Palifermin + Leuprolide Acetate for Hematopoietic Stem Cell Transplantation in Blood Cancers

Age: 18 - 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Memorial Sloan Kettering Cancer Center

Disqualifiers: Active infection, Prior transplant, HIV, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 2

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether two treatments, palifermin and leuprolide acetate, can help the immune system recover faster after a stem cell transplant for individuals with certain blood cancers. The goal is to enhance recovery following an allogeneic transplant, which uses donor stem cells to replace unhealthy blood cells. The trial consists of two parts: one group will receive palifermin with Lupron (another name for leuprolide acetate), and another group will receive palifermin with Degarelix. Potential participants include those with certain types of leukemia or lymphoma who are in remission and planning to undergo a specific type of stem cell transplant. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Do I need to stop my current medications for this 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.

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

A previous study found palifermin to be safe, with no impact on blood clotting or liver function. Research also shows that palifermin can reduce the risk and duration of severe mouth sores in patients with blood cancers undergoing stem cell transplants. Although 13% of patients treated with palifermin reported secondary cancers, this was similar to the 11% in the placebo group, indicating no major safety concerns.

Studies have shown that when used with Lupron, palifermin is well-tolerated and may help the immune system recover faster after a stem cell transplant.

When combined with Degarelix, study results revealed no major safety issues.

Overall, these treatments are generally well-tolerated, with manageable side effects, according to previous research. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard treatments for blood cancer stem cell transplants, which mainly focus on chemotherapy and radiation, the combination of palifermin with hormone therapies like Lupron or Degarelix offers a unique approach. Palifermin is particularly exciting because it helps protect and repair mouth and throat tissues damaged by cancer treatments. This approach could lead to fewer side effects like painful mouth sores, making the transplant process more tolerable for patients. Researchers are also keen on the hormone therapies, Lupron and Degarelix, for their potential to modify the body's hormonal environment, possibly enhancing the effectiveness of the transplant and further reducing complications.

What evidence suggests that this trial's treatments could be effective for faster immune recovery after stem cell transplantation?

Research shows that palifermin can help prevent painful mouth sores in patients undergoing stem cell transplants. These sores, known as oral mucositis, are a common and painful side effect of the treatment. In this trial, participants will receive palifermin with either leuprolide acetate (Lupron) or degarelix. Studies suggest that palifermin, when combined with leuprolide acetate, might help the immune system recover more quickly after a transplant. Although research on this combination continues, the goal is to ease recovery for patients. Additionally, palifermin is being tested with degarelix, which may also aid in immune recovery. ^{2 5 6 7 8}

Who Is on the Research Team?

Miguel Angel Perales, MD

Principal Investigator

Memorial Sloan Kettering Cancer Center

Are You a Good Fit for This Trial?

Adults aged 18-60 with certain blood cancers like leukemia, lymphoma, or myelodysplastic syndrome who are in remission but at high risk for relapse. They must be fit enough for a stem cell transplant (with performance status ≥70%) and have good organ function. Pregnant women, recent transplant recipients, those with active infections or HIV are excluded.

Inclusion Criteria

My condition is chronic myelomonocytic leukemia, either CMML-1 or CMML-2.

My leukemia is in its first remission but at high risk of returning, or it's in a second or later remission.

My AML is in its second or later remission.

Exclusion Criteria

I am HIV positive.

Pregnant or breast feeding

Patient is felt to not be a candidate for TBI by the BMT service

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

3-6 weeks

1 visit (in-person)

Pre-transplant Conditioning

Participants receive Lupron or Degarelix and undergo total body irradiation, thiotepa, cyclophosphamide, and anti-thymocyte globulin administration

9 days

Daily visits for treatment administration

Transplantation

Participants undergo T-cell depleted allogeneic hematopoietic stem cell transplant

1 day

1 visit (in-person)

Post-transplant Treatment

Participants receive additional doses of palifermin and Lupron or Degarelix

3 months

Regular follow-up visits

Follow-up

Participants are monitored for immune recovery, overall survival, and incidence of infections

2 years

Regular follow-up visits

What Are the Treatments Tested in This Trial?

Interventions

• Lupron
• Palifermin

Trial Overview The trial is testing if palifermin combined with leuprolide acetate can speed up immune recovery after an allogeneic stem cell transplant following total body irradiation. Participants will receive these drugs along with standard treatments including chemotherapy and radiation.

How Is the Trial Designed?

Group I: palifermin with Lupron Experimental Treatment 6 Interventions

All patients undergo total body irradiation (TBI) on days -9 to -6 \& receive thiotepa intravenously (IV) over 2-4 hours on days -5 to -4, cyclophosphamide IV over 30-60 minutes on days -3 to -2, \& anti-thymocyte globulin infused over 12 hours on days -3 to -2 Pts undergo T-cell depleted allogeneic hematopoietic stem cell transplant on day 0. Pts will receive a three month depot dose of Lupron 3-6 weeks prior to the start date of the pre-transplant conditioning regimen. Pts will receive palifermin at 60mcg/kg/day IV on three consecutive days, 24 hours apart with the last dose administered no less than 24 \& no more than 48 hours prior to the start of cytoreduction. Pts will receive three additional daily doses of palifermin the first approximately 6 hours after the stem cell infusion on day 0, followed by two daily doses given at 24 hour intervals on d+1 \& d+2. Pts will receive a further 3-month depot injection of Lupron approximately 3 months (+/- one week) post the first dose.

Group II: palifermin with Degarelix Experimental Treatment 6 Interventions

Participants on the degarelix arm will receive a loading dose of degarelix 240 mcg subcutaneous 4-14 days before the start of pre-transplant conditioning. All participants will receive palifermin at 60mcg/kg/day IV on three consecutive days, 24 hours apart with the last dose administered no less than 24 and no more than 48 hours prior to the start of cytoreduction.

Who Is Running the Clinical Trial?

Memorial Sloan Kettering Cancer Center

Lead Sponsor

Trials

1,998

Recruited

602,000+

Swedish Orphan Biovitrum

Industry Sponsor

Trials

103

Recruited

13,300+

Dr. Guido Oelkers

Swedish Orphan Biovitrum

Chief Executive Officer since 2017

PhD in Economics

Dr. Lydia Abad-Franch

Swedish Orphan Biovitrum

Chief Medical Officer since 2023

MD, MBA

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT01746849?term=PALIFERMIN&viewType=Table&rank=1

Study Details | NCT01746849 | Palifermin With Leuprolide ... The purpose of this study is to help determine if palifermin and leuprolide acetate can help the immune system recover faster following a stem cell ...

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

The Impact Of Palifermin Use On Hematopoietic Cell ... In one study involving children undergoing autologous hematopoietic cell transplantation (HCT), palifermin was shown to be effective at preventing OM and ...

3. astctjournal.org astctjournal.org/article/S2666-6367(21)01031-9/fulltext

A Prospective Cohort Study Comparing Long-Term ... A prospective cohort study comparing long-term outcomes with and without palifermin in patients receiving hematopoietic cell transplantation for hematologic ...

4. cancer.gov cancer.gov/research/participate/clinical-trials-search/v?id=NCI-2012-02983

Palifermin with Leuprolide Acetate or Degarelix after Total ... This phase II trial studies how well palifermin with leuprolide acetate or degarelix works after total body-irradiation based donor stem cell transplant in ...

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. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/26303102/

Long-Term Safety Outcomes in Patients With ... Secondary malignancies were reported by 13% of palifermin-treated patients versus 11% of placebo patients (P = .477). Breakdown into secondary hematological ...

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

The Impact of Palifermin Use on Hematopoietic Cell ... No difference in rate of post–hematopoietic cell transplantation outcomes among palifermin-treated pediatric patients and matched controls.

8. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/34224914/

A Prospective Cohort Study Comparing Long-Term Outcomes ... The long-term safety of KGF has not been well established, however. In this long-term prospective matched-cohort study, patients who received KGF (cases) and ...