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Palifermin

Phase 3

Head and Neck Cancer | Small molecule | Oncology |Amgen Inc.|Last Updated: Mar 14, 2017

Success Probability

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment241

FDA Designations

No designations recorded

Clinical trial landscape

Palifermin · 6 trials · 14 indications

Phase 3 3Phase 2 3
NCT00109031Palifermin for the Reduction of Oral Mucositis in Single-dose Evaluation (PROMISE)Cancer
COMPLETED47 Analytics
NCT00101582Palifermin for the Reduction of Oral Mucositis in Patients With Locally Advanced Head and Neck CancerMucositis
COMPLETED188 Analytics
NCT00131638A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Advanced Head and Neck CancerHead and Neck Cancer
COMPLETED241 Analytics
PHASE3COMPLETED
Palifermin for the Reduction of Oral Mucositis in Single-dose Evaluation (PROMISE)
CancerUnlock trial analytics
PHASE3COMPLETED
Palifermin for the Reduction of Oral Mucositis in Patients With Locally Advanced Head and Neck Cancer
MucositisUnlock trial analytics
PHASE3COMPLETED
A Study of Palifermin for the Reduction of Oral Mucositis in Subjects With Advanced Head and Neck Cancer
Head and Neck CancerUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Severe Oral Mucositis (WHO Grade 3 and 4)
Up to Day 28

Participants underwent evaluations of oral mucosal (OM) surfaces (mucositis assessments) daily during hospitalization and daily thereafter until severe OM returned to grade ≤ 2. A trained evaluator documented the findings using the World Health Organization (WHO) oral toxicity scale according to the following: Grade 0 = None; Grade 1 = Soreness, erythema; Grade 2 = Erythema, ulcers, ability to eat solids; Grade 3 = Ulcers, requires liquid diet; Grade 4 = Alimentation not possible.

Number of Participants With Severe (Grade 3 or 4) Oral Mucositis
Up to Week 15

Participants underwent evaluations of oral mucosal (OM) surfaces (mucositis assessments) 2 times weekly throughout radio/chemotherapy, and 2 times weekly thereafter until severe OM returned to grade ≤ 2 or until Week 15. During each evaluation, the following anatomical areas were assessed: upper lip; lower lip; right cheek; left cheek; right ventral \& lateral tongue; left ventral \& lateral tongue; floor of the mouth; hard palate; soft palate. A trained evaluator documented the findings using the World Health Organization (WHO) oral toxicity scale according to the following: Grade 0 = None; Grade 1 = Soreness, erythema; Grade 2 = Erythema, ulcers, ability to eat solids; Grade 3 = Ulcers, requires liquid diet; Grade 4 = Alimentation not possible.

Incidence percentage of severe oral mucositis (Grades 3 or 4 on the WHO oral mucositis scale)
Up to 24 weeks
Number of Participants With Grade 2 or Higher Dysphagia
Start of treatment through Week 16

Participants underwent acute dysphagia assessments twice weekly during Weeks 1 through 7, and twice weekly thereafter (Weeks 8 through 12) and once weekly after Week 12 until dysphagia resolved to grade ≤ 1 but not beyond Week 16. Dysphagia (difficulty swallowing) was graded using the Common Terminology Criteria for Adverse Events, Version 3.0 (CTCAE v3.0) dysphagia scale according to the following: Grade 1: Symptomatic, able to eat regular diet; Grade 2: Symptomatic and altered eating/swallowing (e.g., altered dietary habits, oral supplements), IV fluids indicated \<24 hours; Grade 3: Symptomatic and severely altered eating/swallowing (e.g., inadequate oral caloric or fluid intake), IV fluids, tube feedings, or total parenteral nutrition (TPN) indicated ≥24 hours; Grade 4: Life-threatening consequences (e.g., obstruction, perforation).

Number of Participants With Severe (Grade 3 and 4) Acute Graft Versus Host Disease (GVHD)
From transplant (Day 0) until Day 100

GVHD was graded using the modified Keystone Criteria weekly during the first 2 months after stem cell infusion, then every other week until Day 100. Severity was determined clinically (based on physical exam and laboratory serum values) and from biopsies of affected organs whenever possible. The degree of GVHD in individual organs was scored by at least 2 assessors. Grade 3 GVHD = total bilirubin 3.1 - 15.0 mg/dL or ≥ 1000 mL/day diarrhea or severe abdominal pain with/without ileus. Grade 4 GVHD = skin involvement with bullous formation or total bilirubin \> 15.0 mg/dL.

Incidence of Grade ≥ 2 (WHO scale) oral mucositis
in Cycle 1 of chemotherapy treatment phase

Secondary Endpoints

Duration of Severe Oral Mucositis (WHO Grade 3 and 4)
Up to Day 28
Area Under the Curve (AUC) of Mouth and Throat Soreness Score
From the first day of study drug administration through Day 28
Number of Participants With Parenteral or Transdermal Opioid Analgesic Use
Up to Day 28
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeSUPPORTIVE_CARE

Treatment Arms

ArmTypeDescription
Palifermin 60 µg/kg for 3 daysACTIVE_COMPARATORPalifermin 60 µg/kg plus placebo to match the total volume equivalent to a 180 µg/kg dose on the 3 days prior to fractionated total body irradiation (fTBI) and palifermin 60 µg/kg on Days 0, 1 and 2 after peripheral blood progenitor cell transplantation (PBPC). Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0.
Palifermin 180 μg/kg on Day -1EXPERIMENTALPalifermin 180 μg/kg on Day -1 and matched placebo on Days -2 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC. Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0.
Palifermin 180 μg/kg on Day -2EXPERIMENTALPalifermin 180 μg/kg on Day -2 and placebo on Days -1 and -3 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC. Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0.
Palifermin 180 μg/kg on Day -3EXPERIMENTALPalifermin 180 μg/kg on Day -3 and placebo on Days -1 and -2 prior to fTBI, and palifermin 60 μg/kg on Days 0, 1, and 2 after PBPC. Participants also received conditioning therapy with fTBI and cyclophosphamide/etoposide prior to PBPC transplantation on Day 0.
PaliferminEXPERIMENTALParticipants received a single intravenous dose of palifermin at 180 μg/kg three days before the start of radiotherapy, and then 7 once weekly palifermin doses at the same dose level during a 7-week radiotherapy/chemotherapy course.
PlaceboPLACEBO_COMPARATORParticipants received a single IV dose of placebo three days before the start of radiotherapy, and then 7 once weekly placebo doses during a 7-week radiotherapy/chemotherapy course.
Control GroupPLACEBO_COMPARATOR50 subjects to receive matched placebo 3 days prior to the first day (day 1) of each cycle of 5-FU/ LV chemotherapy.

Interventions

NameTypeDescription
paliferminDRUGAdministered as one daily intravenous bolus.
Total Body IrradiationRADIATIONTo be delivered before the administration of chemotherapy in 6, 8, or 10 fractions over 3 or 4 days.
CyclophosphamideDRUGCyclophosphamide is administered at a total dose of 100 mg/kg given in 1 dose on Day -2
EtoposideDRUGEtoposide may be administered (optional) as a single intravenous infusion over 4 hours on the day after the last fTBI fraction.
PlaceboDRUGAdministered as one daily intravenous bolus.
cisplatin chemotherapyDRUGCommercially available cisplatin was administered as an intravenous infusion at a dose of 100 mg/m\^2 on Days 1, 22, and 43.
RadiotherapyRADIATIONRadiotherapy was delivered in 200 cGy daily fractions, 5 days a week.
PaclitaxelDRUG -
CarboplatinDRUG -
Conditioning RegimenOTHEREach participant received 1 of the following conditioning regimens: * Cyclophosphamide (Cy) / total body irradiation (TBI) with and without etoposide (VP-16) * TBI/VP-16 * Melphalan (Mel)/TBI (TBI regimens must include fully ablative doses ie \> 1100 cGy; sequence of chemotherapy/radiation (CT/RT) flexible) * Busulfan (Bu)/Cy * Bu/Mel (non-TBI but fully ablative regimens/doses \[Mel dose \> 140 mg/m\^2\]) * Fludarabine (Flu)/Mel (non-TBI but fully ablative regimens/doses \[Mel dose \> 140 mg/m\^2\])
Allogeneic stem cell transplantPROCEDUREAllogeneic marrow/peripheral blood progenitor cell transplantation
MethotrexateDRUG -
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo

Inclusion Criteria: * Written informed consent * Subjects with: non-Hodgkin's lymphoma, Hodgkin's disease, acute myelogenous leukemia, acute lymphoblastic leukemia, chronic myelogenous leukemia, chronic lymphocytic leukemia, or multiple myeloma * Minimum of 1.5 x 10\^6 CD34+ cells/kg cryopreserved ...

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Frequently asked questions about Palifermin

What is Palifermin used for?

Palifermin is an investigational small molecule being studied for the reduction of oral mucositis in patients with head and neck cancer, colon cancer, and graft versus host disease. It is also being evaluated in conditions such as dysphagia and other cancers. Palifermin is developed by Amgen Inc. and is currently in Phase 2 clinical development.

What does Palifermin target?

Palifermin is a small molecule developed by Amgen Inc. that targets pathways involved in mucositis and graft versus host disease. It is being studied for its potential to reduce oral mucositis in patients with head and neck cancer and colon cancer, as well as to reduce acute graft versus host disease in patients with hematologic malignancies.

Who makes Palifermin?

Palifermin is developed by Amgen Inc., a biopharmaceutical company listed on NASDAQ under the ticker symbol AMGN. Amgen is conducting clinical trials to evaluate Palifermin for the reduction of oral mucositis in cancer patients and for the reduction of acute graft versus host disease in patients undergoing allogeneic transplantation.

What phase is Palifermin in?

Palifermin is currently in Phase 2 clinical development. It has completed multiple trials, including Phase 3 studies for oral mucositis in head and neck cancer and Phase 2 studies for graft versus host disease and colon cancer. Palifermin is not yet approved and remains an investigational drug.

What clinical trials is Palifermin in?

Palifermin has been studied in several clinical trials, including NCT00101582 and NCT00131638 for oral mucositis in head and neck cancer, NCT00189488 for acute graft versus host disease, and NCT00393822 for oral mucositis in colon cancer. All trials are completed, with enrollment ranging from 100 to 241 participants.

Is Palifermin the same as Kepivance?

Palifermin is the same as Kepivance, a brand name for the drug. Kepivance is the marketed name for palifermin, which is developed by Amgen Inc. Palifermin is being studied for the reduction of oral mucositis in cancer patients and for graft versus host disease.