Trial Outcomes & Findings for Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas (NCT NCT03231306)
NCT ID: NCT03231306
Last Updated: 2025-05-02
Results Overview
To determine the objective response defined as 20% or greater tumor volume reduction. Patients will undergo volumetric assays of their target PN using MRI.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
45 participants
Primary outcome timeframe
Approximately 12 months
Results posted on
2025-05-02
Participant Flow
Participant milestones
| Measure |
Open Label Study of Binimetinib (MEK162)
Subjects (≥ 18 years) (Stratum A) will receive a course of binimetinib by mouth twice a day (12 hours apart) of 45 mg/dose. Duration of each course is 4 weeks. After 8 courses, subjects will receive additional courses if MRI results showed at least 15% reduction in volume of the target tumor. Subjects can continue on therapy and will be evaluated at the end of 12 courses. Subjects who have ≥ 20% reduction in volume of the target tumor according to the MRI results can continue therapy up to an additional year (maximum of 24 total courses). Subjects who have not met the tumor reduction at the specified times will be removed from the study therapy. Subjects will be carefully monitored for toxicities associated with binimetinib.
Recruitment of subjects 1 - 17 years of age (Stratum B) is currently available. The pediatric maximum tolerated dose (MTD) of binimetinib the pediatric patients (Statum B) was established by a phase 1 study (NCT022).
Binimetinib: Adult subjects (18 years and older) will receive binimetinib by mouth twice daily of 45mg/dose. Pediatric subjects (1-17 years of age) are being treated on the pediatric MTD established by a phase I study (NCT02285439).
|
|---|---|
|
Overall Study
STARTED
|
45
|
|
Overall Study
COMPLETED
|
19
|
|
Overall Study
NOT COMPLETED
|
26
|
Reasons for withdrawal
| Measure |
Open Label Study of Binimetinib (MEK162)
Subjects (≥ 18 years) (Stratum A) will receive a course of binimetinib by mouth twice a day (12 hours apart) of 45 mg/dose. Duration of each course is 4 weeks. After 8 courses, subjects will receive additional courses if MRI results showed at least 15% reduction in volume of the target tumor. Subjects can continue on therapy and will be evaluated at the end of 12 courses. Subjects who have ≥ 20% reduction in volume of the target tumor according to the MRI results can continue therapy up to an additional year (maximum of 24 total courses). Subjects who have not met the tumor reduction at the specified times will be removed from the study therapy. Subjects will be carefully monitored for toxicities associated with binimetinib.
Recruitment of subjects 1 - 17 years of age (Stratum B) is currently available. The pediatric maximum tolerated dose (MTD) of binimetinib the pediatric patients (Statum B) was established by a phase 1 study (NCT022).
Binimetinib: Adult subjects (18 years and older) will receive binimetinib by mouth twice daily of 45mg/dose. Pediatric subjects (1-17 years of age) are being treated on the pediatric MTD established by a phase I study (NCT02285439).
|
|---|---|
|
Overall Study
<15% reduction in target tumor volume from baseline after Course 8 of therapy
|
3
|
|
Overall Study
<20% reduction in target tumor volume from baseline after Course 12 of therapy
|
4
|
|
Overall Study
Treatment delay > 21 days
|
2
|
|
Overall Study
Refusal of further protocol therapy by subject/parent/guardian
|
6
|
|
Overall Study
Non-compliance that in the opinion of the investigator does not allow for ongoing participation
|
5
|
|
Overall Study
Binimetinib-related toxicity requiring removal from protocol therapy
|
3
|
|
Overall Study
Subject Relocation Due to Covid Restrictions
|
1
|
|
Overall Study
More than 1 Reason
|
2
|
Baseline Characteristics
Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform Neurofibromas
Baseline characteristics by cohort
| Measure |
Open Label Study of Binimetinib (MEK162)
n=45 Participants
Subjects (≥ 18 years) (Stratum A) will receive a course of binimetinib by mouth twice a day (12 hours apart) of 45 mg/dose. Duration of each course is 4 weeks. After 8 courses, subjects will receive additional courses if MRI results showed at least 15% reduction in volume of the target tumor. Subjects can continue on therapy and will be evaluated at the end of 12 courses. Subjects who have ≥ 20% reduction in volume of the target tumor according to the MRI results can continue therapy up to an additional year (maximum of 24 total courses). Subjects who have not met the tumor reduction at the specified times will be removed from the study therapy. Subjects will be carefully monitored for toxicities associated with binimetinib.
Recruitment of subjects 1 - 17 years of age (Stratum B) is currently available. The pediatric maximum tolerated dose (MTD) of binimetinib the pediatric patients (Statum B) was established by a phase 1 study (NCT022).
Binimetinib: Adult subjects (18 years and older) will receive binimetinib by mouth twice daily of 45mg/dose. Pediatric subjects (1-17 years of age) are being treated on the pediatric MTD established by a phase I study (NCT02285439).
|
|---|---|
|
Age, Continuous
|
19 Years
n=5 Participants
|
|
Sex: Female, Male
Female
|
22 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
23 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
39 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
|
28 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
10 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
45 participants
n=5 Participants
|
|
Number of Participants
|
45 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Approximately 12 monthsTo determine the objective response defined as 20% or greater tumor volume reduction. Patients will undergo volumetric assays of their target PN using MRI.
Outcome measures
| Measure |
Open Label Study of Binimetinib (MEK162)
n=44 Participants
Subjects (≥ 18 years) (Stratum A) will receive a course of binimetinib by mouth twice a day (12 hours apart) of 45 mg/dose. Duration of each course is 4 weeks. After 8 courses, subjects will receive additional courses if MRI results showed at least 15% reduction in volume of the target tumor. Subjects can continue on therapy and will be evaluated at the end of 12 courses. Subjects who have ≥ 20% reduction in volume of the target tumor according to the MRI results can continue therapy up to an additional year (maximum of 24 total courses). Subjects who have not met the tumor reduction at the specified times will be removed from the study therapy. Subjects will be carefully monitored for toxicities associated with binimetinib.
Recruitment of subjects 1 - 17 years of age (Stratum B) is currently available. The pediatric maximum tolerated dose (MTD) of binimetinib the pediatric patients (Statum B) was established by a phase 1 study (NCT022).
Binimetinib: Adult subjects (18 years and older) will receive binimetinib by mouth twice daily of 45mg/dose. Pediatric subjects (1-17 years of age) are being treated on the pediatric MTD established by a phase I study (NCT02285439).
|
|---|---|
|
Change From Baseline Target Tumor Volume at 12 Months
Did Not Meet Objective Response
|
17 Participants
|
|
Change From Baseline Target Tumor Volume at 12 Months
Met Objective Response
|
27 Participants
|
SECONDARY outcome
Timeframe: Up to 24 monthsTo evaluate the toxicity of protracted binimetinib administration in this patient population. Subjects will be monitored continuously for adverse events and serious adverse events throughout the study.
Outcome measures
| Measure |
Open Label Study of Binimetinib (MEK162)
n=45 Participants
Subjects (≥ 18 years) (Stratum A) will receive a course of binimetinib by mouth twice a day (12 hours apart) of 45 mg/dose. Duration of each course is 4 weeks. After 8 courses, subjects will receive additional courses if MRI results showed at least 15% reduction in volume of the target tumor. Subjects can continue on therapy and will be evaluated at the end of 12 courses. Subjects who have ≥ 20% reduction in volume of the target tumor according to the MRI results can continue therapy up to an additional year (maximum of 24 total courses). Subjects who have not met the tumor reduction at the specified times will be removed from the study therapy. Subjects will be carefully monitored for toxicities associated with binimetinib.
Recruitment of subjects 1 - 17 years of age (Stratum B) is currently available. The pediatric maximum tolerated dose (MTD) of binimetinib the pediatric patients (Statum B) was established by a phase 1 study (NCT022).
Binimetinib: Adult subjects (18 years and older) will receive binimetinib by mouth twice daily of 45mg/dose. Pediatric subjects (1-17 years of age) are being treated on the pediatric MTD established by a phase I study (NCT02285439).
|
|---|---|
|
Incidence of Treatment-Emergent Adverse Events
|
45 Participants
|
Adverse Events
Open Label Study of Binimetinib (MEK162)
Serious events: 11 serious events
Other events: 45 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Open Label Study of Binimetinib (MEK162)
n=45 participants at risk
Subjects (≥ 18 years) (Stratum A) will receive a course of binimetinib by mouth twice a day (12 hours apart) of 45 mg/dose. Duration of each course is 4 weeks. After 8 courses, subjects will receive additional courses if MRI results showed at least 15% reduction in volume of the target tumor. Subjects can continue on therapy and will be evaluated at the end of 12 courses. Subjects who have ≥ 20% reduction in volume of the target tumor according to the MRI results can continue therapy up to an additional year (maximum of 24 total courses). Subjects who have not met the tumor reduction at the specified times will be removed from the study therapy. Subjects will be carefully monitored for toxicities associated with binimetinib.
Recruitment of subjects 1 - 17 years of age (Stratum B) is currently available. The pediatric maximum tolerated dose (MTD) of binimetinib the pediatric patients (Statum B) was established by a phase 1 study (NCT022).
Binimetinib: Adult subjects (18 years and older) will receive binimetinib by mouth twice daily of 45mg/dose. Pediatric subjects (1-17 years of age) are being treated on the pediatric MTD established by a phase I study (NCT02285439).
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Gastrointestinal disorders
Abdominal pain
|
2.2%
1/45 • Number of events 5 • 24 Months
|
|
Gastrointestinal disorders
Diarrhea
|
2.2%
1/45 • Number of events 2 • 24 Months
|
|
Gastrointestinal disorders
Gastric Hemorrhage
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Gastrointestinal disorders
Nausea
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Gastrointestinal disorders
Vomiting
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
General disorders
Fever
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Immune system disorders
Anaphylaxis
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Infections and infestations
Appendicitis
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Infections and infestations
Skin Infection
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Metabolism and nutrition disorders
Weight Loss
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Psychiatric disorders
Suicide attempt
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Renal and urinary disorders
Hematuria
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
2.2%
1/45 • Number of events 1 • 24 Months
|
Other adverse events
| Measure |
Open Label Study of Binimetinib (MEK162)
n=45 participants at risk
Subjects (≥ 18 years) (Stratum A) will receive a course of binimetinib by mouth twice a day (12 hours apart) of 45 mg/dose. Duration of each course is 4 weeks. After 8 courses, subjects will receive additional courses if MRI results showed at least 15% reduction in volume of the target tumor. Subjects can continue on therapy and will be evaluated at the end of 12 courses. Subjects who have ≥ 20% reduction in volume of the target tumor according to the MRI results can continue therapy up to an additional year (maximum of 24 total courses). Subjects who have not met the tumor reduction at the specified times will be removed from the study therapy. Subjects will be carefully monitored for toxicities associated with binimetinib.
Recruitment of subjects 1 - 17 years of age (Stratum B) is currently available. The pediatric maximum tolerated dose (MTD) of binimetinib the pediatric patients (Statum B) was established by a phase 1 study (NCT022).
Binimetinib: Adult subjects (18 years and older) will receive binimetinib by mouth twice daily of 45mg/dose. Pediatric subjects (1-17 years of age) are being treated on the pediatric MTD established by a phase I study (NCT02285439).
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
35.6%
16/45 • Number of events 36 • 24 Months
|
|
Cardiac disorders
Cardiac disorders - Other, Non-Specific St And T-Wave Abnormalities
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Cardiac disorders
Cardiac disorders - Other, Nonspecific T Wave Abnormality
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Cardiac disorders
Cardiac disorders - Other, Specify, Electrocardiogram T Wave Abnormal
|
4.4%
2/45 • Number of events 2 • 24 Months
|
|
Cardiac disorders
Cardiac disorders - Other, Systolic Murmur
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Cardiac disorders
Cardiac disorders - Other, Tachycardia
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Cardiac disorders
Conduction disorder
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Cardiac disorders
Pulmonary Valve Disease
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Cardiac disorders
Sinus bradycardia
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Cardiac disorders
Sinus tachycardia
|
8.9%
4/45 • Number of events 8 • 24 Months
|
|
Cardiac disorders
Ventricular Arrhythmia
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Ear and labyrinth disorders
Ear and labyrinth disorders - Other, Mastoid Effusion
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Ear and labyrinth disorders
External Ear Inflammation
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Endocrine disorders
Endocrine disorders - Other, Increased Intolerance To Heat And Humidity
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Eye disorders
Blurred Vision
|
4.4%
2/45 • Number of events 3 • 24 Months
|
|
Eye disorders
Conjunctivitis
|
4.4%
2/45 • Number of events 2 • 24 Months
|
|
Ear and labyrinth disorders
Corneal ulcer
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Eye disorders
Dry Eye
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Eye disorders
Eye disorders - Other, Blepharitis
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Eye disorders
Eye disorders - Other, Central Lenticular Opacities
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Eye disorders
Eye disorders - Other, Erythema
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Eye disorders
Eye disorders - Other, Macular Edema
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Eye disorders
Eye disorders - Other, Optic Disk Swelling With Peripapillary Hemorrhages
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Eye disorders
Eye pain
|
2.2%
1/45 • Number of events 2 • 24 Months
|
|
Eye disorders
Floaters
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Eye disorders
Keratitis
|
4.4%
2/45 • Number of events 3 • 24 Months
|
|
Eye disorders
Scleral disorder
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Gastrointestinal disorders
Abdominal Distention
|
2.2%
1/45 • Number of events 2 • 24 Months
|
|
Gastrointestinal disorders
Abdominal Pain
|
28.9%
13/45 • Number of events 28 • 24 Months
|
|
Gastrointestinal disorders
Cheilitis
|
4.4%
2/45 • Number of events 3 • 24 Months
|
|
Gastrointestinal disorders
Constipation
|
22.2%
10/45 • Number of events 11 • 24 Months
|
|
Gastrointestinal disorders
Diarrhea
|
62.2%
28/45 • Number of events 77 • 24 Months
|
|
Gastrointestinal disorders
Dry Mouth
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Gastrointestinal disorders
Dyspepsia
|
11.1%
5/45 • Number of events 8 • 24 Months
|
|
Gastrointestinal disorders
Emesis
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Gastrointestinal disorders
Fecal Incontinence
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Gastrointestinal disorders
Flatulence
|
8.9%
4/45 • Number of events 4 • 24 Months
|
|
Gastrointestinal disorders
Gastric Hemorrhage
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Gastrointestinal disorders
Gastroesophageal Reflux Disease
|
4.4%
2/45 • Number of events 3 • 24 Months
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, Specify Tooth Loss
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, Tooth Pain
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, Cecal Volvulus
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Gastrointestinal disorders
Gastrointestinal disorders Gastrointestinal disorders - Other, Gastroenteritis
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, Mouth Sore
|
4.4%
2/45 • Number of events 2 • 24 Months
|
|
Gastrointestinal disorders
Gastrointestinal pain
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Gastrointestinal disorders
Hemorrhoidal Hemorrhage
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Gastrointestinal disorders
Hemorrhoids
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Gastrointestinal disorders
Mucositis oral
|
15.6%
7/45 • Number of events 15 • 24 Months
|
|
Gastrointestinal disorders
Nausea
|
60.0%
27/45 • Number of events 52 • 24 Months
|
|
Gastrointestinal disorders
Small Intestinal Obstruction
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Gastrointestinal disorders
Stomach Pain
|
8.9%
4/45 • Number of events 5 • 24 Months
|
|
Gastrointestinal disorders
Vomiting
|
51.1%
23/45 • Number of events 66 • 24 Months
|
|
General disorders
Chills
|
11.1%
5/45 • Number of events 5 • 24 Months
|
|
General disorders
Edema Face
|
15.6%
7/45 • Number of events 12 • 24 Months
|
|
General disorders
Edema Limbs
|
24.4%
11/45 • Number of events 26 • 24 Months
|
|
Gastrointestinal disorders
Edema Trunk
|
2.2%
1/45 • Number of events 2 • 24 Months
|
|
General disorders
Facial Pain
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
General disorders
Fatigue
|
46.7%
21/45 • Number of events 36 • 24 Months
|
|
General disorders
Fever
|
24.4%
11/45 • Number of events 21 • 24 Months
|
|
General disorders
Flu Like Symptoms
|
6.7%
3/45 • Number of events 3 • 24 Months
|
|
General disorders
General Disorders and Administration Site Conditions - Other, Bleeding Gums
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
General disorders
Irritability
|
4.4%
2/45 • Number of events 3 • 24 Months
|
|
General disorders
Localized Edema
|
6.7%
3/45 • Number of events 10 • 24 Months
|
|
General disorders
Malaise
|
4.4%
2/45 • Number of events 2 • 24 Months
|
|
General disorders
Non-Cardiac Chest Pain
|
8.9%
4/45 • Number of events 5 • 24 Months
|
|
General disorders
Pain
|
6.7%
3/45 • Number of events 3 • 24 Months
|
|
Hepatobiliary disorders
Cholecystitis
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Immune system disorders
Allergic Reaction
|
4.4%
2/45 • Number of events 3 • 24 Months
|
|
Infections and infestations
Eye Infection
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Infections and infestations
Infections And Infestations, Other, Folliculitis
|
2.2%
1/45 • Number of events 2 • 24 Months
|
|
Infections and infestations
Infections and infestations - Other, BK Virus
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Infections and infestations
Infections and infestations - Other, Covid-19
|
4.4%
2/45 • Number of events 2 • 24 Months
|
|
Infections and infestations
Infections and infestations - Other, Gastroenteritis
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Infections and infestations
Nail Infection
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Infections and infestations
Otitis Externa
|
6.7%
3/45 • Number of events 3 • 24 Months
|
|
Infections and infestations
Otitis Media
|
6.7%
3/45 • Number of events 3 • 24 Months
|
|
Infections and infestations
Papulopustular rash
|
8.9%
4/45 • Number of events 4 • 24 Months
|
|
Infections and infestations
Paronychia
|
40.0%
18/45 • Number of events 52 • 24 Months
|
|
Infections and infestations
Pharyngitis
|
4.4%
2/45 • Number of events 2 • 24 Months
|
|
Infections and infestations
Rash pustular
|
2.2%
1/45 • Number of events 2 • 24 Months
|
|
Infections and infestations
Sinusitis
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Infections and infestations
Skin Infection
|
28.9%
13/45 • Number of events 21 • 24 Months
|
|
Infections and infestations
Upper Respiratory Infection
|
8.9%
4/45 • Number of events 4 • 24 Months
|
|
Infections and infestations
Urinary Tract Infection
|
4.4%
2/45 • Number of events 3 • 24 Months
|
|
Infections and infestations
Vaginal Infection
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Injury, poisoning and procedural complications
Bruising
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Injury, poisoning and procedural complications
Burn
|
2.2%
1/45 • Number of events 2 • 24 Months
|
|
Injury, poisoning and procedural complications
Wound Complication
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Investigations
Alanine Aminotransferase Increased
|
26.7%
12/45 • Number of events 32 • 24 Months
|
|
Investigations
Alkaline Phosphatase Increased
|
8.9%
4/45 • Number of events 6 • 24 Months
|
|
Investigations
Aspartate Aminotransferase Increased
|
51.1%
23/45 • Number of events 43 • 24 Months
|
|
Investigations
Blood bilirubin increased
|
8.9%
4/45 • Number of events 8 • 24 Months
|
|
Investigations
CPK increased
|
73.3%
33/45 • Number of events 96 • 24 Months
|
|
Investigations
Creatinine Increased
|
4.4%
2/45 • Number of events 5 • 24 Months
|
|
Investigations
Ejection fraction decreased
|
6.7%
3/45 • Number of events 4 • 24 Months
|
|
Investigations
Electrocardiogram Qt Corrected Interval Prolonged
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Investigations
Growth Hormone Abnormal
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Investigations
Hemoglobin Increased
|
2.2%
1/45 • Number of events 2 • 24 Months
|
|
Investigations
Investigations - Other, Absolute Lymphocyte Count Increased
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Investigations
Investigations - Other, Absolute Neutrophil Count Decreased
|
6.7%
3/45 • Number of events 5 • 24 Months
|
|
Investigations
Investigations - Other, Creatine Kinase Increased
|
2.2%
1/45 • Number of events 2 • 24 Months
|
|
Investigations
Investigations - Other, Lactate Dehydrogenase Increased
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Investigations
Investigations - Other, Partial Thromboplastin Time Prolonged
|
2.2%
1/45 • Number of events 2 • 24 Months
|
|
Investigations
Lipase Increased
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Investigations
Lymphocyte Count Decreased
|
8.9%
4/45 • Number of events 6 • 24 Months
|
|
Investigations
Neutrophil Count Decreased
|
15.6%
7/45 • Number of events 13 • 24 Months
|
|
Investigations
Platelet Count Decreased
|
11.1%
5/45 • Number of events 9 • 24 Months
|
|
Investigations
Weight Gain
|
17.8%
8/45 • Number of events 22 • 24 Months
|
|
Investigations
Weight Loss
|
6.7%
3/45 • Number of events 7 • 24 Months
|
|
Investigations
White Blood Cell Decreased
|
15.6%
7/45 • Number of events 11 • 24 Months
|
|
Metabolism and nutrition disorders
Anorexia
|
13.3%
6/45 • Number of events 7 • 24 Months
|
|
Metabolism and nutrition disorders
Dehydration
|
13.3%
6/45 • Number of events 7 • 24 Months
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
4.4%
2/45 • Number of events 2 • 24 Months
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
6.7%
3/45 • Number of events 6 • 24 Months
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
11.1%
5/45 • Number of events 10 • 24 Months
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Metabolism and nutrition disorders
Hypernatremia
|
6.7%
3/45 • Number of events 3 • 24 Months
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
26.7%
12/45 • Number of events 23 • 24 Months
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
15.6%
7/45 • Number of events 13 • 24 Months
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
8.9%
4/45 • Number of events 12 • 24 Months
|
|
Metabolism and nutrition disorders
Hypokalemia
|
15.6%
7/45 • Number of events 16 • 24 Months
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
6.7%
3/45 • Number of events 4 • 24 Months
|
|
Metabolism and nutrition disorders
Hyponatremia
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
8.9%
4/45 • Number of events 5 • 24 Months
|
|
Metabolism and nutrition disorders
Metabolism And Nutrition Disorders - Other, Decreased Appetite
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Metabolism and nutrition disorders
Metabolism And Nutrition Disorders - Other, Hyperphosphatemia
|
6.7%
3/45 • Number of events 5 • 24 Months
|
|
Metabolism and nutrition disorders
Metabolism And Nutrition Disorders - Other, Total Protein Decreased
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
4.4%
2/45 • Number of events 2 • 24 Months
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
8.9%
4/45 • Number of events 5 • 24 Months
|
|
Musculoskeletal and connective tissue disorders
Bone Pain
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Musculoskeletal and connective tissue disorders
Generalized Muscle Weakness
|
11.1%
5/45 • Number of events 9 • 24 Months
|
|
Musculoskeletal and connective tissue disorders
Lordosis
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness trunk
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal And Connective Tissue Disorder - Other, Pain Head/Neck/Extremities
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal And Connective Tissue Disorders - Other, Leg Cramps
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorders - Other, Joint Pain
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
15.6%
7/45 • Number of events 9 • 24 Months
|
|
Musculoskeletal and connective tissue disorders
Neck Pain
|
6.7%
3/45 • Number of events 3 • 24 Months
|
|
Musculoskeletal and connective tissue disorders
Pain In Extremity
|
11.1%
5/45 • Number of events 6 • 24 Months
|
|
Nervous system disorders
Cognitive Disturbance
|
4.4%
2/45 • Number of events 2 • 24 Months
|
|
Nervous system disorders
Dizziness
|
20.0%
9/45 • Number of events 12 • 24 Months
|
|
Nervous system disorders
Dysgeusia
|
4.4%
2/45 • Number of events 2 • 24 Months
|
|
Nervous system disorders
Headache
|
24.4%
11/45 • Number of events 18 • 24 Months
|
|
Nervous system disorders
Memory Impairment
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Nervous system disorders
Paresthesia
|
6.7%
3/45 • Number of events 3 • 24 Months
|
|
Nervous system disorders
Presyncope
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Nervous system disorders
Seizure
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Nervous system disorders
Syncope
|
4.4%
2/45 • Number of events 2 • 24 Months
|
|
Psychiatric disorders
Agitation
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Psychiatric disorders
Anxiety
|
6.7%
3/45 • Number of events 3 • 24 Months
|
|
Psychiatric disorders
Depression
|
8.9%
4/45 • Number of events 5 • 24 Months
|
|
Psychiatric disorders
Insomnia
|
13.3%
6/45 • Number of events 6 • 24 Months
|
|
Renal and urinary disorders
Cystitis Noninfective
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Renal and urinary disorders
Hematuria
|
8.9%
4/45 • Number of events 4 • 24 Months
|
|
Renal and urinary disorders
Proteinuria
|
4.4%
2/45 • Number of events 3 • 24 Months
|
|
Renal and urinary disorders
Renal And Urinary Disorders - Other, Dysuria
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Renal and urinary disorders
Renal And Urinary Disorders - Other, Elevated Bun
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Renal and urinary disorders
Renal And Urinary Disorders - Other, Weak Urine Stream
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Renal and urinary disorders
Urinary Incontinence
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Renal and urinary disorders
Urinary Retention
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Renal and urinary disorders
Urinary Urgency
|
4.4%
2/45 • Number of events 5 • 24 Months
|
|
Reproductive system and breast disorders
Menorrhagia
|
2.2%
1/45 • Number of events 2 • 24 Months
|
|
Reproductive system and breast disorders
Reproductive System And Breast Disorders - Other, Breast Lump
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
11.1%
5/45 • Number of events 8 • 24 Months
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
13.3%
6/45 • Number of events 8 • 24 Months
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Respiratory, thoracic and mediastinal disorders
Laryngeal Inflammation
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Respiratory, thoracic and mediastinal disorders
Laryngeal Obstruction
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
4.4%
2/45 • Number of events 4 • 24 Months
|
|
Respiratory, thoracic and mediastinal disorders
Postnasal Drip
|
4.4%
2/45 • Number of events 3 • 24 Months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, Thoracic And Mediastinal Disorders - Other, Rhinorrhea
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Respiratory, thoracic and mediastinal disorders
Sore Throat
|
8.9%
4/45 • Number of events 6 • 24 Months
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
33.3%
15/45 • Number of events 19 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Dry Skin
|
42.2%
19/45 • Number of events 28 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Erythema Multiforme
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Palmar-Plantar Erythrodysesthesia Syndrome
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Periorbital Edema
|
6.7%
3/45 • Number of events 3 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Photosensitivity
|
6.7%
3/45 • Number of events 3 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
20.0%
9/45 • Number of events 12 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Purpura
|
2.2%
1/45 • Number of events 3 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
84.4%
38/45 • Number of events 53 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
31.1%
14/45 • Number of events 22 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Scalp Pain
|
4.4%
2/45 • Number of events 3 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Skin Atrophy
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, Achromotrichia
|
4.4%
2/45 • Number of events 2 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, Diaper Dermatitis
|
2.2%
1/45 • Number of events 5 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, Eczema
|
4.4%
2/45 • Number of events 2 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, Erythemous Rash
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, Hair Color Changes
|
8.9%
4/45 • Number of events 4 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, Insect Bites
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, Mottled Skin
|
4.4%
2/45 • Number of events 2 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, Papulopustular Rash
|
4.4%
2/45 • Number of events 3 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, Petechiae
|
2.2%
1/45 • Number of events 2 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, Seborrheic Dermatitis
|
11.1%
5/45 • Number of events 12 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, Sore on Foot
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, Sunburn
|
2.2%
1/45 • Number of events 1 • 24 Months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, Unspecified Rash
|
4.4%
2/45 • Number of events 3 • 24 Months
|
|
Vascular disorders
Hypertension
|
15.6%
7/45 • Number of events 10 • 24 Months
|
|
Vascular disorders
Hypotension
|
2.2%
1/45 • Number of events 4 • 24 Months
|
Additional Information
Coretta Thomas, Scientist & Data Manager for NF Consortium
UAB
Phone: 2059758629
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place