Trial Outcomes & Findings for Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex (NCT NCT00411619)

NCT ID: NCT00411619

Last Updated: 2014-10-20

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 28 participants
• Primary outcome timeframe: During the entire study
• Results posted on: 2014-10-20

Participant Flow

Participant milestones

Measure	Everolimus This was a non-randomized, open-label, single arm study; all patients in the study received treatment with everolimus.
Overall Study STARTED	28
Overall Study COMPLETED	22
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex

Baseline characteristics by cohort

Measure	Everolimus n=28 Participants This was a non-randomized, open-label, single arm study; all patients in the study received treatment with everolimus.
Age, Categorical <=18 years	22 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	6 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	17 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	2 Participants n=5 Participants
Race (NIH/OMB) White	24 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants
Region of Enrollment United States	28 participants n=5 Participants

PRIMARY outcome

Timeframe: During the entire study

Outcome measures

Measure	Everolimus n=28 Participants This was a non-randomized, open-label, single arm study; all patients in the study received treatment with everolimus. The initial starting dose was to be 3.0 mg/m2/day taken either daily or every other day with titration to achieve target trough concentrations of 5 to 15 ng/mL, subject to tolerability. Study drug was self-administered orally (or administered by a caregiver) at the same time each day.
Number With Observed Adverse Side Effects Upper Respiratory Tract Infection	26 participants
Number With Observed Adverse Side Effects Stomatitis	25 participants
Number With Observed Adverse Side Effects Sinusitis	15 participants
Number With Observed Adverse Side Effects Mouth Ulceration	14 participants
Number With Observed Adverse Side Effects Cellulitis	12 participants
Number With Observed Adverse Side Effects Diarrhoea	12 participants
Number With Observed Adverse Side Effects Gastroenteritis	12 participants
Number With Observed Adverse Side Effects Otitis Media	11 participants
Number With Observed Adverse Side Effects Pyrexia	10 participants
Number With Observed Adverse Side Effects Vomiting	10 participants
Number With Observed Adverse Side Effects Acne	9 participants
Number With Observed Adverse Side Effects Convulsion	9 participants
Number With Observed Adverse Side Effects Nasopharyngitis	9 participants
Number With Observed Adverse Side Effects Rhinitis Allergic	9 participants
Number With Observed Adverse Side Effects Conjunctivitis	8 participants
Number With Observed Adverse Side Effects Pharyngitis	8 participants
Number With Observed Adverse Side Effects Rash	8 participants
Number With Observed Adverse Side Effects Constipation	7 participants
Number With Observed Adverse Side Effects Cough	7 participants
Number With Observed Adverse Side Effects Dermatitis Acneiform	7 participants
Number With Observed Adverse Side Effects Dermatitis Contact	7 participants
Number With Observed Adverse Side Effects Excoriation	7 participants
Number With Observed Adverse Side Effects Laceration	7 participants
Number With Observed Adverse Side Effects Nasal Congestion	7 participants
Number With Observed Adverse Side Effects Otitis Externa	7 participants
Number With Observed Adverse Side Effects Abnormal Behaviour	6 participants
Number With Observed Adverse Side Effects Body Tinea	6 participants
Number With Observed Adverse Side Effects Dry Skin	6 participants
Number With Observed Adverse Side Effects Pneumonia	6 participants
Number With Observed Adverse Side Effects Skin Infection	6 participants
Number With Observed Adverse Side Effects Urinary Tract Infection	6 participants

SECONDARY outcome

Timeframe: During the entire study

Outcome measures

Measure	Everolimus n=26 Participants This was a non-randomized, open-label, single arm study; all patients in the study received treatment with everolimus. The initial starting dose was to be 3.0 mg/m2/day taken either daily or every other day with titration to achieve target trough concentrations of 5 to 15 ng/mL, subject to tolerability. Study drug was self-administered orally (or administered by a caregiver) at the same time each day.
Overall Reduction in SEGA Tumor Volume. # with reductions > 30% relative to baseline	14 participants
Overall Reduction in SEGA Tumor Volume. # with reductions > 50% relative to baseline	12 participants

Adverse Events

Everolimus

Serious events: 9 serious events

Other events: 28 other events

Deaths: 0 deaths

Serious adverse events

Measure	Everolimus n=28 participants at risk This was a non-randomized, open-label, single arm study; all patients in the study received treatment with everolimus.
Infections and infestations Abscess Limb	7.1% 2/28 • Number of events 2 • Adverse events were collected throughout the course of the study, which was open for seven years.
Infections and infestations Cellulitis	7.1% 2/28 • Number of events 2 • Adverse events were collected throughout the course of the study, which was open for seven years.
Nervous system disorders Convulsion	7.1% 2/28 • Number of events 2 • Adverse events were collected throughout the course of the study, which was open for seven years.
Infections and infestations Bronchitis Viral	3.6% 1/28 • Number of events 1 • Adverse events were collected throughout the course of the study, which was open for seven years.
Infections and infestations Gastroenteritis	3.6% 1/28 • Number of events 1 • Adverse events were collected throughout the course of the study, which was open for seven years.
Blood and lymphatic system disorders Leukocytosis	3.6% 1/28 • Number of events 1 • Adverse events were collected throughout the course of the study, which was open for seven years.
Musculoskeletal and connective tissue disorders Pain In Extremity	3.6% 1/28 • Number of events 1 • Adverse events were collected throughout the course of the study, which was open for seven years.
Skin and subcutaneous tissue disorders Petechiae	3.6% 1/28 • Number of events 1 • Adverse events were collected throughout the course of the study, which was open for seven years.
Infections and infestations Pneumonia	3.6% 1/28 • Number of events 1 • Adverse events were collected throughout the course of the study, which was open for seven years.
Injury, poisoning and procedural complications Post Lumbar Puncture Syndrome	3.6% 1/28 • Number of events 1 • Adverse events were collected throughout the course of the study, which was open for seven years.
Infections and infestations Sinusitis	3.6% 1/28 • Number of events 1 • Adverse events were collected throughout the course of the study, which was open for seven years.
General disorders Sudden Unexplained Death In Epilepsy	3.6% 1/28 • Number of events 1 • Adverse events were collected throughout the course of the study, which was open for seven years.

Other adverse events

Measure	Everolimus n=28 participants at risk This was a non-randomized, open-label, single arm study; all patients in the study received treatment with everolimus.
Infections and infestations Upper Respiratory Tract Infection	92.9% 26/28 • Number of events 26 • Adverse events were collected throughout the course of the study, which was open for seven years.
Infections and infestations Sinusitis	53.6% 15/28 • Number of events 15 • Adverse events were collected throughout the course of the study, which was open for seven years.
Infections and infestations Cellulitis	42.9% 12/28 • Number of events 12 • Adverse events were collected throughout the course of the study, which was open for seven years.
Infections and infestations Gastroenteritis	42.9% 12/28 • Number of events 12 • Adverse events were collected throughout the course of the study, which was open for seven years.
Infections and infestations Otitis media	39.3% 11/28 • Number of events 11 • Adverse events were collected throughout the course of the study, which was open for seven years.
Infections and infestations Nasopharyngitis	32.1% 9/28 • Number of events 9 • Adverse events were collected throughout the course of the study, which was open for seven years.
Infections and infestations Pharyngitis	28.6% 8/28 • Number of events 8 • Adverse events were collected throughout the course of the study, which was open for seven years.
Infections and infestations Otitis Externa	25.0% 7/28 • Number of events 7 • Adverse events were collected throughout the course of the study, which was open for seven years.
Infections and infestations Pneumonia	21.4% 6/28 • Number of events 6 • Adverse events were collected throughout the course of the study, which was open for seven years.
Infections and infestations Skin infection	21.4% 6/28 • Number of events 6 • Adverse events were collected throughout the course of the study, which was open for seven years.
Infections and infestations Urinary Tract Infection	21.4% 6/28 • Number of events 6 • Adverse events were collected throughout the course of the study, which was open for seven years.
Gastrointestinal disorders Stomatitis	89.3% 25/28 • Number of events 25 • Adverse events were collected throughout the course of the study, which was open for seven years.
Gastrointestinal disorders Mouth Ulceration	50.0% 14/28 • Number of events 14 • Adverse events were collected throughout the course of the study, which was open for seven years.
Gastrointestinal disorders Diarrhoea	21.4% 6/28 • Number of events 6 • Adverse events were collected throughout the course of the study, which was open for seven years.
Skin and subcutaneous tissue disorders Acne	28.6% 8/28 • Number of events 8 • Adverse events were collected throughout the course of the study, which was open for seven years.
Skin and subcutaneous tissue disorders Dermatitis Acneiform	25.0% 7/28 • Number of events 7 • Adverse events were collected throughout the course of the study, which was open for seven years.
General disorders Pyrexia	28.6% 8/28 • Number of events 8 • Adverse events were collected throughout the course of the study, which was open for seven years.
Eye disorders Conjunctivitis	28.6% 8/28 • Number of events 8 • Adverse events were collected throughout the course of the study, which was open for seven years.

Additional Information

Dr. David Franz

Cincinnati Children's Hospital Medical Center

Phone: 513-636-4222

Email: david.franz@cchmc.org

Results disclosure agreements

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