Trial Outcomes & Findings for Everolimus Therapy in People With Birt-Hogg-Dube Syndrome (BHD)-Associated Kidney Cancer or Sporadic Chromophobe Renal Cancer (NCT NCT02504892)

Last Updated: 2019-03-22

Results Overview

Overall best response is defined as the best response recorded from the start of the treatment until disease progression/recurrence (taking as reference for progressive disease the smallest measurements recorded since the treatment started). Response was assessed by the Response Evaluation in Solid Tumors (RECIST) criteria v1.1. Progressive disease is at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes baseline sum if that is the smallest on study). The sum must also demonstrate an absolute increase of at least 5mm. Note: the appearance of one or more new lesions is also progression. Complete response is disappearance of all target lesions. Partial response is at least a 30% decrease in the sum of the diameters of target lesions. Stable disease is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum of diameters while on study.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

End of treatment: every 12 weeks up to 1 year

Results posted on

2019-03-22

Participant Flow

The Sporadic chromophobe renal tumors arm/group is not included here and throughout because the protocol was amended to remove the Sporadic chromophobe renal tumors arm/group. The study was unable to enroll participants with this disease.

Participant milestones

Participant milestones
Measure	Birt-Hogg-Dube Syndrome Birt-Hogg-Dube Syndrome (BHD)-associated renal tumors Everolimus: Everolimus is a commercial agent and is supplied by Novartis.
Overall Study STARTED	3
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Birt-Hogg-Dube Syndrome Birt-Hogg-Dube Syndrome (BHD)-associated renal tumors Everolimus: Everolimus is a commercial agent and is supplied by Novartis.
Overall Study Adverse event-pneumonitis	2

Baseline Characteristics

Everolimus Therapy in People With Birt-Hogg-Dube Syndrome (BHD)-Associated Kidney Cancer or Sporadic Chromophobe Renal Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Birt-Hogg-Dube Syndrome n=3 Participants Birt-Hogg-Dube Syndrome (BHD)-associated renal tumors Everolimus: Everolimus is a commercial agent and is supplied by Novartis.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	56 years STANDARD_DEVIATION 10.75 • n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Race/Ethnicity, Customized White	2 Participants n=5 Participants
Race/Ethnicity, Customized Asian	1 Participants n=5 Participants
Race/Ethnicity, Customized A person not meeting definition of Hispanic/Latino	3 Participants n=5 Participants
Region of Enrollment United States	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: End of treatment: every 12 weeks up to 1 year

Outcome measures

Outcome measures
Measure	Birt-Hogg-Dube Syndrome n=3 Participants Birt-Hogg-Dube Syndrome (BHD)-associated renal tumors Everolimus: Everolimus is a commercial agent and is supplied by Novartis.
Overall Response Rate With Everolimus Treatment. Complete Response	0 Participants
Overall Response Rate With Everolimus Treatment. Partial Response	0 Participants
Overall Response Rate With Everolimus Treatment. Stable Disease	3 Participants
Overall Response Rate With Everolimus Treatment. Progressive Disease	0 Participants

SECONDARY outcome

Timeframe: median follow-up time: 9 months

Population: Because all the 3 patients were alive without progression as of date of off study their median time to progression cannot be determined.

Progression-free survival is defined as the time interval from start of treatment to documented evidence of disease progression. Progression is at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes baseline sum if that is the smallest on study). The sum must also demonstrate an absolute increase of at least 5mm. Note: the appearance of one or more new lesions is also progression. Progressive disease was assessed by the Response Evaluation in Solid Tumors (RECIST) criteria v1.1.

Outcome measures

Outcome measures
Measure	Birt-Hogg-Dube Syndrome n=3 Participants Birt-Hogg-Dube Syndrome (BHD)-associated renal tumors Everolimus: Everolimus is a commercial agent and is supplied by Novartis.
Progression-free Survival (PFS)	NA Months Insufficient number of patients with events.

SECONDARY outcome

Timeframe: From the first day of treatment to the day of death, up to 1 year

Population: Because all the 3 patients were alive as of date of off study, their median time to death is unknown.

Overall Survival is defined as the time between the first day of treatment to the day of death.

Outcome measures

Outcome measures
Measure	Birt-Hogg-Dube Syndrome n=3 Participants Birt-Hogg-Dube Syndrome (BHD)-associated renal tumors Everolimus: Everolimus is a commercial agent and is supplied by Novartis.
Overall Survival (OS)	NA Months Insufficient number of patients with events.

SECONDARY outcome

Timeframe: Date treatment consent signed to date off study, approximately 8 months and 28 days.

Here is the count of participants with non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence.

Outcome measures

Outcome measures
Measure	Birt-Hogg-Dube Syndrome n=3 Participants Birt-Hogg-Dube Syndrome (BHD)-associated renal tumors Everolimus: Everolimus is a commercial agent and is supplied by Novartis.
Count of Participants With Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)	3 Participants

Adverse Events

Birt-Hogg-Dube Syndrome

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Birt-Hogg-Dube Syndrome n=3 participants at risk Birt-Hogg-Dube Syndrome (BHD)-associated renal tumors Everolimus: Everolimus is a commercial agent and is supplied by Novartis.
Gastrointestinal disorders Abdominal pain	33.3% 1/3 • Number of events 1 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Investigations Alanine aminotransferase increased	33.3% 1/3 • Number of events 2 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Musculoskeletal and connective tissue disorders Arthralgia	33.3% 1/3 • Number of events 1 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Investigations Aspartate aminotransferase increased	33.3% 1/3 • Number of events 1 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Investigations Cholesterol high	33.3% 1/3 • Number of events 2 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Gastrointestinal disorders Constipation	33.3% 1/3 • Number of events 2 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Gastrointestinal disorders Diarrhea	66.7% 2/3 • Number of events 5 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Nervous system disorders Dizziness	33.3% 1/3 • Number of events 1 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Gastrointestinal disorders Dyspepsia	66.7% 2/3 • Number of events 2 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Gastrointestinal disorders Fatigue	33.3% 1/3 • Number of events 2 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Nervous system disorders Headache	66.7% 2/3 • Number of events 2 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Renal and urinary disorders Hematuria	33.3% 1/3 • Number of events 3 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Renal and urinary disorders Hemoglobinuria	33.3% 1/3 • Number of events 1 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Investigations Hyperglycemia	33.3% 1/3 • Number of events 1 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Investigations Hypertriglyceridemia	100.0% 3/3 • Number of events 6 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Infections and infestations Infections and infestations - Other, Upper Respiratory Infection	33.3% 1/3 • Number of events 1 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Investigations Lymphocyte count decreased	66.7% 2/3 • Number of events 2 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Gastrointestinal disorders Mucositis oral	66.7% 2/3 • Number of events 3 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Musculoskeletal and connective tissue disorders Myalgia	33.3% 1/3 • Number of events 1 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Gastrointestinal disorders Nausea	33.3% 1/3 • Number of events 2 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Investigations Neutrophil count decreased	33.3% 1/3 • Number of events 9 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Gastrointestinal disorders Pain	33.3% 1/3 • Number of events 1 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Investigations Platelet count decreased	33.3% 1/3 • Number of events 1 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Respiratory, thoracic and mediastinal disorders Pneumonitis	66.7% 2/3 • Number of events 2 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Skin and subcutaneous tissue disorders Pruritus	66.7% 2/3 • Number of events 2 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Skin and subcutaneous tissue disorders Rash acneiform	33.3% 1/3 • Number of events 2 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Ear and labyrinth disorders Tinnitus	33.3% 1/3 • Number of events 1 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Infections and infestations Tooth infection	33.3% 1/3 • Number of events 1 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Respiratory, thoracic and mediastinal disorders Upper respiratory infection	33.3% 1/3 • Number of events 1 • Date treatment consent signed to date off study, approximately 8 months and 28 days.
Investigations White blood cell decreased	33.3% 1/3 • Number of events 8 • Date treatment consent signed to date off study, approximately 8 months and 28 days.

Additional Information

Dr. Ramaprasad Srinivasan

National Cancer Institute

Phone: 301-451-2298

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place