Trial Outcomes & Findings for Everolimus for Cancer With TSC1 or TSC2 Mutation (NCT NCT02201212)

NCT ID: NCT02201212

Last Updated: 2020-09-16

Results Overview

RECIST 1.1 criteria for Objective Response: Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by scan: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 30 participants
• Primary outcome timeframe: Baseline, Every 8 weeks, 2 Years
• Results posted on: 2020-09-16

Participant Flow

Participant milestones

Measure	Everolimus Everolimus * Fixed doses orally once a day per each 28 day cycle * Participants will stay on study as long as they do not progress for a maximum of 24 months. * Tumor assessments will be performed after every 2 cycles for as long as they are on study. Everolimus
Overall Study STARTED	30
Overall Study COMPLETED	30
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Everolimus for Cancer With TSC1 or TSC2 Mutation

Baseline characteristics by cohort

Measure	Everolimus n=30 Participants Everolimus * Fixed doses orally once a day per each 28 day cycle * Participants will stay on study as long as they do not progress for a maximum of 24 months. * Tumor assessments will be performed after every 2 cycles for as long as they are on study. Everolimus
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	16 Participants n=5 Participants
Age, Categorical >=65 years	14 Participants n=5 Participants
Age, Continuous	61.5 years n=5 Participants
Sex: Female, Male Female	15 Participants n=5 Participants
Sex: Female, Male Male	15 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	2 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	0 Participants n=5 Participants
Race (NIH/OMB) White	25 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants
Region of Enrollment United States	30 participants n=5 Participants
ECOG Performance Status	1 units on a scale n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Every 8 weeks, 2 Years

RECIST 1.1 criteria for Objective Response:

Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) for target lesions and assessed by scan: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR

Outcome measures

Measure	Everolimus n=30 Participants Everolimus * Fixed doses orally once a day per each 28 day cycle * Participants will stay on study as long as they do not progress for a maximum of 24 months. * Tumor assessments will be performed after every 2 cycles for as long as they are on study. Everolimus
Objective Response Rate	2 Participants

SECONDARY outcome

Timeframe: Baseline, Every 8 weeks, 2 Years

Population: Subjects with Partial Response

Duration of Response Rate

Outcome measures

Measure	Everolimus n=2 Participants Everolimus * Fixed doses orally once a day per each 28 day cycle * Participants will stay on study as long as they do not progress for a maximum of 24 months. * Tumor assessments will be performed after every 2 cycles for as long as they are on study. Everolimus
Duration of Response	12.7 months Interval 4.8 to 20.7

SECONDARY outcome

Timeframe: Baseline, Up to 2 Years

Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions

Outcome measures

Measure	Everolimus n=30 Participants Everolimus * Fixed doses orally once a day per each 28 day cycle * Participants will stay on study as long as they do not progress for a maximum of 24 months. * Tumor assessments will be performed after every 2 cycles for as long as they are on study. Everolimus
Progression-free Survival	2.0 months Interval 0.4 to 20.7

SECONDARY outcome

Timeframe: 4 Years

Overall Survival Rate

Outcome measures

Measure	Everolimus n=30 Participants Everolimus * Fixed doses orally once a day per each 28 day cycle * Participants will stay on study as long as they do not progress for a maximum of 24 months. * Tumor assessments will be performed after every 2 cycles for as long as they are on study. Everolimus
Overall Survival	7.27 months Interval 0.43 to 39.93

SECONDARY outcome

Timeframe: 2 Years

CTCAE v4.0 Toxicity Rate Grade 3 or higher

Outcome measures

Measure	Everolimus n=30 Participants Everolimus * Fixed doses orally once a day per each 28 day cycle * Participants will stay on study as long as they do not progress for a maximum of 24 months. * Tumor assessments will be performed after every 2 cycles for as long as they are on study. Everolimus
Toxicity Rate	3 Participants

Adverse Events

Everolimus

Serious events: 3 serious events

Other events: 20 other events

Deaths: 1 deaths

Serious adverse events

Measure	Everolimus n=30 participants at risk Everolimus * Fixed doses orally once a day per each 28 day cycle * Participants will stay on study as long as they do not progress for a maximum of 24 months. * Tumor assessments will be performed after every 2 cycles for as long as they are on study. Everolimus
Respiratory, thoracic and mediastinal disorders Pneumonitis	6.7% 2/30 • Number of events 2 • 2 months after study discontinuation, up to 5 years National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE v4.0)
Blood and lymphatic system disorders Anemia	3.3% 1/30 • Number of events 1 • 2 months after study discontinuation, up to 5 years National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE v4.0)

Other adverse events

Measure	Everolimus n=30 participants at risk Everolimus * Fixed doses orally once a day per each 28 day cycle * Participants will stay on study as long as they do not progress for a maximum of 24 months. * Tumor assessments will be performed after every 2 cycles for as long as they are on study. Everolimus
Respiratory, thoracic and mediastinal disorders any adverse event per CTCAE v4.0	66.7% 20/30 • Number of events 100 • 2 months after study discontinuation, up to 5 years National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE v4.0)

Additional Information

Dr. David Kwiatkowski

Dana Farber Cancer Institute

Phone: 8573070781

Email: dk@rics.bwh.harvard.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place