Palifermin for Stem Cell Transplant Outcomes
What You Need to Know Before You Apply
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.
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 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.12345
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.678910
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.1251112
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Induction Chemotherapy
Participants receive disease-specific induction chemotherapy (EPOCH-F/R or FLAG) prior to transplant for disease control and immune depletion
Palifermin Administration
Participants receive palifermin at escalating doses in a phase 1, open label design
Conditioning Chemotherapy
Participants receive cyclophosphamide and fludarabine as part of the conditioning regimen
Stem Cell Transplantation
Participants receive a peripheral blood stem cell product from an unrelated donor
Post-Transplant Hospitalization
Participants are hospitalized for monitoring and recovery post-transplant
Follow-up
Participants are monitored for safety and effectiveness after treatment, including tests for GVHD at 60 days and 6 months
What Are the Treatments Tested in This Trial?
Interventions
- Palifermin
Find a Clinic Near You
Who Is Running the Clinical Trial?
National Cancer Institute (NCI)
Lead Sponsor