Trial Outcomes & Findings for R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas (NCT NCT00021541)
NCT ID: NCT00021541
Last Updated: 2018-04-17
Results Overview
Median time to progression is defined as a greater than or equal to 20% increase increase in the sum of the volume of all index lesions based on volumetric analysis utilizing magnetic resonance imaging (MRI).Start of phase A or phase B to time of progression.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
62 participants
Primary outcome timeframe
8 years
Results posted on
2018-04-17
Participant Flow
Participant milestones
| Measure |
Phase A -Tipifarnib
Patients receive oral tipifarnib every 12 hours on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.Patients receive tipifarnib ONLY in the first treatment until they progress to the second treatment/crossover to receive placebo.
|
Phase B - Placebo
Patients receive oral placebo every 12 hours on days 1-21. Courses repeat as in arm I.Patients receive placebo ONLY in the first treatment until they progress to the second treatment/crossover to receive tipifarnib.
|
|---|---|---|
|
Period 1-First Treatment
STARTED
|
31
|
31
|
|
Period 1-First Treatment
Pts Who Progressed to Second Treatment
|
20
|
23
|
|
Period 1-First Treatment
Ineligible Patients
|
0
|
2
|
|
Period 1-First Treatment
COMPLETED
|
20
|
23
|
|
Period 1-First Treatment
NOT COMPLETED
|
11
|
8
|
|
Period 2-crossover
STARTED
|
20
|
23
|
|
Period 2-crossover
COMPLETED
|
10
|
12
|
|
Period 2-crossover
NOT COMPLETED
|
10
|
11
|
Reasons for withdrawal
| Measure |
Phase A -Tipifarnib
Patients receive oral tipifarnib every 12 hours on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.Patients receive tipifarnib ONLY in the first treatment until they progress to the second treatment/crossover to receive placebo.
|
Phase B - Placebo
Patients receive oral placebo every 12 hours on days 1-21. Courses repeat as in arm I.Patients receive placebo ONLY in the first treatment until they progress to the second treatment/crossover to receive tipifarnib.
|
|---|---|---|
|
Period 1-First Treatment
Withdrawal by Subject
|
3
|
3
|
|
Period 1-First Treatment
Adverse Event
|
2
|
1
|
|
Period 1-First Treatment
non-adherence
|
3
|
0
|
|
Period 1-First Treatment
Death
|
1
|
0
|
|
Period 1-First Treatment
Pts that did not progress
|
2
|
2
|
|
Period 1-First Treatment
Ineligible
|
0
|
2
|
|
Period 2-crossover
refused phase B
|
2
|
2
|
|
Period 2-crossover
Withdrawal by Subject
|
3
|
4
|
|
Period 2-crossover
Adverse Event
|
2
|
2
|
|
Period 2-crossover
non-adherence
|
1
|
0
|
|
Period 2-crossover
Pts that did not progress
|
2
|
3
|
Baseline Characteristics
R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas
Baseline characteristics by cohort
| Measure |
Phase A-Tipifarnib
n=31 Participants
Patients receive oral tipifarnib every 12 hours on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. These are the patients that started on tipifarnib only. This number does not reflect the total amount of patients that crossed over to placebo.
|
Phase B - Placebo
n=31 Participants
Patients receive oral placebo every 12 hours on days 1-21. Courses repeat as in arm I. These are the patients that started on placebo only. This number does not reflect the total amount of patients that crossed over to tipifarnib. Two of the 31 patients were deemed ineligible, thus 29 started placebo.
|
Total
n=62 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
28 Participants
n=5 Participants
|
31 Participants
n=7 Participants
|
59 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
10.2 years
STANDARD_DEVIATION 5.13 • n=5 Participants
|
9.8 years
STANDARD_DEVIATION 4.69 • n=7 Participants
|
10. years
STANDARD_DEVIATION 4.87 • n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
25 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
21 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
37 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
29 Participants
n=5 Participants
|
30 Participants
n=7 Participants
|
59 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
25 Participants
n=5 Participants
|
25 Participants
n=7 Participants
|
50 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Hispanic
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
31 Participants
n=5 Participants
|
31 Participants
n=7 Participants
|
62 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 8 yearsPopulation: phase A - 62 started and 2 were ineligible = 60 phase B - 43 started
Median time to progression is defined as a greater than or equal to 20% increase increase in the sum of the volume of all index lesions based on volumetric analysis utilizing magnetic resonance imaging (MRI).Start of phase A or phase B to time of progression.
Outcome measures
| Measure |
Phase A - Tipifarnib
n=60 Participants
Tipifarnib
|
Phase B - Placebo
n=43 Participants
Placebo
|
|---|---|---|
|
Median Time to Progression
Placebo
|
10.6 Months
Interval 8.1 to 18.3
|
14.5 Months
Interval 8.2 to 26.1
|
|
Median Time to Progression
Tipifarnib
|
19.2 Months
Interval 8.1 to 30.3
|
13.3 Months
Interval 8.0 to 19.6
|
PRIMARY outcome
Timeframe: 8 yearsPopulation: Adverse event data is in compliance with DSMB (Data Safety Monitoring Board).
Here are the number of participants with adverse events. For the detailed list of adverse events see the adverse event module.
Outcome measures
| Measure |
Phase A - Tipifarnib
n=60 Participants
Tipifarnib
|
Phase B - Placebo
Placebo
|
|---|---|---|
|
Number of Participants With Adverse Events
|
59 Participants
|
—
|
SECONDARY outcome
Timeframe: Baseline to pre cycle 4Population: Participants analyzed includes only those whose parents completed the IPI Scale at baseline and pre cycle 4 on phase A.
Parents of participants aged 6-18 years completed the Impact of Pediatric Illness (IPI) Scale about their child prior to the start of cycles 1, 4, 7, and 10 and then after every 6 cycles. The IPI Scale assesses QOL in 4 domains: adaptive behavior, emotional functioning, medical/physical status, and cognitive functioning. Responses to the 43 items are made on a 5-point Likert scale (1-5) ranging from "not al all" to "a lot". Higher mean scores indicate better QOL. Parent total scores for participants on placebo were compared with scores from participants receiving tipifarnib on phase A.
Outcome measures
| Measure |
Phase A - Tipifarnib
n=18 Participants
Tipifarnib
|
Phase B - Placebo
n=17 Participants
Placebo
|
|---|---|---|
|
Quality of Life (QOL)
Baseline
|
3.69 Total scores on a scale
Standard Deviation 0.43
|
3.70 Total scores on a scale
Standard Deviation 0.53
|
|
Quality of Life (QOL)
Pre cycle 4
|
3.91 Total scores on a scale
Standard Deviation 0.50
|
3.68 Total scores on a scale
Standard Deviation 0.53
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 8 yearsPopulation: Phase B - Placebo group is not shown because this outcome measure only applies to the Phase A - Tipifarnib group.
Median time to progression is defined as ≥20% increase in diameter based on volumetric analysis using the 1-dimensional RECIST method. Start of phase A or phase B to time of progression.
Outcome measures
| Measure |
Phase A - Tipifarnib
n=1 Participants
Tipifarnib
|
Phase B - Placebo
Placebo
|
|---|---|---|
|
Median Time to Progression Using the Conventional 1-Dimensional Response Evaluation Criteria in Solid Tumors (RECIST) Method
|
NA Months
TTP was not reached because only one patient met criteria for progressive disease (e.g. insufficient number of participants to calculate TTP per protocol).
|
—
|
OTHER_PRE_SPECIFIED outcome
Timeframe: 8 yearsPopulation: Phase B- Placebo group is not shown because this outcome measure only applies to the Phase A - Tipifarnib group.
Median time to progression is defined as ≥25% increase in area based on volumetric analysis using the 2-dimensional WHO solid tumor method.
Outcome measures
| Measure |
Phase A - Tipifarnib
n=60 Participants
Tipifarnib
|
Phase B - Placebo
Placebo
|
|---|---|---|
|
Median Time to Progression Using the 2-Dimensional World Health Organization (WHO) Solid Tumor Method
|
52.5 Months
Interval 36.3 to 52.5
|
—
|
Adverse Events
Tipifarnib & Placebo
Serious events: 9 serious events
Other events: 59 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Tipifarnib & Placebo
n=60 participants at risk
Patients receive oral tipifarnib every 12 hours on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Patients receive oral placebo every 12 hours on days 1-21. Courses repeat as in arm I.
|
|---|---|
|
Gastrointestinal disorders
Diarrhea
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Psychiatric disorders
Hallucinations
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Blood and lymphatic system disorders
Hypofibrinogenemia
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Blood and lymphatic system disorders
Neutropenia
|
6.7%
4/60 • Number of events 5 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Skin and subcutaneous tissue disorders
Rash
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Investigations
SGOT (AST)
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Investigations
SGPT (ALT)
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
Other adverse events
| Measure |
Tipifarnib & Placebo
n=60 participants at risk
Patients receive oral tipifarnib every 12 hours on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Patients receive oral placebo every 12 hours on days 1-21. Courses repeat as in arm I.
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
33.3%
20/60 • Number of events 26 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Metabolism and nutrition disorders
Alkaline phosphatase
|
3.3%
2/60 • Number of events 2 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Immune system disorders
Allergic rhinitis
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
3.3%
2/60 • Number of events 2 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Gastrointestinal disorders
Anorexia
|
16.7%
10/60 • Number of events 11 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Psychiatric disorders
Anxiety
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Metabolism and nutrition disorders
Bicarbonate
|
28.3%
17/60 • Number of events 21 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Hepatobiliary disorders
Bilirubin
|
13.3%
8/60 • Number of events 9 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Eye disorders
Blurred vision
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Injury, poisoning and procedural complications
Bruising
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
General disorders
Chest pain
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Gastrointestinal disorders
Constipation
|
11.7%
7/60 • Number of events 7 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
10.0%
6/60 • Number of events 6 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Investigations
Creatinine
|
5.0%
3/60 • Number of events 3 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Psychiatric disorders
Depression
|
3.3%
2/60 • Number of events 2 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Gastrointestinal disorders
Diarrhea
|
46.7%
28/60 • Number of events 33 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Nervous system disorders
Dizziness
|
3.3%
2/60 • Number of events 2 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Gastrointestinal disorders
Dry mouth
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
3.3%
2/60 • Number of events 2 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Gastrointestinal disorders
Dyspepsia
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Renal and urinary disorders
Dysuria
|
3.3%
2/60 • Number of events 2 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
General disorders
Edema
|
3.3%
2/60 • Number of events 2 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Vascular disorders
Flushing
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Psychiatric disorders
Hallucinations
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Nervous system disorders
Headache
|
18.3%
11/60 • Number of events 16 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Renal and urinary disorders
Hematuria
|
10.0%
6/60 • Number of events 7 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Blood and lymphatic system disorders
Hemoglobin
|
26.7%
16/60 • Number of events 19 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Blood and lymphatic system disorders
Hemorrhage/bleeding
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
8.3%
5/60 • Number of events 5 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
11.7%
7/60 • Number of events 7 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
13.3%
8/60 • Number of events 9 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
15.0%
9/60 • Number of events 10 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Metabolism and nutrition disorders
Hypernatremia
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Vascular disorders
Hypertension
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
15.0%
9/60 • Number of events 11 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
11.7%
7/60 • Number of events 8 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Investigations
Hypofribrinogenemia
|
8.3%
5/60 • Number of events 6 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
11.7%
7/60 • Number of events 8 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Metabolism and nutrition disorders
Hypokalemia
|
10.0%
6/60 • Number of events 6 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
5.0%
3/60 • Number of events 3 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Metabolism and nutrition disorders
Hyponatremia
|
15.0%
9/60 • Number of events 9 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
5.0%
3/60 • Number of events 3 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Psychiatric disorders
Insomnia
|
6.7%
4/60 • Number of events 4 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Investigations
Leukopenia
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Investigations
Lymphopenia
|
20.0%
12/60 • Number of events 14 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Psychiatric disorders
Mood alteration-anxiety
|
5.0%
3/60 • Number of events 3 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Psychiatric disorders
Mood alteration-depression
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Gastrointestinal disorders
Mouth dryness
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Gastrointestinal disorders
Nausea
|
50.0%
30/60 • Number of events 37 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Nervous system disorders
Neuropathy motor
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Nervous system disorders
Neuropathy sensory
|
3.3%
2/60 • Number of events 2 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Blood and lymphatic system disorders
Neutropenia
|
21.7%
13/60 • Number of events 16 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Ear and labyrinth disorders
Otalgia
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Musculoskeletal and connective tissue disorders
Pain (neck)
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Musculoskeletal and connective tissue disorders
Pain hands and feet
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Cardiac disorders
Palpitations
|
3.3%
2/60 • Number of events 2 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Psychiatric disorders
Personality/behavioral
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Infections and infestations
Pharyngitis
|
3.3%
2/60 • Number of events 2 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Investigations
Platelets
|
13.3%
8/60 • Number of events 9 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Investigations
Prolonged PT
|
43.3%
26/60 • Number of events 33 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Investigations
Prolonged PTT
|
41.7%
25/60 • Number of events 33 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Renal and urinary disorders
Proteinuria
|
20.0%
12/60 • Number of events 12 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
13.3%
8/60 • Number of events 8 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Skin and subcutaneous tissue disorders
Rash
|
23.3%
14/60 • Number of events 16 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Nervous system disorders
Seizure
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Investigations
SGOT (AST)
|
16.7%
10/60 • Number of events 13 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Investigations
SGPT (ALT)
|
13.3%
8/60 • Number of events 11 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Gastrointestinal disorders
Taste disturbance
|
3.3%
2/60 • Number of events 3 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Tumor pain
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Renal and urinary disorders
Urinary urgency
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Gastrointestinal disorders
Vomiting
|
40.0%
24/60 • Number of events 28 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Investigations
white blood cell (WBC)
|
38.3%
23/60 • Number of events 26 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Investigations
Weight loss
|
5.0%
3/60 • Number of events 3 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
General disorders
Fatigue
|
16.7%
10/60 • Number of events 12 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Investigations
Fever
|
3.3%
2/60 • Number of events 2 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Blood and lymphatic system disorders
Fibrinogen
|
11.7%
7/60 • Number of events 7 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
|
Vascular disorders
Facial flushing
|
1.7%
1/60 • Number of events 1 • 8 years
Adverse event data is in compliance with DSMB(Data Safety Monitoring Board).
|
Additional Information
Brigitte Widemann, M.D.
National Institutes of Health, National Cancer Institute
Phone: 301-496-7387
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place