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

NCT ID: NCT02962414

Last Updated: 2026-02-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

206 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-06-08

Study Completion Date

2027-08-13

Brief Summary

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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

Detailed Description

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This is a multi-center, open label study to evaluate the long term safety of everolimus in patients with TSC and refractory seizures currently being treated in the Novartis-sponsored study EXIST-3 study and are judged by their parent study investigator as benefiting from the current study treatment. The study is expected to remain open for approximately 10 years (or until 31-Dec-2022 in the UK) from First Patient First Visit (FPFV).

Patients will continue to be treated in this study until they are no longer benefiting from their everolimus treatment as judged by the Investigator or until one of the protocol defined discontinuation criteria is met or everolimus becomes commercially available according to local regulations.

Conditions

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Tuberous Sclerosis Complex

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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everolimus

everolimus, 2mg dispersible tablets

Group Type EXPERIMENTAL

everolimus

Intervention Type DRUG

everolimus, 2mg dispersible tablets

Interventions

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everolimus

everolimus, 2mg dispersible tablets

Intervention Type DRUG

Other Intervention Names

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RAD001

Eligibility Criteria

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Inclusion Criteria

* 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.

Exclusion Criteria

* 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.
Minimum Eligible Age

2 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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TGen APNNA

Phoenix, Arizona, United States

Site Status

David Geffen School of Medicine at UCLA

Los Angeles, California, United States

Site Status

UCSF Benioff Children s Hospital

Oakland, California, United States

Site Status

Rady Children s Hospital

San Diego, California, United States

Site Status

University of Colorado School of Medicine

Aurora, Colorado, United States

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Connecticut Childrens Medical Cntr

Hartford, Connecticut, United States

Site Status

University of Chicago Medical Center

Chicago, Illinois, United States

Site Status

Minnesota Epilepsy Group

Saint Paul, Minnesota, United States

Site Status

Atlantic Health Systems

Morristown, New Jersey, United States

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Cinn Children Hosp Medical Center

Cincinnati, Ohio, United States

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Oregon Health Sciences University

Portland, Oregon, United States

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Childrens Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

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Texas Scottish Rite Hos for Child

Dallas, Texas, United States

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Texas Childrens Hospital

Houston, Texas, United States

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Novartis Investigative Site

Sydney, New South Wales, Australia

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Novartis Investigative Site

Nedlands, Western Australia, Australia

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Novartis Investigative Site

Brussels, , Belgium

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Novartis Investigative Site

Brussels, , Belgium

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Novartis Investigative Site

Ghent, , Belgium

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Novartis Investigative Site

Leuven, , Belgium

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Novartis Investigative Site

Vancouver, British Columbia, Canada

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Novartis Investigative Site

Cali, Valle del Cauca Department, Colombia

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Novartis Investigative Site

Amiens, , France

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Novartis Investigative Site

Bron, , France

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Novartis Investigative Site

Lille, , France

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Novartis Investigative Site

Marseille, , France

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Novartis Investigative Site

Strasbourg, , France

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Novartis Investigative Site

Budapest, , Hungary

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Novartis Investigative Site

Bari, BA, Italy

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Novartis Investigative Site

Bologna, BO, Italy

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Novartis Investigative Site

Pavia, PV, Italy

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Novartis Investigative Site

Roma, RM, Italy

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Novartis Investigative Site

Siena, SI, Italy

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Novartis Investigative Site

Suita, Osaka, Japan

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Novartis Investigative Site

Shizuoka, Shizuoka, Japan

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Novartis Investigative Site

Okayama, , Japan

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Novartis Investigative Site

Osaka, , Japan

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Novartis Investigative Site

Guadalajara, Jalisco, Mexico

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Novartis Investigative Site

Warsaw, , Poland

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Novartis Investigative Site

Samara, Samara Oblast, Russia

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Novartis Investigative Site

Moscow, , Russia

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Novartis Investigative Site

Moscow, , Russia

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Novartis Investigative Site

Voronezh, , Russia

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Novartis Investigative Site

Seoul, Korea, South Korea

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Novartis Investigative Site

Seoul, , South Korea

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Novartis Investigative Site

Seoul, , South Korea

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Novartis Investigative Site

Donostia / San Sebastian, Gipuzkoa, Spain

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Novartis Investigative Site

Barcelona, , Spain

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Novartis Investigative Site

Madrid, , Spain

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Novartis Investigative Site

Madrid, , Spain

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Novartis Investigative Site

Seville, , Spain

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Novartis Investigative Site

Valencia, , Spain

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Novartis Investigative Site

Kaohsiung City, , Taiwan

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Novartis Investigative Site

Tainan, , Taiwan

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Novartis Investigative Site

Taipei, , Taiwan

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Novartis Investigative Site

Bangkok, , Thailand

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Novartis Investigative Site

Bangkok, , Thailand

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Novartis Investigative Site

Bangkok, , Thailand

Site Status

Novartis Investigative Site

Istanbul, Fatih, Turkey (Türkiye)

Site Status

Novartis Investigative Site

Ankara, Yenimahalle, Turkey (Türkiye)

Site Status

Novartis Investigative Site

Birmingham, West Midlands, United Kingdom

Site Status

Novartis Investigative Site

Buckinghamshire, , United Kingdom

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Novartis Investigative Site

Cambridge, , United Kingdom

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Novartis Investigative Site

Liverpool, , United Kingdom

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Novartis Investigative Site

London, , United Kingdom

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Novartis Investigative Site

London, , United Kingdom

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Novartis Investigative Site

Sheffield, , United Kingdom

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Novartis Investigative Site

York, , United Kingdom

Site Status

Countries

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United States Australia Belgium Canada Colombia France Hungary Italy Japan Mexico Poland Russia South Korea Spain Taiwan Thailand Turkey (Türkiye) United Kingdom

Other Identifiers

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2024-516746-19-00

Identifier Type: REGISTRY

Identifier Source: secondary_id

CRAD001M2X02B

Identifier Type: -

Identifier Source: org_study_id

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