Trial Outcomes & Findings for Palifermin in Lessening Oral Mucositis in Patients Undergoing Radiation Therapy and Chemotherapy for Locally Advanced Head and Neck Cancer (NCT NCT00360971)

Last Updated: 2017-12-26

Results Overview

Duration in days of World Heath Organization (WHO) Grades 3 and 4 oral mucositis during the acute period (defined to be 105 days \[15 weeks\] or less from the start of treatment); duration is calculated from the onset of a Grade 3 or 4 oral mucositis to the day when an oral mucositis of ≤ Grade 2 is reported after the last oral mucositis of Grade 3 or 4. Patients with grade 0-2 mucositis have a duration of 0. This study required 298 patients to detect via two-sided t-test a reduction of mean duration of at least 9 days from 29 days (standard deviation = 23 days) on the placebo arm with 90% power and alpha = 0.05. Statistical testing was not done due to the small sample size.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

21 participants

Primary outcome timeframe

Twice-weekly from start of treatment up to 15 weeks after the start of treatment.

Results posted on

2017-12-26

Participant Flow

Participant milestones

Participant milestones
Measure	Placebo Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients.	Palifermin Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.
Overall Study STARTED	10	11
Overall Study COMPLETED	10	11
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Palifermin in Lessening Oral Mucositis in Patients Undergoing Radiation Therapy and Chemotherapy for Locally Advanced Head and Neck Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo n=10 Participants Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients.	Palifermin n=11 Participants Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.	Total n=21 Participants Total of all reporting groups
Age, Continuous	52 years n=5 Participants	55 years n=7 Participants	55 years n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants	10 Participants n=7 Participants	20 Participants n=5 Participants

PRIMARY outcome

Timeframe: Twice-weekly from start of treatment up to 15 weeks after the start of treatment.

Population: All eligible patients.

Outcome measures

Outcome measures
Measure	Placebo n=10 Participants Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients.	Palifermin n=11 Participants Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.
Duration of Oral Mucositis as Measured in Terms of Days	32 Days Standard Deviation 24	13 Days Standard Deviation 23

SECONDARY outcome

Timeframe: Twice-weekly from start of treatment up to 15 weeks after the start of treatment.

Population: All randomized patients

Adverse events are graded using CTCAE v3.0. Grade refers to the severity of the AE. The CTCAE v3.0 assigns Grades 1 through 5 with unique clinical descriptions of severity for each AE based on this general guideline: Grade 1 Mild AE, Grade 2 Moderate AE, Grade 3 Severe AE, Grade 4 Life-threatening or disabling AE, Grade 5 Death related to AE.

Outcome measures

Outcome measures
Measure	Placebo n=10 Participants Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients.	Palifermin n=11 Participants Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.
Number of Patients With Grade 3 or 4 Mucositis as Measured by the World Heath Organization (WHO) Scale	8 Participants	4 Participants

SECONDARY outcome

Timeframe: Twice-weekly from start of treatment up to 15 weeks after the start of treatment.

Population: All randomized patients

Outcome measures

Outcome measures
Measure	Placebo n=10 Participants Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients.	Palifermin n=11 Participants Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.
Time to Onset of Grade 3 or 4 Oral Mucositis as Measured by the World Heath Organization (WHO) Scale	48 days Standard Deviation 10	41 days Standard Deviation 6

SECONDARY outcome

Timeframe: From randomization to maximum follow-up at time of analysis of 21 months

Population: Randomized patients who started protocol treatment

An event is death from any cause. Overall survival was not calculated due to the limited number of events. Number of patients with an event is reported.

Outcome measures

Outcome measures
Measure	Placebo n=8 Participants Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients.	Palifermin n=11 Participants Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.
Overall Survival	2 Participants	0 Participants

SECONDARY outcome

Timeframe: From randomization to maximum follow-up at time of analysis of 21 months

Population: Randomized patients who started protocol treatment

An event is defined as the first occurrence of local, regional, distant disease. Progression-free survival is calculated at the time from registration to the death of progression, death in the absence of progression, or last follow-up. Progression-free survival was not calculated due to the limited number of events. Number of patients with an event is reported.

Outcome measures

Outcome measures
Measure	Placebo n=8 Participants Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients.	Palifermin n=11 Participants Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.
Progression-free Survival	2 Participants	0 Participants

SECONDARY outcome

Timeframe: From randomization to maximum follow-up at time of analysis of 21 months

Population: Randomized patients who started protocol treatment

An event is occurrence of a second primary other than basal cell. Time to second primary tumor was not calculated because there were no events. Number of patients with an event is reported.

Outcome measures

Outcome measures
Measure	Placebo n=8 Participants Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients.	Palifermin n=11 Participants Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.
Time to Second Primary Tumor	0 Participants	0 Participants

Adverse Events

Placebo

Serious events: 8 serious events

Other events: 9 other events

Deaths: 0 deaths

Palifermin

Serious events: 5 serious events

Other events: 11 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Wendy Seiferheld

NRG Oncology

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee PI's are required to abide by the sponsor's publication guidelines which require review by coauthors and subsequent review and approval by the sponsor.
Publication restrictions are in place

Restriction type: OTHER

Measure	Placebo n=10 participants at risk Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients.	Palifermin n=11 participants at risk Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.
Gastrointestinal disorders Mucositis	80.0% 8/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	36.4% 4/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Gastrointestinal disorders Dry mouth	10.0% 1/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	0.00% 0/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Gastrointestinal disorders Dysphagia	10.0% 1/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	9.1% 1/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Gastrointestinal disorders Nausea	20.0% 2/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	0.00% 0/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Gastrointestinal disorders Vomiting NOS	10.0% 1/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	0.00% 0/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
General disorders Fatigue	0.00% 0/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	9.1% 1/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Infections and infestations Infection with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L): Appendix	10.0% 1/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	0.00% 0/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Investigations Metabolic/laboratory - Other:	10.0% 1/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	0.00% 0/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Investigations Weight decreased	0.00% 0/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	27.3% 3/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Metabolism and nutrition disorders Anorexia	20.0% 2/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	0.00% 0/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Metabolism and nutrition disorders Dehydration	30.0% 3/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	18.2% 2/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Metabolism and nutrition disorders Hypercalcaemia	10.0% 1/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	0.00% 0/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Metabolism and nutrition disorders Hyperglycaemia NOS	10.0% 1/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	0.00% 0/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Metabolism and nutrition disorders Hypokalemia	10.0% 1/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	0.00% 0/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Metabolism and nutrition disorders Hyponatremia	10.0% 1/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	0.00% 0/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Musculoskeletal and connective tissue disorders Bone development abnormal	10.0% 1/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	0.00% 0/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Renal and urinary disorders Renal failure NOS	10.0% 1/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	0.00% 0/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Renal and urinary disorders Renal/genitourinary - Other:	10.0% 1/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	0.00% 0/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Respiratory, thoracic and mediastinal disorders Acute respiratory distress syndrome	10.0% 1/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	0.00% 0/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Respiratory, thoracic and mediastinal disorders Atelectasis	10.0% 1/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	0.00% 0/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Respiratory, thoracic and mediastinal disorders Pharyngolaryngeal pain	0.00% 0/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	9.1% 1/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Skin and subcutaneous tissue disorders Alopecia	0.00% 0/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	9.1% 1/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.
Vascular disorders Vascular - Other:	10.0% 1/10 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.	0.00% 0/11 Subjects experiencing more than one of a given SAE (serious adverse event) are counted only once for that SAE. The same methodology was applied for non-SAE adverse events (AE). Mucositis was collected separately from other AE's using the World Health Organization (WHO) grading criteria.