is a lab-made protein that protects and heals the mouth and gut lining during treatment. ^{1 5 6 7 8}

Who Is on the Research Team?

Alain Mina, M.D.

Principal Investigator

National Cancer Institute (NCI)

Are You a Good Fit for This Trial?

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

Patients must agree to use adequate contraception if of child-bearing potential

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.

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4054935/

Palifermin is efficacious in recipients of TBI-based but not ... Abstract. Palifermin, a recombinant human keratinocyte growth factor, is commonly given to prevent mucositis following autologous transplantation.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5798422/

Efficacy of palifermin on oral mucositis and acute GVHD ... Palifermin reduces incidence and severity of oral mucositis in allogeneic stem-cell transplant recipients. Bone Marrow Transplant. 2008;42:275–9. doi ...

3. astctjournal.org astctjournal.org/article/S1083-8791(16)30027-1/fulltext

The Impact of Palifermin Use on Hematopoietic Cell ... Palifermin reduces incidence and severity of oral mucositis in allogeneic stem-cell transplant recipients. Bone Marrow Transplant. 2008; 42:275-279. Crossref.

4. ar.iiarjournals.org ar.iiarjournals.org/content/40/11/6531

Impact of Palifermin on Transplant Outcomes in Children ... Conclusion: Palifermin had no impact on transplant outcomes in children and adolescents undergoing allo-HCT. Hematopoietic cell transplantation · mucositis ...

5. jenci.springeropen.com jenci.springeropen.com/articles/10.1186/s43046-024-00247-x

Efficacy of palifermin in the treatment of oral mucositis in non ... This study demonstrated that palifermin utilization is safe and well tolerated and has some effects on non-hematopoietic stem cell transplant ...

6. sciencedirect.com sciencedirect.com/science/article/pii/S1083879115005479

Long-Term Safety Outcomes in Patients with ... We evaluated long-term safety outcomes (up to 15 years) of palifermin in hematopoietic stem cell transplantation patients.

7. astctjournal.org astctjournal.org/article/S1083-8791(16)00140-3/fulltext

Safety, Pharmacokinetics, and Efficacy of Palifermin in ... Palifermin has been proven to decrease the incidence and duration of severe OM in adults with hematologic malignancies undergoing hematopoietic stem cell ...

8. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4949099/

The Impact Of Palifermin Use On Hematopoietic Cell ... We compared hematopoietic cell transplant (HCT) outcomes among pediatric patients from a large database who did or did not receive palifermin as a preventive ...