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Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas

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ClinicalTrials.gov Identifier: NCT00076102
Recruitment Status : Completed
First Posted : January 14, 2004
Results First Posted : July 12, 2012
Last Update Posted : April 23, 2018
Sponsor:
Information provided by (Responsible Party):
Brigitte Widemann, M.D., National Cancer Institute (NCI)

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Neurofibromatosis 1
Neurofibroma, Plexiform
Intervention Drug: Pirfenidone
Enrollment 36

Recruitment Details 36 participants were enrolled in this study
Pre-assignment Details  
Arm/Group Title Pirfenidone
Hide Arm/Group Description Pirfenidone orally as capsules three times a day approximately every 8 hours for cycles of 28 days with no rest period between cycles (28 day treatment cycles); 500 mg/m^2 every 8 hours (1500 mg/m2/day).
Period Title: Overall Study
Started 36
Completed 31
Not Completed 5
Reason Not Completed
Clinical progression             1
Progression in pre-existing brain tumor             1
plexiform neurofibroma surgery             1
refusal of further therapy             2
Arm/Group Title Pirfenidone
Hide Arm/Group Description Pirfenidone orally as capsules three times a day approximately every 8 hours for cycles of 28 days with no rest period between cycles (28 day treatment cycles); 500 mg/m^2 every 8 hours (1500 mg/m2/day).
Overall Number of Baseline Participants 36
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 36 participants
<=18 years
34
  94.4%
Between 18 and 65 years
2
   5.6%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 36 participants
9.81  (4.52)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 36 participants
Female
10
  27.8%
Male
26
  72.2%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 36 participants
Hispanic or Latino
6
  16.7%
Not Hispanic or Latino
29
  80.6%
Unknown or Not Reported
1
   2.8%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 36 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
5
  13.9%
White
29
  80.6%
More than one race
0
   0.0%
Unknown or Not Reported
2
   5.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 36 participants
36
1.Primary Outcome
Title Median Time to Disease Progression
Hide Description Time to progression is defined as greater than or equal to 20% increase in plexiform neurofibromas (PN) volume on magnetic resonance imaging (MRI).
Time Frame 5 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Pirfenidone
Hide Arm/Group Description:
Pirfenidone orally as capsules three times a day approximately every 8 hours for cycles of 28 days with no rest period between cycles (28 day treatment cycles); 500 mg/m^2 every 8 hours (1500 mg/m2/day).
Overall Number of Participants Analyzed 36
Median (95% Confidence Interval)
Unit of Measure: Months
13.2
(8.2 to 18.6)
2.Primary Outcome
Title Number of Participants With Adverse Events
Hide Description Here are the number of participants with adverse events. For the detailed list of adverse events see the adverse event module.
Time Frame 5 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Pirfenidine
Hide Arm/Group Description:
Pirfenidone orally as capsules three times a day approximately every 8 hours for cycles of 28 days with no rest period between cycles (28 day treatment cycles); 500 mg/m^2 every 8 hours (1500 mg/m2/day).
Overall Number of Participants Analyzed 36
Measure Type: Count of Participants
Unit of Measure: Participants
36
 100.0%
3.Primary Outcome
Title Percentage of Participants Who Had an Objective Response Rate
Hide Description Objective response rate is defined as a complete response (CR) or partial response (PR). Complete response is a complete resolution of all measurable or palpable soft tissue tumors for ≥4 weeks and no appearance of new lesions. Partial response is a ≥50% reduction in the sum of the volume of all index lesions for ≥4 weeks.
Time Frame ≥4 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Pirfenidine
Hide Arm/Group Description:
Pirfenidone orally as capsules three times a day approximately every 8 hours for cycles of 28 days with no rest period between cycles (28 day treatment cycles); 500 mg/m^2 every 8 hours (1500 mg/m2/day).
Overall Number of Participants Analyzed 31
Measure Type: Number
Unit of Measure: percentage of participants
Complete Response 0
Partial Response 0
4.Secondary Outcome
Title Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline
Hide Description Quality of life was assessed by the Impact of Pediatric Illness scale for children 6-18 years of age. The child's primary caregiver completed the proxy Parent Form and children answered either the self-report Child or Adolescent (11-18 years) Form. The parallel IPI Scale forms assess four domains: adaptive behavior, emotional functioning, medical/physical status, and cognitive functioning. Responses to the 43 items are made on a 3-or5-point Likert scale (1 to 5 for Parent and Adolescent Form and 1, 3, 5 for the Child Form) ranging from "not at all" to "a lot". Item scores are transformed to a scale of 0-100, and then mean scores are calculated for the four domains and total scale with higher scores indicating better QOL. Baseline comparisons between child and parent total and domain scores were performed.
Time Frame Baseline
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Of the 36 patients enrolled, 28 were within the age range of the IPI Scale at baseline. Two patients did not have any QOL forms completed; one did not have a baseline evaluation, and six patients had missing follow-up evaluations. Thus, QOL data is presented for 19 subjects.
Arm/Group Title Pirfenidine
Hide Arm/Group Description:
Pirfenidone orally as capsules three times a day approximately every 8 hours for cycles of 28 days with no rest period between cycles (28 day treatment cycles); 500 mg/m^2 every 8 hours (1500 mg/m2/day).
Overall Number of Participants Analyzed 19
Mean (Standard Deviation)
Unit of Measure: Scores on a scale
Total score parent 72.7  (10.7)
Total score child 69.1  (12.0)
Parent proxy-adaptive behavior 72.1  (10.2)
Child Self-Report-adaptive behavior 76.7  (11.8)
Parent proxy - emotional functioning 72.4  (13.4)
Child Self-Report-emotional functioning 63.7  (14.7)
Parent proxy - medical/physical status 77.6  (15.1)
Child Self-Report - medical/physical status 67.1  (16.2)
Parent proxy - cognitive functioning 62.9  (19.6)
Child Self-Report- cognitive functioning 69.4  (18.5)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Pirfenidine
Comments F=5.45 under the null hypothesis
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0301
Comments The reported F statistic and p-value are representative of the difference in the Parent proxy Form and the Child Self-Report Form response for Adaptive Behavior.
Method ANOVA
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Pirfenidine
Comments F = 6.56 under the null hypothesis
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0186
Comments The reported p-value is representative of the difference in the Parent proxy Form and the Child Self-Report Form response for Emotional Functioning.
Method ANOVA
Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Pirfenidine
Comments F=11.23 under the null hypothesis
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0032
Comments The reported p-value is representative of the difference in the Parent proxy Form and the Child Self-Report Form response for Medical/Physical Status.
Method ANOVA
Comments [Not Specified]
5.Secondary Outcome
Title Longitudinal Total Quality of Life Scores Assessed by the Impact of Pediatric Illness Scale
Hide Description Quality of life was assessed by the Impact of Pediatric Illness scale for children 6-18 years of age. The child's primary caregiver completed the proxy Parent Form and children answered either the self-report Child or Adolescent (11-18 years) Form prior to cycles 1, 4, 7 and 10. The parallel IPI Scale forms assess four domains: adaptive behavior, emotional functioning, medical/physical status, and cognitive functioning. Responses to the 43 items are made on a 3-or5-point Likert scale (1 to 5 for Parent and Adolescent Form and 1, 3, 5 for the Child Form) ranging from "not at all" to "a lot". Item scores are transformed to a scale of 0-100, and then mean scores are calculated for the four domains and total scale with higher scores indicating better QOL.
Time Frame prior to cycles 1, 4, 7 and 10.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Of the 36 pts enrolled, 28 were within the age range of the IPI Scale at baseline. 2 pts did not have any QOL forms completed; 1 did not have a baseline & 6 pts had missing f/u evals. QOL data is presented for 19 pts. Due to pts not completing forms at random f/u evals, some time points have fewer than 19 pts included in the longitudinal analysis.
Arm/Group Title Pirfenidine
Hide Arm/Group Description:
Pirfenidone orally as capsules three times a day approximately every 8 hours for cycles of 28 days with no rest period between cycles (28 day treatment cycles); 500 mg/m^2 every 8 hours (1500 mg/m2/day).
Overall Number of Participants Analyzed 19
Mean (Standard Deviation)
Unit of Measure: scores on a scale
Parent Baseline (to Cycle 4) Number Analyzed 19 participants
71.1  (13.1)
Parent Pre Cycle 4 Number Analyzed 19 participants
71.8  (10.7)
Child Baseline (to Cycle 4) Number Analyzed 19 participants
67.9  (12.9)
Child Pre Cycle 4 Number Analyzed 19 participants
69.8  (10.8)
Parent Baseline (to Cycle 7) Number Analyzed 16 participants
71.7  (11.5)
Parent Pre Cycle 7 Number Analyzed 17 participants
70.6  (9.0)
Child Baseline (to Cycle 7) Number Analyzed 16 participants
69.5  (12.8)
Child Pre Cycle 7 Number Analyzed 17 participants
71.4  (12.3)
Parent Baseline (to Pre Cycle 10) Number Analyzed 13 participants
71.56  (12.6)
Parent Pre Cycle 10 Number Analyzed 15 participants
72.1  (7.8)
Child Baseline (to Pre Cycle 10) Number Analyzed 13 participants
67.5  (13.0)
Child Pre Cycle 10 Number Analyzed 15 participants
68.3  (14.1)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Pirfenidine
Comments F=0.25 under the null hypothesis
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6263
Comments [Not Specified]
Method ANOVA
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Pirfenidine
Comments F= 0.87 under the null hypothesis
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3625
Comments [Not Specified]
Method ANOVA
Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Pirfenidine
Comments F=0.31 under the null hypothesis
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5877
Comments [Not Specified]
Method ANOVA
Comments [Not Specified]
Show Statistical Analysis 4 Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Pirfenidine
Comments F=1.27 under the null hypothesis
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2767
Comments [Not Specified]
Method ANOVA
Comments [Not Specified]
Show Statistical Analysis 5 Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Pirfenidine
Comments F=0.04 under the null hypothesis
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8466
Comments [Not Specified]
Method ANOVA
Comments [Not Specified]
Show Statistical Analysis 6 Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Pirfenidine
Comments F=0.18 under the null hypothesis
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6774
Comments [Not Specified]
Method ANOVA
Comments [Not Specified]
6.Secondary Outcome
Title Number of Participants With A Response Evaluation Determined by the Comparison of One-Dimensional (1D) Magnetic Resonance Imaging
Hide Description Index lesions will be followed for progression by 1D magnetic resonance imaging. Progression is defined as a ≥20% increase in the volume of at least one of the index plexiform neurofibromas compared to the pretreatment volume measured prior to the start of treatment.
Time Frame Prior to cycles 1, 4, 7, and 10 and then every 6 cycles thereafter until progression
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
This outcome measure was not done because only 3D imaging was performed. Due to a detailed comparison of 1D-2D and 3D imaging for another study (Tipifarnib R115777) the investigator determined that a detailed comparison of 1D-2D and 3D analysis would really add no new knowledge. Therefore only 3D analysis was performed for this trial.
Arm/Group Title Pirfenidone
Hide Arm/Group Description:
Pirfenidone orally as capsules three times a day approximately every 8 hours for cycles of 28 days with no rest period between cycles (28 day treatment cycles); 500 mg/m^2 every 8 hours (1500 mg/m2/day).
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
7.Secondary Outcome
Title Number of Participants With A Response Evaluation Determined by the Comparison of Two-Dimensional (2D) Magnetic Resonance Imaging
Hide Description Index lesions will be followed for progression by 2D magnetic resonance imaging. Progression is defined as a ≥20% increase in the volume of at least one of the index plexiform neurofibromas compared to the pretreatment volume measured prior to the start of treatment.
Time Frame Prior to cycles 1, 4, 7, and 10 and then every 6 cycles thereafter until progression
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
This outcome measure was not done because only 3D imaging was performed. Due to a detailed comparison of 1D-2D and 3D imaging for another study (Tipifarnib R115777) the investigator determined that a detailed comparison of 1D-2D and 3D analysis would really add no new knowledge. Therefore only 3D analysis was performed for this trial.
Arm/Group Title Pirfenidone
Hide Arm/Group Description:
Pirfenidone orally as capsules three times a day approximately every 8 hours for cycles of 28 days with no rest period between cycles (28 day treatment cycles); 500 mg/m^2 every 8 hours (1500 mg/m2/day).
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
8.Secondary Outcome
Title Number of Participants With A Response Evaluation Determined by the Comparison of Three-Dimensional (3D) Magnetic Resonance Imaging
Hide Description Index lesions will be followed for progression by 3D magnetic resonance imaging. Compete response is a complete resolution of all measurable or palpable soft tissue tumors for ≥4 weeks and no appearance of new lesions. Partial response is a ≥50% reduction in the sum of the volume of all index lesions for ≥4 weeks. Progression is defined as a ≥20% increase in the volume of at least one of the index plexiform neurofibromas compared to the pretreatment volume measured prior to the start of treatment. Stable disease is a <20% increase, and <25% decrease in the sum of the volume of all index lesions for ≥4 weeks. Minor response is a ≥25% but <50% reduction in the sum of the volume of all index lesions for ≥4 weeks.
Time Frame Prior to cycles 1, 4, 7, and 10 and then every 6 cycles thereafter, approximately 5 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Five patients were not analyzed due to clinical progression (n=1), plexiform neurofibroma surgery (n=1), progression in a pre-existing brain tumor (n=1), and refusal of further therapy (n=2).
Arm/Group Title Pirfenidone
Hide Arm/Group Description:
Pirfenidone orally as capsules three times a day approximately every 8 hours for cycles of 28 days with no rest period between cycles (28 day treatment cycles); 500 mg/m^2 every 8 hours (1500 mg/m^2/day).
Overall Number of Participants Analyzed 31
Measure Type: Count of Participants
Unit of Measure: Participants
Progressive disease
31
 100.0%
Complete response
0
   0.0%
Partial response
0
   0.0%
Stable disease
0
   0.0%
MInor response
0
   0.0%
9.Secondary Outcome
Title Number of Participants Who Contributed to the Tissue Bank
Hide Description Tumor specimens from patients who undergo tumor surgery or biopsies for clinical reasons.
Time Frame 5 years
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Participants declined to contribute specimens for the tissue bank.
Arm/Group Title Pirfenidine
Hide Arm/Group Description:
Pirfenidone orally as capsules three times a day approximately every 8 hours for cycles of 28 days with no rest period between cycles (28 day treatment cycles); 500 mg/m^2 every 8 hours (1500 mg/m2/day).
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame 5 years
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Pirfenidone
Hide Arm/Group Description Pirfenidone orally as capsules three times a day approximately every 8 hours for cycles of 28 days with no rest period between cycles (28 day treatment cycles); 500 mg/m^2 every 8 hours (1500 mg/m2/day).
All-Cause Mortality
Pirfenidone
Affected / at Risk (%)
Total   0/36 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Pirfenidone
Affected / at Risk (%) # Events
Total   3/36 (8.33%)    
Blood and lymphatic system disorders   
Hemorrhage, CNS  1  1/36 (2.78%)  1
Respiratory, thoracic and mediastinal disorders   
Adult Respiratory Distress Syndrome (ARDS)  1  1/36 (2.78%)  1
Atelectasis  1  1/36 (2.78%)  1
Bronchospasm, wheezing  1  1/36 (2.78%)  1
Cough  1  1/36 (2.78%)  1
Dyspnea (shortness of breath)  1  1/36 (2.78%)  1
Hypoxia  1  1/36 (2.78%)  1
Pneumothorax  1  1/36 (2.78%)  1
1
Term from vocabulary, CTCAE (3.0)
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Pirfenidone
Affected / at Risk (%) # Events
Total   36/36 (100.00%)    
Blood and lymphatic system disorders   
Hemoglobin  1  5/36 (13.89%)  10
Lymphatics-Other (Specify-bilateral lymphadenopathy)  1 [1]  4/36 (11.11%)  4
Lymphopenia  1  6/36 (16.67%)  8
Cardiac disorders   
Cardiac arrhythmia-Other, specify- sinus tachycardia  1  1/36 (2.78%)  1
Cardiac general, Other-(specify-fluid retention)  1  1/36 (2.78%)  1
Supraventricular and nodal arrhythmia: sinus tachycardia  1  1/36 (2.78%)  1
Ear and labyrinth disorders   
Hearing: patients without baseline audiogram and not enrolled in a monitoring program  1  1/36 (2.78%)  1
Otitis, middle ear (non-infectious)  1  3/36 (8.33%)  6
Pain: middle ear  1  1/36 (2.78%)  1
Pain-Other (Specify-left ear pain)  1 [2]  4/36 (11.11%)  6
Eye disorders   
Dry eye syndrome  1  1/36 (2.78%)  1
Ocular/Visual-Other (Specify-blurred vision intermittent)  1  2/36 (5.56%)  2
Vision-photophobia  1  1/36 (2.78%)  1
Gastrointestinal disorders   
Anorexia  1  9/36 (25.00%)  22
Constipation  1  6/36 (16.67%)  6
Dehydration  1  3/36 (8.33%)  3
Dental: teeth  1  1/36 (2.78%)  1
Diarrhea  1  21/36 (58.33%)  63
Gastrointestinal-Other (Specify- 1 episode of incontinence of bowel)  1 [3]  2/36 (5.56%)  2
Heartburn/dyspepsia  1  2/36 (5.56%)  2
Mucositis/stomatitis (clinical exam): oral cavity  1  2/36 (5.56%)  3
Nausea  1  29/36 (80.56%)  157
Pain: dental/teeth/peridontal  1  2/36 (5.56%)  3
Pain: esophagus  1  1/36 (2.78%)  1
Pain: stomach  1  2/36 (5.56%)  2
Pain: throat/pharynx/larynx  1  4/36 (11.11%)  5
Taste alteration (dysgeusia)  1  1/36 (2.78%)  1
Vomiting  1  25/36 (69.44%)  119
General disorders   
Constitutional symptoms (Other, specify-cold symptoms)  1 [4]  3/36 (8.33%)  4
Edema: head and neck  1  1/36 (2.78%)  1
Edema: limb  1  1/36 (2.78%)  1
Fatigue (asthenia, lethargy, malaise)  1  11/36 (30.56%)  21
Fever (in the absence of neutropenia, where neutropenia is defined as ANC < 1.0 x 10e9/L)  1  10/36 (27.78%)  20
Flu-like syndrome  1  6/36 (16.67%)  7
Immune system disorders   
Allergic rhinitis (including sneezing, nasal stuffiness, postnasal drip)  1  7/36 (19.44%)  8
Infections and infestations   
Infection (documented clinically or microbiologically) with grade 3 or 4 neutrophils (ANC < 1.0 x 10  1 [5]  1/36 (2.78%)  1
Infection-Other (specify-infection)  1 [6]  6/36 (16.67%)  7
Infection with normal ANC or Grade 1 or 2 neutrophils : bronchus  1  2/36 (5.56%)  2
Infection with normal ANC or Grade 1 or 2 neutrophils : catheter-related  1  1/36 (2.78%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils : conjunctiva  1  2/36 (5.56%)  2
Infection with normal ANC or Grade 1 or 2 neutrophils : foreign body (e.g. graft, implant, prosthesi  1  1/36 (2.78%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils : lung (pneumonia)  1  1/36 (2.78%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils : middle ear (otitis media)  1  2/36 (5.56%)  2
Infection with normal ANC or Grade 1 or 2 neutrophils : penis  1  1/36 (2.78%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils : pharynx  1  1/36 (2.78%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils : sinus  1  2/36 (5.56%)  2
Infection with normal ANC or Grade 1 or 2 neutrophils : skin (cellulitis)  1  2/36 (5.56%)  3
Infection with normal ANC or Grade 1 or 2 neutrophils : upper aerodigestive NOS  1  1/36 (2.78%)  1
Infection with normal ANC or Grade 1 or 2 neutrophils : upper airway NOS  1  5/36 (13.89%)  7
Infection with unknown ANC : external ear (otitis externa)  1  2/36 (5.56%)  3
Infection with unknown ANC : lung (pneumonia)  1  1/36 (2.78%)  1
Infection with unknown ANC : sinus  1  2/36 (5.56%)  2
Infection with unknown ANC : trachea  1  1/36 (2.78%)  1
Infection with unknown ANC : upper airway NOS  1  2/36 (5.56%)  3
Investigations   
ALT, SGPT (serum glutamic pyruvic transaminase)  1  2/36 (5.56%)  2
Bicarbonate, serum low  1  4/36 (11.11%)  7
Leukocytes (total WBC)  1  8/36 (22.22%)  22
Neutrophils/granulocytes (ANC/AGC)  1  7/36 (19.44%)  24
PTT (partial thromboplastin time)  1  1/36 (2.78%)  1
Platelets  1  3/36 (8.33%)  5
AST, SGOT (serum oxaloacetic transaminase)  1  2/36 (5.56%)  3
Alkaline phosphatase  1  3/36 (8.33%)  3
Metabolism and nutrition disorders   
Albumin, serum low (hypoalbuminemia)  1  5/36 (13.89%)  7
Calcium, serum-high (hypercalcemia)  1  4/36 (11.11%)  5
Calcium, hypocalcemia  1  9/36 (25.00%)  13
Glucose, serum-high (hyperglycemia)  1  4/36 (11.11%)  4
Glucose, serum low (hypoglycemia)  1  1/36 (2.78%)  1
Magnesium, serum high (hype  1  8/36 (22.22%)  9
Magnesium, serum low (hypomagnesemia)  1  3/36 (8.33%)  3
Phosphate, serum low (hypophosphatemia)  1  2/36 (5.56%)  3
Potassium, serum high (hyperkalemia)  1  1/36 (2.78%)  1
Potassium, serum low (hypokalemia)  1  4/36 (11.11%)  6
Sodium, serum-high (hypernatremia)  1  3/36 (8.33%)  3
Sodium, serum-low (hyponatremia)  1  2/36 (5.56%)  2
Musculoskeletal and connective tissue disorders   
Lumbar spine-range of motion  1  1/36 (2.78%)  3
Muscle weaknesses, generalized or specific area (not due to neuropathy): extreme-lower  1  2/36 (5.56%)  2
Pain: back  1  5/36 (13.89%)  6
Pain: bone  1  1/36 (2.78%)  2
Pain: buttock  1  1/36 (2.78%)  1
Pain: chest wall  1  1/36 (2.78%)  2
Pain: chest/thorax NOS  1  4/36 (11.11%)  4
Pain: extremity-limb  1  7/36 (19.44%)  13
Pain: joint  1  2/36 (5.56%)  3
Pain: muscle  1  4/36 (11.11%)  7
Pain: neck  1  5/36 (13.89%)  15
Nervous system disorders   
Dizziness  1  4/36 (11.11%)  5
Mood alteration: agitation  1  2/36 (5.56%)  2
Mood alteration: depression  1  2/36 (5.56%)  3
Neurology-Other (specify-tic, constant clearing of throat)  1  1/36 (2.78%)  1
Neuropathy: motor  1  3/36 (8.33%)  3
Neuropathy: sensory  1  2/36 (5.56%)  2
Pain: head/headache  1  21/36 (58.33%)  52
Pain: pain NOS  1  4/36 (11.11%)  4
Tremor  1  1/36 (2.78%)  1
Psychiatric disorders   
Personality/behavioral  1  5/36 (13.89%)  7
Renal and urinary disorders   
Hemorrhage, GU: urinary NOA  1  1/36 (2.78%)  2
Incontinence, urinary  1  2/36 (5.56%)  2
Pain: bladder  1  1/36 (2.78%)  3
Reproductive system and breast disorders   
Hemorrhage, GU: Ovary  1  1/36 (2.78%)  1
Pain: abdomen NOS  1  5/36 (13.89%)  8
Pain: pelvis  1  1/36 (2.78%)  1
Pain: penis  1  1/36 (2.78%)  1
Respiratory, thoracic and mediastinal disorders   
Bronchospasm, wheezing  1  1/36 (2.78%)  2
Hemorrhage, pulmonary/upper respiratory: nose  1  2/36 (5.56%)  3
Obstruction/stenosis of airway: bronchus  1  1/36 (2.78%)  1
Obstruction/stenosis of airway: larynx  1  1/36 (2.78%)  1
Pneumonitis/pulmonary infiltrates  1  1/36 (2.78%)  1
Pulmonary/upper respiratory-Other (Specify-bloody nose)  1 [7]  3/36 (8.33%)  5
Cough  1  10/36 (27.78%)  11
Skin and subcutaneous tissue disorders   
Atrophy, skin  1  1/36 (2.78%)  1
Dermatology/Skin-Other (Specify-multiple bug bites)  1 [8]  4/36 (11.11%)  6
Pain: skin  1  1/36 (2.78%)  1
Pruritis/itching  1  5/36 (13.89%)  7
Rash/desquamation  1  2/36 (5.56%)  4
Rash: acne/acneiform  1  1/36 (2.78%)  1
Rash: hand-foot skin reaction  1  2/36 (5.56%)  3
Ulceration  1  1/36 (2.78%)  1
Vascular disorders   
Hypertension  1  3/36 (8.33%)  3
Vascular-Other (Specify-vascular other specify, coolness LL)  1  1/36 (2.78%)  1
Flushing  1  1/36 (2.78%)  1
1
Term from vocabulary, CTCAE (3.0)
Indicates events were collected by systematic assessment
[1]
lymphatics-other (specify-cervical lymphadenopathy) snoring and gasping shotty lymphadenopathy cervical chain
[2]
mild mouth aching orthodontic discomfort pain (head site of surgery) pain rt side
[3]
gastrointestinal-other, specify, stool, green
[4]
cough insomnia
[5]
Infection (documented clinically or microbiologically) with grade 3 or 4 neutrophils (ANC < 1.0 x 10e9/L: External ear (otitis externa)
[6]
infection oral cavity, thrush sinus infection sinus infection + cough bronchitis, congestion infection-ear
[7]
nasal congestion cold symptoms wheezing secondary to tracheitis
[8]
nodule in L scapula rash caused by fungal infection cut foot 3 stitches insect bites
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.
Results Point of Contact
Name/Title: Brigitte C. Widemann, M.D.
Organization: National Cancer Institute (NCI), National Institutes of Health (NIH)
Phone: 301-496-7387
Responsible Party: Brigitte Widemann, M.D., National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT00076102     History of Changes
Obsolete Identifiers: NCT00078936
Other Study ID Numbers: 040080
04-C-0080
FD-R-0002128 ( Other Grant/Funding Number: FDA OOPD )
First Submitted: January 13, 2004
First Posted: January 14, 2004
Results First Submitted: January 4, 2012
Results First Posted: July 12, 2012
Last Update Posted: April 23, 2018