Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment
An Open-label, Multi-center Long-term Safety Roll-over Study in Patients With Tuberous Sclerosis Complex (TSC) and Refractory Seizures Who Are Judged by the Investigator to Benefit From Continued Treatment With Everolimus After Completion of Study CRAD001M2304.
2 other identifiers
interventional
206
18 countries
68
Brief Summary
The purpose of this study is to evaluate the long-term safety in patients with TSC and refractory seizures who are currently receiving everolimus treatment in the Novartis-sponsored EXIST-3 study and who are determined to be benefiting from continued treatment as judged by the investigator at the completion of EXIST-3
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Jun 2017
Longer than P75 for phase_3
68 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 9, 2016
CompletedFirst Posted
Study publicly available on registry
November 11, 2016
CompletedStudy Start
First participant enrolled
June 8, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 4, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
August 13, 2027
April 2, 2026
March 1, 2026
10.2 years
November 9, 2016
April 1, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Occurances of adverse events and serious adverse events
The patients will be followed continuously for tolerability (i.e adverse events, serious adverse events)
Day 1 up to approximately 10 years
Secondary Outcomes (1)
Percentage of patients with clinical benefit
Day 1 up to approximately 10 years, assessed every 12 weeks,
Study Arms (1)
everolimus
EXPERIMENTALeverolimus, 2mg dispersible tablets
Interventions
Eligibility Criteria
You may qualify if:
- Patient is currently enrolled in the Novartis-sponsored EXIST-3 study, receiving everolimus, and has fulfilled all its requirements
- Patient is currently benefiting from treatment with everolimus, as determined by the Investigator.
- Patient has demonstrated compliance, as assessed by the Investigator,with the parent study protocol requirements.
- Patient is willing and able to comply with scheduled visits and treatment plans.
- Written informed consent/adolescent assent obtained prior to enrolling into the roll-over study.
You may not qualify if:
- Patient has been permanently discontinued from everolimus study treatment in EXIST-3 study
- Everolimus is approved for patients with TSC and refractory seizures and is reimbursed in the local country.
- Patients who are receiving everolimus in combination with unapproved or experimental treatments for seizure control Anti-epileptic drug (AEDs) are allowed for the purpose of seizure control.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (68)
TGen APNNA
Phoenix, Arizona, 85012, United States
David Geffen School of Medicine at UCLA
Los Angeles, California, 90005-1752, United States
UCSF Benioff Children s Hospital
Oakland, California, 94609, United States
Rady Children s Hospital
San Diego, California, 92123, United States
University of Colorado School of Medicine
Aurora, Colorado, 80045, United States
Connecticut Childrens Medical Cntr
Hartford, Connecticut, 06106, United States
University of Chicago Medical Center
Chicago, Illinois, 60637, United States
Minnesota Epilepsy Group
Saint Paul, Minnesota, 55102, United States
Atlantic Health Systems
Morristown, New Jersey, 07962, United States
Cinn Children Hosp Medical Center
Cincinnati, Ohio, 45229-3039, United States
Oregon Health Sciences University
Portland, Oregon, 97239, United States
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Texas Scottish Rite Hos for Child
Dallas, Texas, 75219, United States
Texas Childrens Hospital
Houston, Texas, 77030, United States
Novartis Investigative Site
Sydney, New South Wales, 2031, Australia
Novartis Investigative Site
Nedlands, Western Australia, 6009, Australia
Novartis Investigative Site
Brussels, 1090, Belgium
Novartis Investigative Site
Brussels, 1200, Belgium
Novartis Investigative Site
Ghent, 9000, Belgium
Novartis Investigative Site
Leuven, 3000, Belgium
Novartis Investigative Site
Vancouver, British Columbia, V6H 3V4, Canada
Novartis Investigative Site
Cali, Valle del Cauca Department, 760032, Colombia
Novartis Investigative Site
Amiens, 80054, France
Novartis Investigative Site
Bron, 69677, France
Novartis Investigative Site
Lille, 59037, France
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Marseille, 13885, France
Novartis Investigative Site
Strasbourg, 67000, France
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Budapest, 1145, Hungary
Novartis Investigative Site
Bari, BA, 70124, Italy
Novartis Investigative Site
Bologna, BO, 40139, Italy
Novartis Investigative Site
Pavia, PV, 27100, Italy
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Roma, RM, 00161, Italy
Novartis Investigative Site
Siena, SI, 53100, Italy
Novartis Investigative Site
Suita, Osaka, 565-0871, Japan
Novartis Investigative Site
Shizuoka, Shizuoka, 4208688, Japan
Novartis Investigative Site
Okayama, 7008558, Japan
Novartis Investigative Site
Osaka, 5340021, Japan
Novartis Investigative Site
Guadalajara, Jalisco, 44280, Mexico
Novartis Investigative Site
Warsaw, 04-730, Poland
Novartis Investigative Site
Samara, Samara Oblast, 443095, Russia
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Moscow, 119991, Russia
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Moscow, 127412, Russia
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Voronezh, 394024, Russia
Novartis Investigative Site
Seoul, 03080, South Korea
Novartis Investigative Site
Seoul, 03722, South Korea
Novartis Investigative Site
Seoul, 06351, South Korea
Novartis Investigative Site
Donostia / San Sebastian, Gipuzkoa, 20014, Spain
Novartis Investigative Site
Barcelona, 08035, Spain
Novartis Investigative Site
Madrid, 28009, Spain
Novartis Investigative Site
Madrid, 28041, Spain
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Seville, 41013, Spain
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Valencia, 46026, Spain
Novartis Investigative Site
Kaohsiung City, 83301, Taiwan
Novartis Investigative Site
Tainan, 704302, Taiwan
Novartis Investigative Site
Taipei, 10002, Taiwan
Novartis Investigative Site
Bangkok, 10330, Thailand
Novartis Investigative Site
Bangkok, 10400, Thailand
Novartis Investigative Site
Bangkok, 10700, Thailand
Novartis Investigative Site
Istanbul, Fatih, 34093, Turkey (Türkiye)
Novartis Investigative Site
Ankara, Yenimahalle, 06500, Turkey (Türkiye)
Novartis Investigative Site
Birmingham, West Midlands, B15 2TH, United Kingdom
Novartis Investigative Site
Buckinghamshire, SL9 0RJ, United Kingdom
Novartis Investigative Site
Cambridge, CB2 0QQ, United Kingdom
Novartis Investigative Site
Liverpool, L12 2AP, United Kingdom
Novartis Investigative Site
London, SW17 0QT, United Kingdom
Novartis Investigative Site
London, WC1N 3JH, United Kingdom
Novartis Investigative Site
Sheffield, S10 2TH, United Kingdom
Novartis Investigative Site
York, YO31 7EX, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 9, 2016
First Posted
November 11, 2016
Study Start
June 8, 2017
Primary Completion (Estimated)
August 4, 2027
Study Completion (Estimated)
August 13, 2027
Last Updated
April 2, 2026
Record last verified: 2026-03