Trial Outcomes & Findings for Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas (NCT NCT00076102)
NCT ID: NCT00076102
Last Updated: 2018-04-23
Results Overview
Time to progression is defined as greater than or equal to 20% increase in plexiform neurofibromas (PN) volume on magnetic resonance imaging (MRI).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
36 participants
Primary outcome timeframe
5 years
Results posted on
2018-04-23
Participant Flow
36 participants were enrolled in this study
Participant milestones
| Measure |
Pirfenidone
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 Study
STARTED
|
36
|
|
Overall Study
COMPLETED
|
31
|
|
Overall Study
NOT COMPLETED
|
5
|
Reasons for withdrawal
| Measure |
Pirfenidone
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 Study
Clinical progression
|
1
|
|
Overall Study
Progression in pre-existing brain tumor
|
1
|
|
Overall Study
plexiform neurofibroma surgery
|
1
|
|
Overall Study
refusal of further therapy
|
2
|
Baseline Characteristics
Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas
Baseline characteristics by cohort
| Measure |
Pirfenidone
n=36 Participants
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).
|
|---|---|
|
Age, Categorical
<=18 years
|
34 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
9.81 years
STANDARD_DEVIATION 4.52 • n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
26 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
29 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
29 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
36 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 5 yearsTime to progression is defined as greater than or equal to 20% increase in plexiform neurofibromas (PN) volume on magnetic resonance imaging (MRI).
Outcome measures
| Measure |
Pirfenidone
n=36 Participants
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).
|
|---|---|
|
Median Time to Disease Progression
|
13.2 Months
Interval 8.2 to 18.6
|
PRIMARY outcome
Timeframe: 5 yearsHere are the number of participants with adverse events. For the detailed list of adverse events see the adverse event module.
Outcome measures
| Measure |
Pirfenidone
n=36 Participants
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).
|
|---|---|
|
Number of Participants With Adverse Events
|
36 Participants
|
PRIMARY outcome
Timeframe: ≥4 weeksObjective 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.
Outcome measures
| Measure |
Pirfenidone
n=31 Participants
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).
|
|---|---|
|
Percentage of Participants Who Had an Objective Response Rate
Complete Response
|
0 percentage of participants
|
|
Percentage of Participants Who Had an Objective Response Rate
Partial Response
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: BaselinePopulation: 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.
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.
Outcome measures
| Measure |
Pirfenidone
n=19 Participants
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).
|
|---|---|
|
Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline
Total score parent
|
72.7 Scores on a scale
Standard Deviation 10.7
|
|
Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline
Total score child
|
69.1 Scores on a scale
Standard Deviation 12.0
|
|
Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline
Parent proxy-adaptive behavior
|
72.1 Scores on a scale
Standard Deviation 10.2
|
|
Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline
Child Self-Report-adaptive behavior
|
76.7 Scores on a scale
Standard Deviation 11.8
|
|
Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline
Parent proxy - emotional functioning
|
72.4 Scores on a scale
Standard Deviation 13.4
|
|
Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline
Child Self-Report-emotional functioning
|
63.7 Scores on a scale
Standard Deviation 14.7
|
|
Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline
Parent proxy - medical/physical status
|
77.6 Scores on a scale
Standard Deviation 15.1
|
|
Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline
Child Self-Report - medical/physical status
|
67.1 Scores on a scale
Standard Deviation 16.2
|
|
Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline
Parent proxy - cognitive functioning
|
62.9 Scores on a scale
Standard Deviation 19.6
|
|
Quality of Life (QOL) Using the Impact of Pediatric Illness (IPI) Scale at Baseline
Child Self-Report- cognitive functioning
|
69.4 Scores on a scale
Standard Deviation 18.5
|
SECONDARY outcome
Timeframe: prior to cycles 1, 4, 7 and 10.Population: 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.
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.
Outcome measures
| Measure |
Pirfenidone
n=19 Participants
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).
|
|---|---|
|
Longitudinal Total Quality of Life Scores Assessed by the Impact of Pediatric Illness Scale
Parent Baseline (to Cycle 4)
|
71.1 scores on a scale
Standard Deviation 13.1
|
|
Longitudinal Total Quality of Life Scores Assessed by the Impact of Pediatric Illness Scale
Parent Pre Cycle 4
|
71.8 scores on a scale
Standard Deviation 10.7
|
|
Longitudinal Total Quality of Life Scores Assessed by the Impact of Pediatric Illness Scale
Child Baseline (to Cycle 4)
|
67.9 scores on a scale
Standard Deviation 12.9
|
|
Longitudinal Total Quality of Life Scores Assessed by the Impact of Pediatric Illness Scale
Child Pre Cycle 4
|
69.8 scores on a scale
Standard Deviation 10.8
|
|
Longitudinal Total Quality of Life Scores Assessed by the Impact of Pediatric Illness Scale
Parent Baseline (to Cycle 7)
|
71.7 scores on a scale
Standard Deviation 11.5
|
|
Longitudinal Total Quality of Life Scores Assessed by the Impact of Pediatric Illness Scale
Parent Pre Cycle 7
|
70.6 scores on a scale
Standard Deviation 9.0
|
|
Longitudinal Total Quality of Life Scores Assessed by the Impact of Pediatric Illness Scale
Child Baseline (to Cycle 7)
|
69.5 scores on a scale
Standard Deviation 12.8
|
|
Longitudinal Total Quality of Life Scores Assessed by the Impact of Pediatric Illness Scale
Child Pre Cycle 7
|
71.4 scores on a scale
Standard Deviation 12.3
|
|
Longitudinal Total Quality of Life Scores Assessed by the Impact of Pediatric Illness Scale
Parent Baseline (to Pre Cycle 10)
|
71.56 scores on a scale
Standard Deviation 12.6
|
|
Longitudinal Total Quality of Life Scores Assessed by the Impact of Pediatric Illness Scale
Parent Pre Cycle 10
|
72.1 scores on a scale
Standard Deviation 7.8
|
|
Longitudinal Total Quality of Life Scores Assessed by the Impact of Pediatric Illness Scale
Child Baseline (to Pre Cycle 10)
|
67.5 scores on a scale
Standard Deviation 13.0
|
|
Longitudinal Total Quality of Life Scores Assessed by the Impact of Pediatric Illness Scale
Child Pre Cycle 10
|
68.3 scores on a scale
Standard Deviation 14.1
|
SECONDARY outcome
Timeframe: Prior to cycles 1, 4, 7, and 10 and then every 6 cycles thereafter until progressionPopulation: 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.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Prior to cycles 1, 4, 7, and 10 and then every 6 cycles thereafter until progressionPopulation: 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.
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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Prior to cycles 1, 4, 7, and 10 and then every 6 cycles thereafter, approximately 5 yearsPopulation: 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).
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.
Outcome measures
| Measure |
Pirfenidone
n=31 Participants
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).
|
|---|---|
|
Number of Participants With A Response Evaluation Determined by the Comparison of Three-Dimensional (3D) Magnetic Resonance Imaging
Progressive disease
|
31 Participants
|
|
Number of Participants With A Response Evaluation Determined by the Comparison of Three-Dimensional (3D) Magnetic Resonance Imaging
Complete response
|
0 Participants
|
|
Number of Participants With A Response Evaluation Determined by the Comparison of Three-Dimensional (3D) Magnetic Resonance Imaging
Partial response
|
0 Participants
|
|
Number of Participants With A Response Evaluation Determined by the Comparison of Three-Dimensional (3D) Magnetic Resonance Imaging
Stable disease
|
0 Participants
|
|
Number of Participants With A Response Evaluation Determined by the Comparison of Three-Dimensional (3D) Magnetic Resonance Imaging
MInor response
|
0 Participants
|
SECONDARY outcome
Timeframe: 5 yearsPopulation: Participants declined to contribute specimens for the tissue bank.
Tumor specimens from patients who undergo tumor surgery or biopsies for clinical reasons.
Outcome measures
Outcome data not reported
Adverse Events
Pirfenidone
Serious events: 3 serious events
Other events: 36 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Pirfenidone
n=36 participants at risk
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).
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Adult Respiratory Distress Syndrome (ARDS)
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm, wheezing
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Blood and lymphatic system disorders
Hemorrhage, CNS
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
2.8%
1/36 • Number of events 1 • 5 years
|
Other adverse events
| Measure |
Pirfenidone
n=36 participants at risk
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).
|
|---|---|
|
Investigations
ALT, SGPT (serum glutamic pyruvic transaminase)
|
5.6%
2/36 • Number of events 2 • 5 years
|
|
Metabolism and nutrition disorders
Albumin, serum low (hypoalbuminemia)
|
13.9%
5/36 • Number of events 7 • 5 years
|
|
Immune system disorders
Allergic rhinitis (including sneezing, nasal stuffiness, postnasal drip)
|
19.4%
7/36 • Number of events 8 • 5 years
|
|
Gastrointestinal disorders
Anorexia
|
25.0%
9/36 • Number of events 22 • 5 years
|
|
Skin and subcutaneous tissue disorders
Atrophy, skin
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Investigations
Bicarbonate, serum low
|
11.1%
4/36 • Number of events 7 • 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm, wheezing
|
2.8%
1/36 • Number of events 2 • 5 years
|
|
Metabolism and nutrition disorders
Calcium, serum-high (hypercalcemia)
|
11.1%
4/36 • Number of events 5 • 5 years
|
|
Metabolism and nutrition disorders
Calcium, hypocalcemia
|
25.0%
9/36 • Number of events 13 • 5 years
|
|
Cardiac disorders
Cardiac arrhythmia-Other, specify- sinus tachycardia
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Cardiac disorders
Cardiac general, Other-(specify-fluid retention)
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Gastrointestinal disorders
Constipation
|
16.7%
6/36 • Number of events 6 • 5 years
|
|
General disorders
Constitutional symptoms (Other, specify-cold symptoms)
|
8.3%
3/36 • Number of events 4 • 5 years
|
|
Gastrointestinal disorders
Dehydration
|
8.3%
3/36 • Number of events 3 • 5 years
|
|
Gastrointestinal disorders
Dental: teeth
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Skin and subcutaneous tissue disorders
Dermatology/Skin-Other (Specify-multiple bug bites)
|
11.1%
4/36 • Number of events 6 • 5 years
|
|
Gastrointestinal disorders
Diarrhea
|
58.3%
21/36 • Number of events 63 • 5 years
|
|
Nervous system disorders
Dizziness
|
11.1%
4/36 • Number of events 5 • 5 years
|
|
Eye disorders
Dry eye syndrome
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
General disorders
Edema: head and neck
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
General disorders
Edema: limb
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
General disorders
Fatigue (asthenia, lethargy, malaise)
|
30.6%
11/36 • Number of events 21 • 5 years
|
|
General disorders
Fever (in the absence of neutropenia, where neutropenia is defined as ANC < 1.0 x 10e9/L)
|
27.8%
10/36 • Number of events 20 • 5 years
|
|
General disorders
Flu-like syndrome
|
16.7%
6/36 • Number of events 7 • 5 years
|
|
Gastrointestinal disorders
Gastrointestinal-Other (Specify- 1 episode of incontinence of bowel)
|
5.6%
2/36 • Number of events 2 • 5 years
|
|
Metabolism and nutrition disorders
Glucose, serum-high (hyperglycemia)
|
11.1%
4/36 • Number of events 4 • 5 years
|
|
Metabolism and nutrition disorders
Glucose, serum low (hypoglycemia)
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Ear and labyrinth disorders
Hearing: patients without baseline audiogram and not enrolled in a monitoring program
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Gastrointestinal disorders
Heartburn/dyspepsia
|
5.6%
2/36 • Number of events 2 • 5 years
|
|
Blood and lymphatic system disorders
Hemoglobin
|
13.9%
5/36 • Number of events 10 • 5 years
|
|
Reproductive system and breast disorders
Hemorrhage, GU: Ovary
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Renal and urinary disorders
Hemorrhage, GU: urinary NOA
|
2.8%
1/36 • Number of events 2 • 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Hemorrhage, pulmonary/upper respiratory: nose
|
5.6%
2/36 • Number of events 3 • 5 years
|
|
Vascular disorders
Hypertension
|
8.3%
3/36 • Number of events 3 • 5 years
|
|
Renal and urinary disorders
Incontinence, urinary
|
5.6%
2/36 • Number of events 2 • 5 years
|
|
Infections and infestations
Infection (documented clinically or microbiologically) with grade 3 or 4 neutrophils (ANC < 1.0 x 10
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Infections and infestations
Infection-Other (specify-infection)
|
16.7%
6/36 • Number of events 7 • 5 years
|
|
Investigations
Leukocytes (total WBC)
|
22.2%
8/36 • Number of events 22 • 5 years
|
|
Musculoskeletal and connective tissue disorders
Lumbar spine-range of motion
|
2.8%
1/36 • Number of events 3 • 5 years
|
|
Blood and lymphatic system disorders
Lymphatics-Other (Specify-bilateral lymphadenopathy)
|
11.1%
4/36 • Number of events 4 • 5 years
|
|
Blood and lymphatic system disorders
Lymphopenia
|
16.7%
6/36 • Number of events 8 • 5 years
|
|
Metabolism and nutrition disorders
Magnesium, serum high (hype
|
22.2%
8/36 • Number of events 9 • 5 years
|
|
Metabolism and nutrition disorders
Magnesium, serum low (hypomagnesemia)
|
8.3%
3/36 • Number of events 3 • 5 years
|
|
Nervous system disorders
Mood alteration: agitation
|
5.6%
2/36 • Number of events 2 • 5 years
|
|
Nervous system disorders
Mood alteration: depression
|
5.6%
2/36 • Number of events 3 • 5 years
|
|
Gastrointestinal disorders
Mucositis/stomatitis (clinical exam): oral cavity
|
5.6%
2/36 • Number of events 3 • 5 years
|
|
Musculoskeletal and connective tissue disorders
Muscle weaknesses, generalized or specific area (not due to neuropathy): extreme-lower
|
5.6%
2/36 • Number of events 2 • 5 years
|
|
Gastrointestinal disorders
Nausea
|
80.6%
29/36 • Number of events 157 • 5 years
|
|
Nervous system disorders
Neurology-Other (specify-tic, constant clearing of throat)
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Nervous system disorders
Neuropathy: motor
|
8.3%
3/36 • Number of events 3 • 5 years
|
|
Nervous system disorders
Neuropathy: sensory
|
5.6%
2/36 • Number of events 2 • 5 years
|
|
Investigations
Neutrophils/granulocytes (ANC/AGC)
|
19.4%
7/36 • Number of events 24 • 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Obstruction/stenosis of airway: bronchus
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Obstruction/stenosis of airway: larynx
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Eye disorders
Ocular/Visual-Other (Specify-blurred vision intermittent)
|
5.6%
2/36 • Number of events 2 • 5 years
|
|
Ear and labyrinth disorders
Otitis, middle ear (non-infectious)
|
8.3%
3/36 • Number of events 6 • 5 years
|
|
Investigations
PTT (partial thromboplastin time)
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Reproductive system and breast disorders
Pain: abdomen NOS
|
13.9%
5/36 • Number of events 8 • 5 years
|
|
Musculoskeletal and connective tissue disorders
Pain: back
|
13.9%
5/36 • Number of events 6 • 5 years
|
|
Renal and urinary disorders
Pain: bladder
|
2.8%
1/36 • Number of events 3 • 5 years
|
|
Musculoskeletal and connective tissue disorders
Pain: bone
|
2.8%
1/36 • Number of events 2 • 5 years
|
|
Musculoskeletal and connective tissue disorders
Pain: buttock
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Musculoskeletal and connective tissue disorders
Pain: chest wall
|
2.8%
1/36 • Number of events 2 • 5 years
|
|
Musculoskeletal and connective tissue disorders
Pain: chest/thorax NOS
|
11.1%
4/36 • Number of events 4 • 5 years
|
|
Gastrointestinal disorders
Pain: dental/teeth/peridontal
|
5.6%
2/36 • Number of events 3 • 5 years
|
|
Gastrointestinal disorders
Pain: esophagus
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Musculoskeletal and connective tissue disorders
Pain: extremity-limb
|
19.4%
7/36 • Number of events 13 • 5 years
|
|
Nervous system disorders
Pain: head/headache
|
58.3%
21/36 • Number of events 52 • 5 years
|
|
Musculoskeletal and connective tissue disorders
Pain: joint
|
5.6%
2/36 • Number of events 3 • 5 years
|
|
Ear and labyrinth disorders
Pain: middle ear
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Musculoskeletal and connective tissue disorders
Pain: muscle
|
11.1%
4/36 • Number of events 7 • 5 years
|
|
Musculoskeletal and connective tissue disorders
Pain: neck
|
13.9%
5/36 • Number of events 15 • 5 years
|
|
Nervous system disorders
Pain: pain NOS
|
11.1%
4/36 • Number of events 4 • 5 years
|
|
Reproductive system and breast disorders
Pain: pelvis
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Reproductive system and breast disorders
Pain: penis
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Skin and subcutaneous tissue disorders
Pain: skin
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Gastrointestinal disorders
Pain: stomach
|
5.6%
2/36 • Number of events 2 • 5 years
|
|
Gastrointestinal disorders
Pain: throat/pharynx/larynx
|
11.1%
4/36 • Number of events 5 • 5 years
|
|
Psychiatric disorders
Personality/behavioral
|
13.9%
5/36 • Number of events 7 • 5 years
|
|
Metabolism and nutrition disorders
Phosphate, serum low (hypophosphatemia)
|
5.6%
2/36 • Number of events 3 • 5 years
|
|
Investigations
Platelets
|
8.3%
3/36 • Number of events 5 • 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis/pulmonary infiltrates
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Metabolism and nutrition disorders
Potassium, serum high (hyperkalemia)
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Metabolism and nutrition disorders
Potassium, serum low (hypokalemia)
|
11.1%
4/36 • Number of events 6 • 5 years
|
|
Skin and subcutaneous tissue disorders
Pruritis/itching
|
13.9%
5/36 • Number of events 7 • 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary/upper respiratory-Other (Specify-bloody nose)
|
8.3%
3/36 • Number of events 5 • 5 years
|
|
Skin and subcutaneous tissue disorders
Rash/desquamation
|
5.6%
2/36 • Number of events 4 • 5 years
|
|
Skin and subcutaneous tissue disorders
Rash: acne/acneiform
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Skin and subcutaneous tissue disorders
Rash: hand-foot skin reaction
|
5.6%
2/36 • Number of events 3 • 5 years
|
|
Metabolism and nutrition disorders
Sodium, serum-high (hypernatremia)
|
8.3%
3/36 • Number of events 3 • 5 years
|
|
Metabolism and nutrition disorders
Sodium, serum-low (hyponatremia)
|
5.6%
2/36 • Number of events 2 • 5 years
|
|
Cardiac disorders
Supraventricular and nodal arrhythmia: sinus tachycardia
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Gastrointestinal disorders
Taste alteration (dysgeusia)
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Nervous system disorders
Tremor
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Skin and subcutaneous tissue disorders
Ulceration
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Vascular disorders
Vascular-Other (Specify-vascular other specify, coolness LL)
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Eye disorders
Vision-photophobia
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Gastrointestinal disorders
Vomiting
|
69.4%
25/36 • Number of events 119 • 5 years
|
|
Vascular disorders
Flushing
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
27.8%
10/36 • Number of events 11 • 5 years
|
|
Ear and labyrinth disorders
Pain-Other (Specify-left ear pain)
|
11.1%
4/36 • Number of events 6 • 5 years
|
|
Investigations
AST, SGOT (serum oxaloacetic transaminase)
|
5.6%
2/36 • Number of events 3 • 5 years
|
|
Investigations
Alkaline phosphatase
|
8.3%
3/36 • Number of events 3 • 5 years
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils : bronchus
|
5.6%
2/36 • Number of events 2 • 5 years
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils : catheter-related
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils : conjunctiva
|
5.6%
2/36 • Number of events 2 • 5 years
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils : foreign body (e.g. graft, implant, prosthesi
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils : lung (pneumonia)
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils : middle ear (otitis media)
|
5.6%
2/36 • Number of events 2 • 5 years
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils : penis
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils : pharynx
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils : sinus
|
5.6%
2/36 • Number of events 2 • 5 years
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils : skin (cellulitis)
|
5.6%
2/36 • Number of events 3 • 5 years
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils : upper aerodigestive NOS
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils : upper airway NOS
|
13.9%
5/36 • Number of events 7 • 5 years
|
|
Infections and infestations
Infection with unknown ANC : external ear (otitis externa)
|
5.6%
2/36 • Number of events 3 • 5 years
|
|
Infections and infestations
Infection with unknown ANC : lung (pneumonia)
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Infections and infestations
Infection with unknown ANC : sinus
|
5.6%
2/36 • Number of events 2 • 5 years
|
|
Infections and infestations
Infection with unknown ANC : trachea
|
2.8%
1/36 • Number of events 1 • 5 years
|
|
Infections and infestations
Infection with unknown ANC : upper airway NOS
|
5.6%
2/36 • Number of events 3 • 5 years
|
Additional Information
Brigitte C. Widemann, M.D.
National Cancer Institute (NCI), National Institutes of Health (NIH)
Phone: 301-496-7387
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place