Trial Outcomes & Findings for Study of Sutent®/Sunitinib (SU11248) in Subjects With NF-1 Plexiform Neurofibromas (NCT NCT01402817)

Last Updated: 2018-03-14

Results Overview

To estimate the disease control rate (SD, PR, CR) with Sutent® in patients with neurofibromas (NF1). Tumor response criteria are determined by changes in size using all 3 dimensional measurements: width (W), transvers (T) , and length (L) measurements. Partial Response: ≥20% decrease in the sum of the products of the three perpendicular diameters of all target lesions (up to 5), taking as reference the initial baseline measurements.Stable Disease (SD): Neither sufficient decrease in the sum of the products of the three perpendicular diameters of all target lesions to qualify for PR (taking as reference the initial baseline measurements), nor sufficient increase in a single target lesions to qualify for PD, (taking as reference the smallest disease measurement since the treatment started).Progressive Disease (PD): 40% or more increase in the product of perpendicular diameters of ANY target lesion, taking as reference the smallest product observed.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

19 participants

Primary outcome timeframe

6 months

Results posted on

2018-03-14

Participant Flow

Participant milestones

Participant milestones
Measure	Sutent®/Sunitinib Upon enrollment, subjects will receive Sutent® orally. Adults (Age \>18) will receive 25mg. Children will receive 10mg/m2/day. All subjects will take the daily dose for 28 days followed by a 14 day rest period. If subjects tolerate the initial dose, adults will be increased to 37.5mg and children will be increased to 15mg/m2/day. Again, subjects will take that dose for 28 days followed by a rest period of 14 days. Adults who tolerate the increase will go up to the maximum dose of 50mg.The maximum dose for children is 15mg/m2/day.
Overall Study STARTED	19
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	11

Reasons for withdrawal

Reasons for withdrawal
Measure	Sutent®/Sunitinib Upon enrollment, subjects will receive Sutent® orally. Adults (Age \>18) will receive 25mg. Children will receive 10mg/m2/day. All subjects will take the daily dose for 28 days followed by a 14 day rest period. If subjects tolerate the initial dose, adults will be increased to 37.5mg and children will be increased to 15mg/m2/day. Again, subjects will take that dose for 28 days followed by a rest period of 14 days. Adults who tolerate the increase will go up to the maximum dose of 50mg.The maximum dose for children is 15mg/m2/day.
Overall Study Withdrawal by Subject	3
Overall Study Study Suspended by FDA	8

Baseline Characteristics

Study of Sutent®/Sunitinib (SU11248) in Subjects With NF-1 Plexiform Neurofibromas

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Sutent®/Sunitinib n=19 Participants Upon enrollment, subjects will receive Sutent® orally. Adults (Age \>18) will receive 25mg. Children will receive 10mg/m2/day. All subjects will take the daily dose for 28 days followed by a 14 day rest period. If subjects tolerate the initial dose, adults will be increased to 37.5mg and children will be increased to 15mg/m2/day. Again, subjects will take that dose for 28 days followed by a rest period of 14 days. Adults who tolerate the increase will go up to the maximum dose of 50mg.The maximum dose for children is 15mg/m2/day.
Age, Categorical <=18 years	15 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	11 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	19 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 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	1 Participants n=5 Participants
Race (NIH/OMB) White	15 Participants n=5 Participants
Race (NIH/OMB) More than one race	2 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	19 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Outcome measures
Measure	Sutent®/Sunitinib n=19 Participants Upon enrollment, subjects will receive Sutent® orally. Adults (Age \>18) will receive 25mg. Children will receive 10mg/m2/day. All subjects will take the daily dose for 28 days followed by a 14 day rest period. If subjects tolerate the initial dose, adults will be increased to 37.5mg and children will be increased to 15mg/m2/day. Adults who tolerate the increase will go up to the maximum dose of 50mg. The maximum dose for children is 15mg/m2/day.
Disease Response	52 percentage of subjects Interval 39.21 to 77.73

SECONDARY outcome

Timeframe: 6 months

To determine the response rate with Sutent® in patients with plexiform neurofibromas using volumetric analysis of MRI scans

Outcome measures

Outcome measures
Measure	Sutent®/Sunitinib n=19 Participants Upon enrollment, subjects will receive Sutent® orally. Adults (Age \>18) will receive 25mg. Children will receive 10mg/m2/day. All subjects will take the daily dose for 28 days followed by a 14 day rest period. If subjects tolerate the initial dose, adults will be increased to 37.5mg and children will be increased to 15mg/m2/day. Adults who tolerate the increase will go up to the maximum dose of 50mg. The maximum dose for children is 15mg/m2/day.
Volumetric Disease Evaluation	0 percentage of subjects

Adverse Events

Sutent®/Sunitinib

Serious events: 3 serious events

Other events: 19 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Sutent®/Sunitinib n=19 participants at risk Upon enrollment, subjects will receive Sutent® orally. Adults (Age \>18) will receive 25mg. Children will receive 10mg/m2/day. All subjects will take the daily dose for 28 days followed by a 14 day rest period. If subjects tolerate the initial dose, adults will be increased to 37.5mg and children will be increased to 15mg/m2/day. Again, subjects will take that dose for 28 days followed by a rest period of 14 days. Adults who tolerate the increase will go up to the maximum dose of 50mg.The maximum dose for children is 15mg/m2/day.
Nervous system disorders seizure	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Injury, poisoning and procedural complications wound dehiscence	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Cardiac disorders cardiac arrest	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy

Other adverse events

Other adverse events
Measure	Sutent®/Sunitinib n=19 participants at risk Upon enrollment, subjects will receive Sutent® orally. Adults (Age \>18) will receive 25mg. Children will receive 10mg/m2/day. All subjects will take the daily dose for 28 days followed by a 14 day rest period. If subjects tolerate the initial dose, adults will be increased to 37.5mg and children will be increased to 15mg/m2/day. Again, subjects will take that dose for 28 days followed by a rest period of 14 days. Adults who tolerate the increase will go up to the maximum dose of 50mg.The maximum dose for children is 15mg/m2/day.
Metabolism and nutrition disorders Acidosis	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Gastrointestinal disorders Abdominal pain	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Investigations alanine aminotransferase increased	5.3% 1/19 • Number of events 2 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Investigations alkaline phosphatase increased	10.5% 2/19 • Number of events 3 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Blood and lymphatic system disorders Anemia	26.3% 5/19 • Number of events 9 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Musculoskeletal and connective tissue disorders Back pain	5.3% 1/19 • Number of events 2 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Investigations blood bilirubin increased	15.8% 3/19 • Number of events 4 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Nervous system disorders cognitive disturbance	5.3% 1/19 • Number of events 2 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Musculoskeletal and connective tissue disorders chest wall pain	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Investigations CPK increased	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Gastrointestinal disorders constipation	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Psychiatric disorders depression	10.5% 2/19 • Number of events 2 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Gastrointestinal disorders diarrhea	10.5% 2/19 • Number of events 2 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Ear and labyrinth disorders Ear and labyrinth disorders	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Cardiac disorders Electrocardiogram QT corrected interval prolonged	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Gastrointestinal disorders Esophagitis	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
General disorders fatigue	21.1% 4/19 • Number of events 4 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Gastrointestinal disorders Gastritis	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Musculoskeletal and connective tissue disorders gait disturbance	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Nervous system disorders headache	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Investigations Hypercalcemia	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Cardiac disorders Hypertension	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Investigations Hyperuricemia	10.5% 2/19 • Number of events 4 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Investigations Hypoalbuminemia	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Investigations hypocalcemia	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Investigations hypokalemia	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Investigations Hpomagnesemia	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Investigations Hypophoshatemia	10.5% 2/19 • Number of events 2 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Psychiatric disorders Insomnia	10.5% 2/19 • Number of events 2 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Investigations Lipase increased	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Investigations Lymphocyte count decreased	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Gastrointestinal disorders nausea	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Investigations neutrophil count decreased	47.4% 9/19 • Number of events 11 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Musculoskeletal and connective tissue disorders neck pain	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
General disorders pain	21.1% 4/19 • Number of events 4 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Skin and subcutaneous tissue disorders palmar-plantar erythrodysesthesia syndrome	10.5% 2/19 • Number of events 2 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
General disorders pain in extremity	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Blood and lymphatic system disorders platelet count decreased	26.3% 5/19 • Number of events 5 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Skin and subcutaneous tissue disorders Rash aceniform	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Musculoskeletal and connective tissue disorders scoliosis	10.5% 2/19 • Number of events 2 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Investigations serum amylase increased	10.5% 2/19 • Number of events 3 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Infections and infestations sinusitis	5.3% 1/19 • Number of events 1 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Renal and urinary disorders urinary tract infection	5.3% 1/19 • Number of events 3 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Gastrointestinal disorders vomiting	10.5% 2/19 • Number of events 3 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
Investigations white blood cell decreased	68.4% 13/19 • Number of events 13 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy
General disorders generalized muscle weakness	15.8% 3/19 • Number of events 3 • Adverse events were collected for the duration of the time subjects were taking drug and 30 days following completion of therapy

Additional Information

Chie-Schin Shih, MD

Indiana University School of Medicine - Pediatrics

Phone: 317-948-8568

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place