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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00101582
Recruitment Status : Completed
First Posted : January 13, 2005
Results First Posted : September 15, 2014
Last Update Posted : September 27, 2016
Sponsor:
Collaborator:
Amgen
Information provided by (Responsible Party):
Swedish Orphan Biovitrum

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Mucositis
Solid Tumors
Stomatitis
Head and Neck Cancer
Squamous Cell Carcinoma
Interventions Drug: Placebo
Drug: palifermin
Drug: cisplatin chemotherapy
Radiation: Radiotherapy
Enrollment 188
Recruitment Details This study was conducted from 03 August 2005 (first participant enrolled) to 11 September 2007 (last participant's last visit at 4 months of follow-up) at 46 study centers in the United States, Canada, and Europe At the time of this report, the long-term safety follow-up period is ongoing.
Pre-assignment Details  
Arm/Group Title Placebo Palifermin
Hide Arm/Group Description Participants received a single intravenous (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. Chemotherapy consisted of 100 mg/m^2 cisplatin administered by IV infusion on Days 1, 22, and 43. 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. Chemotherapy consisted of 100 mg/m^2 cisplatin administered by IV infusion on Days 1, 22, and 43.
Period Title: Overall Study
Started 94 94
Received Study Drug 91 94
Completed 83 [1] 79 [1]
Not Completed 11 15
Reason Not Completed
Ineligibility determined             0             1
Adverse Event             1             4
Withdrawal by Subject             6             4
Physician Decision             1             0
Death             3             5
Other             0             1
[1]
Participants who completed the acute oral mucositis evaluation phase
Arm/Group Title Placebo Palifermin Total
Hide Arm/Group 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. 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. Total of all reporting groups
Overall Number of Baseline Participants 94 94 188
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 94 participants 94 participants 188 participants
55.4  (8.3) 55.5  (8.6) 55.5  (8.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 94 participants 94 participants 188 participants
Female
14
  14.9%
15
  16.0%
29
  15.4%
Male
80
  85.1%
79
  84.0%
159
  84.6%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 94 participants 94 participants 188 participants
Caucasian 90 83 173
Black 2 7 9
Hispanic 2 3 5
American Indian/ Alaska Native 0 1 1
Weight   [1] 
Mean (Standard Deviation)
Unit of measure:  Kilograms
Number Analyzed 94 participants 94 participants 188 participants
71.8  (15.7) 74.5  (16.4) 73.2  (16.1)
[1]
Measure Description: Data available for 91 and 94 participants in each treatment group respectively.
Disease Stage   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 94 participants 94 participants 188 participants
Stage III 29 26 55
Stage IV A 54 60 114
Stage IV B 11 8 19
[1]
Measure Description: American Joint Committee on Cancer staging system.
Tumor Site  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 94 participants 94 participants 188 participants
Oral Cavity 9 5 14
Oropharynx 51 55 106
Nasopharynx 3 4 7
Larynx 9 16 25
Hypopharynx 22 14 36
1.Primary Outcome
Title Number of Participants With Severe (Grade 3 or 4) Oral Mucositis
Hide Description 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.
Time Frame Up to Week 15
Hide Outcome Measure Data
Hide Analysis Population Description
The Full Analysis Set included all randomized participants.
Arm/Group Title Placebo Palifermin
Hide Arm/Group 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.
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.
Overall Number of Participants Analyzed 94 94
Measure Type: Number
Unit of Measure: participants
Yes 62 51
No 29 43
Unknown 3 0
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Palifermin
Comments Sample size calculations were based on the number of participants needed to detect, with 90% power and 5% type 1 error rate, at least a 25% difference in the incidence of severe OM between the treatment groups. A 25% absolute reduction in the incidence of severe OM from the placebo group was considered by investigators as clinically meaningful in this clinical setting.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0410
Comments Generalized Cochran-Mantel-Haenszel test for general association. Participants having no assessment were assumed as having WHO grade 3 or 4 oral mucositis in hypothesis testing.
Method Cochran-Mantel-Haenszel
Comments Stratified by disease stage (III versus IV) and primary anatomical tumor location (oral cavity / oropharynx vs. nasopharynx vs. hypopharynx / larynx).
Method of Estimation Estimation Parameter Chi-Square Statistic
Estimated Value 4.1764
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Duration of Severe (WHO Grade 3 or 4) Oral Mucositis
Hide Description The duration of severe oral mucositis (OM) was calculated as the number of days from the onset of severe OM (first time a WHO grade 3 or 4 was observed) to the day when severe OM was resolved (first time WHO grade 2 or less was observed after last WHO grade 3 or 4). Durations of 0 days were assigned to those participants who did not experience any WHO grade 3 or 4 during the study.
Time Frame Up to 15 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Palifermin
Hide Arm/Group 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.
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.
Overall Number of Participants Analyzed 94 94
Median (Inter-Quartile Range)
Unit of Measure: days
26.0
(0.0 to 50.0)
5.0
(0.0 to 40.0)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Palifermin
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0160
Comments Generalized Cochran-Mantel-Haenszel test for mean score difference.
Method Cochran-Mantel-Haenszel
Comments Stratified by disease stage (III versus IV) and primary anatomical tumor location (oral cavity / oropharynx vs. nasopharynx vs. hypopharynx / larynx).
Method of Estimation Estimation Parameter Chi-Square Statistic
Estimated Value 5.8002
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Palifermin
Comments To protect the overall type 1 error, the Hochberg procedure was used to adjust for multiple statistical testing of the secondary efficacy endpoints.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1122
Comments Adjusted p-value was based on Hochberg procedure to control for the Type 1 error.
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
3.Secondary Outcome
Title Time to Onset of Severe (WHO Grade 3 or 4) Oral Mucositis
Hide Description

Time to onset of severe (WHO Grade 3 or 4) oral mucositis (OM) was analyzed using the Kaplan-Meier procedure.

Participants without an assessed event by the end of the acute OM evaluation phase were censored at the date of last assessment for severe OM.

Time Frame Up to 15 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Palifermin
Hide Arm/Group 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.
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.
Overall Number of Participants Analyzed 94 94
Median (Inter-Quartile Range)
Unit of Measure: days
35.0 [1] 
(22.0 to NA)
47.0 [1] 
(27.0 to NA)
[1]
Could not be estimated due to the low number of events
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Palifermin
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0261
Comments [Not Specified]
Method Stratified Log-Rank test
Comments Stratified by disease stage (III versus IV) and primary anatomical tumor location (oral cavity / oropharynx vs. nasopharynx vs. hypopharynx / larynx).
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 0.6603
Confidence Interval (2-Sided) 95%
0.4546 to 0.9592
Estimation Comments Hazard ratio of palifermin over placebo based on Stratified Cox proportional hazard model.
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Palifermin
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1566
Comments Adjusted p-value was based on Hochberg procedure to control for the Type 1 error.
Method Stratified Log-Rank test
Comments [Not Specified]
4.Secondary Outcome
Title Number of Participants With Xerostomia at Month 4 (Grade 2 or Higher)
Hide Description The number of participants with grade 2 or higher xerostomia (dryness of the oral mucosa) at the Month 4 visit, graded according to the Common Terminology Criteria for Adverse Events (CTCAE) v3.0 Dry Mouth/Xerostomia scale.
Time Frame Month 4
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set
Arm/Group Title Placebo Palifermin
Hide Arm/Group 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.
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.
Overall Number of Participants Analyzed 94 94
Measure Type: Number
Unit of Measure: participants
Yes 57 37
No 19 31
Unknown 18 26
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Palifermin
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0463
Comments Generalized Cochran-Mantel-Haenszel test for general association. Participants having no assessment were assumed to have the event.
Method Cochran-Mantel-Haenszel
Comments Stratified by disease stage (III versus IV) and primary anatomical tumor location (oral cavity / oropharynx vs. nasopharynx vs. hypopharynx / larynx).
Method of Estimation Estimation Parameter Chi-Square Statistic
Estimated Value 3.9715
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Palifermin
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2314
Comments Adjusted p-value was based on Hochberg procedure to control for the Type 1 error.
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
5.Secondary Outcome
Title Patient-Reported Mouth and Throat Soreness Score
Hide Description

The average patient-reported mouth and throat soreness (MTS) score as reported on question 3 of the Oral Mucositis Weekly Questionnaire for Head and Neck Cancer [OMWQ-HN]): "How much mouth and throat soreness did you experience in the past 24 hours?" Participants answered on a scale from 0 (no soreness) to 4 (extreme soreness).

For each participant, an average patient-reported mouth and throat soreness score was calculated by dividing the sum of the MTS scores at each assessment by the total number of assessments.

Time Frame Assessed twice a week for up to 15 weeks.
Hide Outcome Measure Data
Hide Analysis Population Description

The Patient Reported Outcome-evaluable analysis set included all randomized patients with a valid Baseline assessment for MTS question 3 of the OMWQ-HN and either:

  • At least 1 completed assessment each week for MTS up to withdrawal/Week 8, whichever came first, or
  • 70% or greater overall compliance for MTS until withdrawal/Week 8.
Arm/Group Title Placebo Palifermin
Hide Arm/Group 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.
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.
Overall Number of Participants Analyzed 88 89
Mean (Standard Deviation)
Unit of Measure: units on a scale
1.86  (0.65) 1.66  (0.73)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Palifermin
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0712
Comments Generalized Cochran-Mantel-Haenszel test for mean score difference using modified ridit score
Method Cochran-Mantel-Haenszel
Comments Stratified by disease stage (III versus IV) and primary anatomical tumor location (oral cavity / oropharynx vs. nasopharynx vs. hypopharynx / larynx).
Method of Estimation Estimation Parameter Chi-Square Statistic
Estimated Value 3.2548
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Palifermin
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2849
Comments Adjusted p-value was based on Hochberg procedure to control for the Type 1 error.
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
6.Secondary Outcome
Title Total Dose of Opioid Analgesics Used for Mucositis Within 15 Weeks
Hide Description

The total dose of opioid analgesics (mg of intravenous [IV] morphine equivalents) used by all participants.

Participants with at least one reported administration of opioid analgesic (parenteral, peroral or transdermal) were considered to have received opioid analgesics. The total dose of opioid analgesics is the sum of all opioid analgesic administrations that have been converted to morphine equivalents.

Time Frame Up to 15 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Palifermin
Hide Arm/Group 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.
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.
Overall Number of Participants Analyzed 94 94
Mean (Standard Deviation)
Unit of Measure: mg of IV morphine equivalents
1219.55  (1769.29) 1243.31  (2700.28)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Palifermin
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2384
Comments Generalized Cochran-Mantel-Haenszel test for mean score difference using modified ridit score.
Method Cochran-Mantel-Haenszel
Comments Stratified by disease stage (III versus IV) and primary anatomical tumor location (oral cavity / oropharynx vs. nasopharynx vs. hypopharynx / larynx).
Method of Estimation Estimation Parameter Chi-Square Statistic
Estimated Value 1.3901
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Palifermin
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6835
Comments Adjusted p-value was based on Hochberg procedure to control for the Type 1 error.
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
7.Secondary Outcome
Title Number of Participants With Unplanned Breaks in Cisplatin Chemotherapy Treatment
Hide Description Cisplatin was administered on Days 1, 22, and 43. An unplanned break in cisplatin refers to a delay of ≥ 5 days from the scheduled Day 22 or Day 43 cisplatin administration or a discontinuation of cisplatin for any reason.
Time Frame During the 7 weeks of chemotherapy treatment
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Palifermin
Hide Arm/Group 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.
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.
Overall Number of Participants Analyzed 94 94
Measure Type: Number
Unit of Measure: participants
42 49
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Palifermin
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3417
Comments Generalized Cochran-Mantel-Haenszel test for general association
Method Cochran-Mantel-Haenszel
Comments Stratified by disease stage (III versus IV) and primary anatomical tumor location (oral cavity / oropharynx vs. nasopharynx vs. hypopharynx / larynx).
Method of Estimation Estimation Parameter Chi-Square Statistic
Estimated Value 0.9039
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Palifermin
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6835
Comments Adjusted p-value was based on Hochberg procedure to control for the Type 1 error.
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
8.Secondary Outcome
Title Number of Participants With Unplanned Breaks in Radiotherapy
Hide Description Participants with a duration of 5 days or more without an administration of radiotherapy or who discontinue radiotherapy prior to completion of planned radiotherapy were considered to have an unplanned break in radiotherapy.
Time Frame During the 7 weeks of radiotherapy
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set
Arm/Group Title Placebo Palifermin
Hide Arm/Group 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.
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.
Overall Number of Participants Analyzed 94 94
Measure Type: Number
Unit of Measure: participants
11 13
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Palifermin
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.9587
Comments Generalized Cochran-Mantel-Haenszel test for general association.
Method Cochran-Mantel-Haenszel
Comments Stratified by disease stage (III versus IV) and primary anatomical tumor location (oral cavity / oropharynx vs. nasopharynx vs. hypopharynx / larynx).
Method of Estimation Estimation Parameter Chi-Square Statistic
Estimated Value 0.0027
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Palifermin
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.9587
Comments Adjusted p-value was based on Hochberg procedure to control for the Type 1 error.
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
Time Frame Approximately 8 weeks
Adverse Event Reporting Description The table of Other Adverse Events summarizes non-serious occurrences of adverse events that exceeded a 5% frequency in either treatment arm.
 
Arm/Group Title Placebo Palifermin
Hide Arm/Group Description Participants received a single intravenous (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. 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.
All-Cause Mortality
Placebo Palifermin
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Hide Serious Adverse Events
Placebo Palifermin
Affected / at Risk (%) Affected / at Risk (%)
Total   25/91 (27.47%)   35/94 (37.23%) 
Blood and lymphatic system disorders     
Agranulocytosis  1  0/91 (0.00%)  1/94 (1.06%) 
Anaemia  1  2/91 (2.20%)  0/94 (0.00%) 
Febrile neutropenia  1  2/91 (2.20%)  2/94 (2.13%) 
Haemoglobinaemia  1  0/91 (0.00%)  1/94 (1.06%) 
Leukopenia  1  1/91 (1.10%)  1/94 (1.06%) 
Neutropenia  1  0/91 (0.00%)  1/94 (1.06%) 
Pancytopenia  1  0/91 (0.00%)  1/94 (1.06%) 
Cardiac disorders     
Cardiac failure  1  1/91 (1.10%)  2/94 (2.13%) 
Cardio-respiratory arrest  1  1/91 (1.10%)  0/94 (0.00%) 
Ear and labyrinth disorders     
Hypoacusis  1  1/91 (1.10%)  1/94 (1.06%) 
Gastrointestinal disorders     
Diarrhoea  1  1/91 (1.10%)  0/94 (0.00%) 
Dysphagia  1  2/91 (2.20%)  5/94 (5.32%) 
Nausea  1  1/91 (1.10%)  2/94 (2.13%) 
Odynophagia  1  1/91 (1.10%)  0/94 (0.00%) 
Oral pain  1  0/91 (0.00%)  1/94 (1.06%) 
Pancreatitis necrotising  1  0/91 (0.00%)  1/94 (1.06%) 
Stomatitis  1  2/91 (2.20%)  1/94 (1.06%) 
Vomiting  1  2/91 (2.20%)  3/94 (3.19%) 
General disorders     
Asthenia  1  0/91 (0.00%)  1/94 (1.06%) 
General physical health deterioration  1  0/91 (0.00%)  2/94 (2.13%) 
Mucosal inflammation  1  1/91 (1.10%)  4/94 (4.26%) 
Hepatobiliary disorders     
Hepatic cirrhosis  1  0/91 (0.00%)  1/94 (1.06%) 
Hepatitis  1  1/91 (1.10%)  0/94 (0.00%) 
Portal vein thrombosis  1  0/91 (0.00%)  1/94 (1.06%) 
Immune system disorders     
Hypersensitivity  1  0/91 (0.00%)  1/94 (1.06%) 
Infections and infestations     
Bronchitis  1  1/91 (1.10%)  0/94 (0.00%) 
Bronchitis acute  1  0/91 (0.00%)  1/94 (1.06%) 
Bronchopneumonia  1  0/91 (0.00%)  1/94 (1.06%) 
Candidiasis  1  1/91 (1.10%)  0/94 (0.00%) 
Catheter related infection  1  0/91 (0.00%)  1/94 (1.06%) 
Catheter site infection  1  0/91 (0.00%)  2/94 (2.13%) 
Clostridial infection  1  0/91 (0.00%)  1/94 (1.06%) 
Colitis pseudomembranous  1  1/91 (1.10%)  1/94 (1.06%) 
Infected epidermal cyst  1  0/91 (0.00%)  1/94 (1.06%) 
Laryngitis  1  1/91 (1.10%)  0/94 (0.00%) 
Pharyngitis  1  1/91 (1.10%)  0/94 (0.00%) 
Pneumonia  1  3/91 (3.30%)  2/94 (2.13%) 
Pulmonary sepsis  1  0/91 (0.00%)  1/94 (1.06%) 
Sepsis  1  1/91 (1.10%)  2/94 (2.13%) 
Urinary tract infection  1  1/91 (1.10%)  0/94 (0.00%) 
Injury, poisoning and procedural complications     
Alcohol poisoning  1  1/91 (1.10%)  0/94 (0.00%) 
Device occlusion  1  0/91 (0.00%)  1/94 (1.06%) 
Injury asphyxiation  1  0/91 (0.00%)  1/94 (1.06%) 
Lumbar vertebral fracture  1  0/91 (0.00%)  1/94 (1.06%) 
Post procedural haemorrhage  1  0/91 (0.00%)  1/94 (1.06%) 
Tracheostomy malfunction  1  0/91 (0.00%)  1/94 (1.06%) 
Investigations     
Blood bilirubin abnormal  1  0/91 (0.00%)  1/94 (1.06%) 
Blood creatinine abnormal  1  0/91 (0.00%)  1/94 (1.06%) 
Blood creatinine increased  1  0/91 (0.00%)  2/94 (2.13%) 
Blood lactate dehydrogenase abnormal  1  0/91 (0.00%)  1/94 (1.06%) 
Blood phosphorus decreased  1  0/91 (0.00%)  1/94 (1.06%) 
Blood potassium decreased  1  0/91 (0.00%)  1/94 (1.06%) 
Blood sodium abnormal  1  0/91 (0.00%)  1/94 (1.06%) 
Gamma-glutamyltransferase abnormal  1  0/91 (0.00%)  1/94 (1.06%) 
Hepatic enzyme increased  1  1/91 (1.10%)  0/94 (0.00%) 
Platelet count decreased  1  0/91 (0.00%)  1/94 (1.06%) 
Weight decreased  1  1/91 (1.10%)  5/94 (5.32%) 
White blood cell count decreased  1  0/91 (0.00%)  1/94 (1.06%) 
Metabolism and nutrition disorders     
Dehydration  1  9/91 (9.89%)  4/94 (4.26%) 
Diabetes mellitus  1  0/91 (0.00%)  1/94 (1.06%) 
Food intolerance  1  0/91 (0.00%)  1/94 (1.06%) 
Hyperglycaemia  1  0/91 (0.00%)  1/94 (1.06%) 
Hypoglycaemia  1  0/91 (0.00%)  1/94 (1.06%) 
Hyponatraemia  1  0/91 (0.00%)  1/94 (1.06%) 
Malnutrition  1  3/91 (3.30%)  0/94 (0.00%) 
Musculoskeletal and connective tissue disorders     
Neck pain  1  0/91 (0.00%)  1/94 (1.06%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Laryngeal neoplasm  1  0/91 (0.00%)  1/94 (1.06%) 
Peritoneal carcinoma  1  0/91 (0.00%)  1/94 (1.06%) 
Tumour haemorrhage  1  0/91 (0.00%)  1/94 (1.06%) 
Nervous system disorders     
Convulsion  1  0/91 (0.00%)  1/94 (1.06%) 
Headache  1  0/91 (0.00%)  1/94 (1.06%) 
Syncope  1  2/91 (2.20%)  2/94 (2.13%) 
Syncope vasovagal  1  1/91 (1.10%)  0/94 (0.00%) 
Psychiatric disorders     
Delirium  1  1/91 (1.10%)  0/94 (0.00%) 
Depression suicidal  1  1/91 (1.10%)  0/94 (0.00%) 
Psychotic disorder due to a general medical condition  1  0/91 (0.00%)  1/94 (1.06%) 
Renal and urinary disorders     
Renal failure  1  4/91 (4.40%)  1/94 (1.06%) 
Renal failure acute  1  2/91 (2.20%)  1/94 (1.06%) 
Respiratory, thoracic and mediastinal disorders     
Cryptogenic organizing pneumonia  1  1/91 (1.10%)  0/94 (0.00%) 
Dyspnoea  1  1/91 (1.10%)  2/94 (2.13%) 
Laryngeal oedema  1  2/91 (2.20%)  1/94 (1.06%) 
Pharyngeal inflammation  1  0/91 (0.00%)  1/94 (1.06%) 
Pharyngeal oedema  1  1/91 (1.10%)  0/94 (0.00%) 
Pharyngolaryngeal pain  1  1/91 (1.10%)  0/94 (0.00%) 
Pneumonia aspiration  1  2/91 (2.20%)  1/94 (1.06%) 
Pneumonitis  1  0/91 (0.00%)  1/94 (1.06%) 
Respiratory distress  1  0/91 (0.00%)  1/94 (1.06%) 
Respiratory failure  1  1/91 (1.10%)  0/94 (0.00%) 
Stridor  1  1/91 (1.10%)  0/94 (0.00%) 
Vascular disorders     
Deep vein thrombosis  1  0/91 (0.00%)  1/94 (1.06%) 
Hypertension  1  0/91 (0.00%)  1/94 (1.06%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 9.0
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo Palifermin
Affected / at Risk (%) Affected / at Risk (%)
Total   81/91 (89.01%)   89/94 (94.68%) 
Blood and lymphatic system disorders     
Anaemia  1  32/91 (35.16%)  21/94 (22.34%) 
Leukopenia  1  11/91 (12.09%)  21/94 (22.34%) 
Neutropenia  1  6/91 (6.59%)  10/94 (10.64%) 
Thrombocytopenia  1  6/91 (6.59%)  2/94 (2.13%) 
Ear and labyrinth disorders     
Tinnitus  1  7/91 (7.69%)  11/94 (11.70%) 
Gastrointestinal disorders     
Constipation  1  24/91 (26.37%)  31/94 (32.98%) 
Diarrhoea  1  14/91 (15.38%)  7/94 (7.45%) 
Dry mouth  1  5/91 (5.49%)  9/94 (9.57%) 
Dyspepsia  1  4/91 (4.40%)  5/94 (5.32%) 
Dysphagia  1  19/91 (20.88%)  27/94 (28.72%) 
Nausea  1  42/91 (46.15%)  46/94 (48.94%) 
Odynophagia  1  5/91 (5.49%)  9/94 (9.57%) 
Oesophagitis  1  0/91 (0.00%)  5/94 (5.32%) 
Oral pain  1  3/91 (3.30%)  9/94 (9.57%) 
Vomiting  1  24/91 (26.37%)  24/94 (25.53%) 
General disorders     
Asthenia  1  5/91 (5.49%)  4/94 (4.26%) 
Fatigue  1  20/91 (21.98%)  21/94 (22.34%) 
Malaise  1  1/91 (1.10%)  5/94 (5.32%) 
Oedema peripheral  1  4/91 (4.40%)  6/94 (6.38%) 
Pyrexia  1  19/91 (20.88%)  16/94 (17.02%) 
Infections and infestations     
Candidiasis  1  14/91 (15.38%)  11/94 (11.70%) 
Oral candidiasis  1  11/91 (12.09%)  17/94 (18.09%) 
Oral fungal infection  1  6/91 (6.59%)  5/94 (5.32%) 
Injury, poisoning and procedural complications     
Radiation skin injury  1  13/91 (14.29%)  25/94 (26.60%) 
Investigations     
Blood creatinine increased  1  4/91 (4.40%)  7/94 (7.45%) 
Weight decreased  1  26/91 (28.57%)  25/94 (26.60%) 
Metabolism and nutrition disorders     
Anorexia  1  10/91 (10.99%)  12/94 (12.77%) 
Dehydration  1  12/91 (13.19%)  10/94 (10.64%) 
Hypokalaemia  1  8/91 (8.79%)  19/94 (20.21%) 
Hypomagnesaemia  1  3/91 (3.30%)  5/94 (5.32%) 
Nervous system disorders     
Dizziness  1  5/91 (5.49%)  3/94 (3.19%) 
Dysgeusia  1  7/91 (7.69%)  18/94 (19.15%) 
Headache  1  5/91 (5.49%)  10/94 (10.64%) 
Psychiatric disorders     
Anxiety  1  8/91 (8.79%)  9/94 (9.57%) 
Depression  1  3/91 (3.30%)  5/94 (5.32%) 
Insomnia  1  10/91 (10.99%)  11/94 (11.70%) 
Respiratory, thoracic and mediastinal disorders     
Cough  1  13/91 (14.29%)  16/94 (17.02%) 
Dysphonia  1  8/91 (8.79%)  11/94 (11.70%) 
Dyspnoea  1  5/91 (5.49%)  6/94 (6.38%) 
Pharyngolaryngeal pain  1  22/91 (24.18%)  20/94 (21.28%) 
Skin and subcutaneous tissue disorders     
Alopecia  1  1/91 (1.10%)  6/94 (6.38%) 
Dermatitis  1  10/91 (10.99%)  4/94 (4.26%) 
Rash  1  4/91 (4.40%)  9/94 (9.57%) 
Skin hyperpigmentation  1  1/91 (1.10%)  5/94 (5.32%) 
Vascular disorders     
Flushing  1  0/91 (0.00%)  6/94 (6.38%) 
Hypertension  1  4/91 (4.40%)  5/94 (5.32%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 9.0
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.
The Clinical Trial Agreement generally does not restrict an investigator's discussion of trial results after completion. The Agreement permits sponsor a limited period of time to review material discussing trial results (typically up to 45 days and possible extension). Sponsor may remove confidential information, but authors have final control and approval of publication content. For multi-center studies, investigator agrees not to publish any results before the first multi-center publication.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Hans Olivecrona, MD PhD
Organization: Biovitrum
Phone: +46 8 697 20 00
EMail: hans.olivecrona@sobi.com
Layout table for additonal information
Responsible Party: Swedish Orphan Biovitrum
ClinicalTrials.gov Identifier: NCT00101582    
Obsolete Identifiers: NCT00963456
Other Study ID Numbers: 20020402
First Submitted: January 12, 2005
First Posted: January 13, 2005
Results First Submitted: January 3, 2014
Results First Posted: September 15, 2014
Last Update Posted: September 27, 2016