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

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ClinicalTrials.gov Identifier: NCT00360971
Recruitment Status : Terminated (Due to positive preliminary results from other palifermin studies.)
First Posted : August 7, 2006
Results First Posted : May 15, 2014
Last Update Posted : December 26, 2017
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Radiation Therapy Oncology Group

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Supportive Care
Conditions Head and Neck Cancer
Mucositis
Pain
Radiation Toxicity
Interventions Biological: palifermin
Drug: cisplatin
Other: placebo
Procedure: neck dissection
Radiation: radiation therapy
Enrollment 21
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Placebo Palifermin
Hide Arm/Group Description Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients. Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.
Period Title: Overall Study
Started 10 11
Completed 10 [1] 11 [1]
Not Completed 0 0
[1]
Subjects with data for planned primary analysis are considered to have completed the study.
Arm/Group Title Placebo Palifermin Total
Hide Arm/Group Description Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients. Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients. Total of all reporting groups
Overall Number of Baseline Participants 10 11 21
Hide Baseline Analysis Population Description
All eligible patients.
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 10 participants 11 participants 21 participants
52
(35 to 82)
55
(48 to 67)
55
(35 to 82)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 11 participants 21 participants
Female
0
   0.0%
1
   9.1%
1
   4.8%
Male
10
 100.0%
10
  90.9%
20
  95.2%
1.Primary Outcome
Title Duration of Oral Mucositis as Measured in Terms of Days
Hide Description

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.

Time Frame Twice-weekly from start of treatment up to 15 weeks after the start of treatment.
Hide Outcome Measure Data
Hide Analysis Population Description
All eligible patients.
Arm/Group Title Placebo Palifermin
Hide Arm/Group Description:
Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients.
Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.
Overall Number of Participants Analyzed 10 11
Mean (Standard Deviation)
Unit of Measure: Days
32  (24) 13  (23)
2.Secondary Outcome
Title Number of Patients With Grade 3 or 4 Mucositis as Measured by the World Heath Organization (WHO) Scale
Hide Description 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.
Time Frame Twice-weekly from start of treatment up to 15 weeks after the start of treatment.
Hide Outcome Measure Data
Hide Analysis Population Description
All randomized patients
Arm/Group Title Placebo Palifermin
Hide Arm/Group Description:
Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients.
Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.
Overall Number of Participants Analyzed 10 11
Measure Type: Count of Participants
Unit of Measure: Participants
8
  80.0%
4
  36.4%
3.Secondary Outcome
Title Time to Onset of Grade 3 or 4 Oral Mucositis as Measured by the World Heath Organization (WHO) Scale
Hide Description 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.
Time Frame Twice-weekly from start of treatment up to 15 weeks after the start of treatment.
Hide Outcome Measure Data
Hide Analysis Population Description
All randomized patients
Arm/Group Title Placebo Palifermin
Hide Arm/Group Description:
Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients.
Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.
Overall Number of Participants Analyzed 10 11
Mean (Standard Deviation)
Unit of Measure: days
48  (10) 41  (6)
4.Secondary Outcome
Title Overall Survival
Hide Description 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.
Time Frame From randomization to maximum follow-up at time of analysis of 21 months
Hide Outcome Measure Data
Hide Analysis Population Description
Randomized patients who started protocol treatment
Arm/Group Title Placebo Palifermin
Hide Arm/Group Description:
Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients.
Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.
Overall Number of Participants Analyzed 8 11
Measure Type: Count of Participants
Unit of Measure: Participants
2
  25.0%
0
   0.0%
5.Secondary Outcome
Title Progression-free Survival
Hide Description 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.
Time Frame From randomization to maximum follow-up at time of analysis of 21 months
Hide Outcome Measure Data
Hide Analysis Population Description
Randomized patients who started protocol treatment
Arm/Group Title Placebo Palifermin
Hide Arm/Group Description:
Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients.
Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.
Overall Number of Participants Analyzed 8 11
Measure Type: Count of Participants
Unit of Measure: Participants
2
  25.0%
0
   0.0%
6.Secondary Outcome
Title Time to Second Primary Tumor
Hide Description 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.
Time Frame From randomization to maximum follow-up at time of analysis of 21 months
Hide Outcome Measure Data
Hide Analysis Population Description
Randomized patients who started protocol treatment
Arm/Group Title Placebo Palifermin
Hide Arm/Group Description:
Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients.
Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.
Overall Number of Participants Analyzed 8 11
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
Time Frame [Not Specified]
Adverse Event Reporting Description 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.
 
Arm/Group Title Placebo Palifermin
Hide Arm/Group Description Concurrent radiation therapy, cisplatin, and placebo followed by neck dissection for indicated patients. Concurrent radiation therapy, cisplatin, and palifermin followed by neck dissection for indicated patients.
All-Cause Mortality
Placebo Palifermin
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Placebo Palifermin
Affected / at Risk (%) Affected / at Risk (%)
Total   8/10 (80.00%)   5/11 (45.45%) 
Gastrointestinal disorders     
Mucositis  1  8/10 (80.00%)  4/11 (36.36%) 
Dry mouth * 2  1/10 (10.00%)  0/11 (0.00%) 
Dysphagia * 2  1/10 (10.00%)  1/11 (9.09%) 
Nausea * 2  2/10 (20.00%)  0/11 (0.00%) 
Vomiting NOS * 2  1/10 (10.00%)  0/11 (0.00%) 
General disorders     
Fatigue * 2  0/10 (0.00%)  1/11 (9.09%) 
Infections and infestations     
Infection with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L): Appendix * 2  1/10 (10.00%)  0/11 (0.00%) 
Investigations     
Metabolic/laboratory - Other: * 2  1/10 (10.00%)  0/11 (0.00%) 
Weight decreased * 2  0/10 (0.00%)  3/11 (27.27%) 
Metabolism and nutrition disorders     
Anorexia * 2  2/10 (20.00%)  0/11 (0.00%) 
Dehydration * 2  3/10 (30.00%)  2/11 (18.18%) 
Hypercalcaemia * 2  1/10 (10.00%)  0/11 (0.00%) 
Hyperglycaemia NOS * 2  1/10 (10.00%)  0/11 (0.00%) 
Hypokalemia * 2  1/10 (10.00%)  0/11 (0.00%) 
Hyponatremia * 2  1/10 (10.00%)  0/11 (0.00%) 
Musculoskeletal and connective tissue disorders     
Bone development abnormal * 2  1/10 (10.00%)  0/11 (0.00%) 
Renal and urinary disorders     
Renal failure NOS * 2  1/10 (10.00%)  0/11 (0.00%) 
Renal/genitourinary - Other: * 2  1/10 (10.00%)  0/11 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Acute respiratory distress syndrome * 2  1/10 (10.00%)  0/11 (0.00%) 
Atelectasis * 2  1/10 (10.00%)  0/11 (0.00%) 
Pharyngolaryngeal pain * 2  0/10 (0.00%)  1/11 (9.09%) 
Skin and subcutaneous tissue disorders     
Alopecia * 2  0/10 (0.00%)  1/11 (9.09%) 
Vascular disorders     
Vascular - Other: * 2  1/10 (10.00%)  0/11 (0.00%) 
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, WHO
2
Term from vocabulary, CTCAE (3.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Placebo Palifermin
Affected / at Risk (%) Affected / at Risk (%)
Total   9/10 (90.00%)   11/11 (100.00%) 
Blood and lymphatic system disorders     
Hemoglobin * 1  4/10 (40.00%)  5/11 (45.45%) 
Ear and labyrinth disorders     
Hearing impaired * 1  2/10 (20.00%)  3/11 (27.27%) 
Tinnitus * 1  2/10 (20.00%)  2/11 (18.18%) 
Endocrine disorders     
Endocrine - Other: * 1  1/10 (10.00%)  0/11 (0.00%) 
Gastrointestinal disorders     
Mucositis  2  2/10 (20.00%)  7/11 (63.64%) 
Caecitis * 1  1/10 (10.00%)  0/11 (0.00%) 
Constipation * 1  4/10 (40.00%)  2/11 (18.18%) 
Diarrhoea NOS * 1  2/10 (20.00%)  0/11 (0.00%) 
Dry mouth * 1  7/10 (70.00%)  9/11 (81.82%) 
Dysphagia * 1  3/10 (30.00%)  6/11 (54.55%) 
Esophageal stenosis acquired * 1  0/10 (0.00%)  1/11 (9.09%) 
Gastrointestinal - Other: * 1  0/10 (0.00%)  1/11 (9.09%) 
Ileal stenosis * 1  0/10 (0.00%)  1/11 (9.09%) 
Nausea * 1  2/10 (20.00%)  4/11 (36.36%) 
Oral pain * 1  2/10 (20.00%)  2/11 (18.18%) 
Oseophagitis NOS * 1  2/10 (20.00%)  1/11 (9.09%) 
Salivary gland disorder NOS * 1  1/10 (10.00%)  0/11 (0.00%) 
Toothache * 1  1/10 (10.00%)  0/11 (0.00%) 
Vomiting NOS * 1  1/10 (10.00%)  3/11 (27.27%) 
General disorders     
Edema: head and neck: * 1  3/10 (30.00%)  0/11 (0.00%) 
Fatigue * 1  5/10 (50.00%)  2/11 (18.18%) 
Pyrexia * 1  0/10 (0.00%)  1/11 (9.09%) 
Infections and infestations     
Infection - Other: * 1  2/10 (20.00%)  1/11 (9.09%) 
Infection with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L): Mucosa * 1  1/10 (10.00%)  0/11 (0.00%) 
Infection with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L): Pharynx * 1  1/10 (10.00%)  0/11 (0.00%) 
Infection with normal ANC or Grade 1 or 2 neutrophils: Neck NOS * 1  1/10 (10.00%)  0/11 (0.00%) 
Infection with normal ANC or Grade 1 or 2 neutrophils: Upper aerodigestive NOS * 1  0/10 (0.00%)  1/11 (9.09%) 
Infection with unknown ANC: Oral cavity-gums (gingivitis) * 1  1/10 (10.00%)  0/11 (0.00%) 
Injury, poisoning and procedural complications     
Dermatitis radiation NOS * 1  2/10 (20.00%)  2/11 (18.18%) 
Ecchymosis * 1  1/10 (10.00%)  0/11 (0.00%) 
Radiation recall syndrome * 1  2/10 (20.00%)  2/11 (18.18%) 
Investigations     
Alanine aminotransferase increased * 1  3/10 (30.00%)  3/11 (27.27%) 
Aspartate aminotransferase increased * 1  2/10 (20.00%)  1/11 (9.09%) 
Blood bilirubin increased * 1  1/10 (10.00%)  0/11 (0.00%) 
Blood creatinine increased * 1  2/10 (20.00%)  2/11 (18.18%) 
Coagulopathy * 1  0/10 (0.00%)  1/11 (9.09%) 
Leukopenia NOS * 1  5/10 (50.00%)  3/11 (27.27%) 
Lymphopenia * 1  1/10 (10.00%)  1/11 (9.09%) 
Metabolic/laboratory - Other: * 1  2/10 (20.00%)  2/11 (18.18%) 
Neutrophil count * 1  2/10 (20.00%)  2/11 (18.18%) 
Platelet count decreased * 1  1/10 (10.00%)  0/11 (0.00%) 
Weight decreased * 1  5/10 (50.00%)  7/11 (63.64%) 
Metabolism and nutrition disorders     
Anorexia * 1  4/10 (40.00%)  4/11 (36.36%) 
Dehydration * 1  1/10 (10.00%)  3/11 (27.27%) 
Hyperglycaemia NOS * 1  2/10 (20.00%)  2/11 (18.18%) 
Hyperkalaemia * 1  1/10 (10.00%)  0/11 (0.00%) 
Hyperuricemia * 1  1/10 (10.00%)  1/11 (9.09%) 
Hypoalbuminemia * 1  1/10 (10.00%)  0/11 (0.00%) 
Hypocalcemia * 1  1/10 (10.00%)  0/11 (0.00%) 
Hypokalemia * 1  0/10 (0.00%)  1/11 (9.09%) 
Hypomagnesemia * 1  1/10 (10.00%)  2/11 (18.18%) 
Hyponatremia * 1  1/10 (10.00%)  2/11 (18.18%) 
Musculoskeletal and connective tissue disorders     
Arthralgia * 1  0/10 (0.00%)  1/11 (9.09%) 
Fibrosis-deep connective tissue: * 1  0/10 (0.00%)  1/11 (9.09%) 
Muscle weakness NOS * 1  0/10 (0.00%)  1/11 (9.09%) 
Neck pain * 1  1/10 (10.00%)  0/11 (0.00%) 
Trismus * 1  1/10 (10.00%)  0/11 (0.00%) 
Nervous system disorders     
Dysgeusia * 1  6/10 (60.00%)  5/11 (45.45%) 
Headache * 1  1/10 (10.00%)  0/11 (0.00%) 
Laryngeal nerve dysfunction: * 1  0/10 (0.00%)  1/11 (9.09%) 
Memory impairment * 1  1/10 (10.00%)  0/11 (0.00%) 
Myelitis NOS * 1  1/10 (10.00%)  0/11 (0.00%) 
Neuralgia NOS * 1  1/10 (10.00%)  0/11 (0.00%) 
Neurology - Other: * 1  1/10 (10.00%)  1/11 (9.09%) 
Peripheral sensory neuropathy * 1  1/10 (10.00%)  1/11 (9.09%) 
Tremor * 1  1/10 (10.00%)  0/11 (0.00%) 
Trigeminal nerve disorder NOS * 1  0/10 (0.00%)  1/11 (9.09%) 
Psychiatric disorders     
Agitation * 1  1/10 (10.00%)  0/11 (0.00%) 
Anxiety * 1  0/10 (0.00%)  2/11 (18.18%) 
Confusional state * 1  1/10 (10.00%)  0/11 (0.00%) 
Depression * 1  1/10 (10.00%)  0/11 (0.00%) 
Insomnia * 1  1/10 (10.00%)  0/11 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Aspiration * 1  0/10 (0.00%)  1/11 (9.09%) 
Atelectasis * 1  0/10 (0.00%)  1/11 (9.09%) 
Hiccups * 1  1/10 (10.00%)  0/11 (0.00%) 
Laryngeal edema * 1  1/10 (10.00%)  0/11 (0.00%) 
Laryngitis NOS * 1  2/10 (20.00%)  1/11 (9.09%) 
Pharyngolaryngeal pain * 1  2/10 (20.00%)  3/11 (27.27%) 
Pneumonitis NOS * 1  0/10 (0.00%)  1/11 (9.09%) 
Pulmonary hemorrhage * 1  1/10 (10.00%)  0/11 (0.00%) 
Skin and subcutaneous tissue disorders     
Acne NOS * 1  0/10 (0.00%)  1/11 (9.09%) 
Alopecia * 1  3/10 (30.00%)  1/11 (9.09%) 
Dermatitis exfoliative NOS * 1  2/10 (20.00%)  1/11 (9.09%) 
Pruritus * 1  1/10 (10.00%)  1/11 (9.09%) 
Skin hyperpigmentation * 1  1/10 (10.00%)  0/11 (0.00%) 
Skin hypopigmentation * 1  1/10 (10.00%)  0/11 (0.00%) 
Vascular disorders     
Flushing * 1  0/10 (0.00%)  1/11 (9.09%) 
Thrombosis * 1  0/10 (0.00%)  1/11 (9.09%) 
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (3.0)
2
Term from vocabulary, WHO
This study terminated early with 21 subjects accrued out of 298 planned, therefore no statistical testing was performed. The termination was decided due to positive preliminary results from other palifermin studies.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Name/Title: Wendy Seiferheld
Organization: NRG Oncology
Responsible Party: Radiation Therapy Oncology Group
ClinicalTrials.gov Identifier: NCT00360971     History of Changes
Other Study ID Numbers: RTOG-0435
CDR0000491088
First Submitted: August 3, 2006
First Posted: August 7, 2006
Results First Submitted: April 16, 2014
Results First Posted: May 15, 2014
Last Update Posted: December 26, 2017