Palifermin for the Reduction of Oral Mucositis in Patients With Locally Advanced Head and Neck Cancer

Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Weekly Doses of Palifermin (rHuKGF) for the Reduction of Oral Mucositis in Subjects With Advanced Head and Neck Cancer Receiving Radiotherapy With Concurrent Chemotherapy (RT/CT)

Sponsors

Lead Sponsor: Swedish Orphan Biovitrum

Collaborator: Amgen

Source Swedish Orphan Biovitrum
Brief Summary

The purpose of this research study is to test the safety and effectiveness of palifermin to determine if weekly doses can be safely administered to reduce the incidence (occurrence of), duration (length of time) and severity (amount of pain) of oral mucositis (painful sores in the mouth). Mucositis is a common side effect for patients receiving chemotherapy (cancer-killing drug) and radiotherapy (cancer-killing x-rays) for the treatment of head and neck cancer (HNC).

Detailed Description

This study consisted of 2 phases. The acute oral mucositis (OM) evaluation phase includes the time from randomization to the time of severe OM (WHO Grade 3 or 4) resolution (up to Week 12 or up to Week 15 for participants whose severe OM is not resolved at Week 12). In the acute OM evaluation phase, participants were randomized to receive either a single IV dose of palifermin or placebo at 180 μg/kg, 3 days before the start of radiotherapy, plus 7 once-weekly palifermin or placebo doses at the same dose level during a 7-week radio/chemotherapy course. In the long-term follow up phase, participants are followed until death, withdrawal of consent, or loss to follow-up. The long-term follow up phase is still ongoing.

Overall Status Completed
Start Date August 2005
Completion Date August 2016
Primary Completion Date September 2007
Phase Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Number of Participants With Severe (Grade 3 or 4) Oral Mucositis Up to Week 15
Secondary Outcome
Measure Time Frame
Duration of Severe (WHO Grade 3 or 4) Oral Mucositis Up to 15 weeks
Time to Onset of Severe (WHO Grade 3 or 4) Oral Mucositis Up to 15 weeks
Number of Participants With Xerostomia at Month 4 (Grade 2 or Higher) Month 4
Patient-Reported Mouth and Throat Soreness Score Assessed twice a week for up to 15 weeks.
Total Dose of Opioid Analgesics Used for Mucositis Within 15 Weeks Up to 15 weeks
Number of Participants With Unplanned Breaks in Cisplatin Chemotherapy Treatment During the 7 weeks of chemotherapy treatment
Number of Participants With Unplanned Breaks in Radiotherapy During the 7 weeks of radiotherapy
Enrollment 188
Condition
Intervention

Intervention Type: Drug

Intervention Name: Placebo

Arm Group Label: Placebo

Intervention Type: Drug

Intervention Name: palifermin

Arm Group Label: Palifermin

Intervention Type: Drug

Intervention Name: cisplatin chemotherapy

Description: Commercially available cisplatin was administered as an intravenous infusion at a dose of 100 mg/m^2 on Days 1, 22, and 43.

Intervention Type: Radiation

Intervention Name: Radiotherapy

Description: Radiotherapy was delivered in 200 cGy daily fractions, 5 days a week.

Eligibility

Criteria:

Key Inclusion Criteria:

- Histologically documented squamous cell carcinoma involving either the oral cavity, oropharynx, nasopharynx, hypopharynx, or larynx

- Newly diagnosed, locally advanced stage head and neck cancer (unresectable/unresected disease); American Joint Committee on Cancer [AJCC] Stage III, IVA or IVB amenable to radiotherapy with concurrent chemotherapy as the definitive treatment modality

- At least 50 Gray of radiation treatment to areas of the oral cavity/oropharynx mucosa that can be visualized

- Concurrent chemotherapy regimen of Cisplatin 100mg/m^2 on days 1, 22, and 43

- Eastern Cooperative Oncology Group (ECOG) performance status (PS) less than or equal to 2

- Adequate hematologic, renal and hepatic function

- Negative pregnancy test by serum or urine

- Signed informed consent

Key Exclusion Criteria:

- Presence or history of any other primary malignancy (other than curatively treated in situ cervical cancer, or basal cell carcinoma of the skin without evidence of disease for greater than 3 years)

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
MD Study Director Amgen
Verification Date

August 2016

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Palifermin

Type: Experimental

Description: Participants 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.

Label: Placebo

Type: Placebo Comparator

Description: Participants 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.

Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Source: ClinicalTrials.gov