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

April 1, 2026 updated by: Novartis Pharmaceuticals

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.

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

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Actual)

206

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Sydney, New South Wales, Australia, 2031
        • Novartis Investigative Site
    • Western Australia
      • Nedlands, Western Australia, Australia, 6009
        • Novartis Investigative Site
  • Belgium
      • Brussels, Belgium, 1200
        • Novartis Investigative Site
      • Brussels, Belgium, 1090
        • Novartis Investigative Site
      • Ghent, Belgium, 9000
        • Novartis Investigative Site
      • Leuven, Belgium, 3000
        • Novartis Investigative Site
  • Canada
    • British Columbia
      • Vancouver, British Columbia, Canada, V6H 3V4
        • Novartis Investigative Site
  • Colombia
    • Valle del Cauca Department
      • Cali, Valle del Cauca Department, Colombia, 760032
        • Novartis Investigative Site
  • France
      • Amiens, France, 80054
        • Novartis Investigative Site
      • Bron, France, 69677
        • Novartis Investigative Site
      • Lille, France, 59037
        • Novartis Investigative Site
      • Marseille, France, 13885
        • Novartis Investigative Site
      • Strasbourg, France, 67000
        • Novartis Investigative Site
  • Hungary
      • Budapest, Hungary, 1145
        • Novartis Investigative Site
  • Italy
    • BA
      • Bari, BA, Italy, 70124
        • Novartis Investigative Site
    • BO
      • Bologna, BO, Italy, 40139
        • Novartis Investigative Site
    • PV
      • Pavia, PV, Italy, 27100
        • Novartis Investigative Site
    • RM
      • Roma, RM, Italy, 00161
        • Novartis Investigative Site
    • SI
      • Siena, SI, Italy, 53100
        • Novartis Investigative Site
  • Japan
      • Okayama, Japan, 7008558
        • Novartis Investigative Site
      • Osaka, Japan, 5340021
        • Novartis Investigative Site
    • Osaka
      • Suita, Osaka, Japan, 565-0871
        • Novartis Investigative Site
    • Shizuoka
      • Shizuoka, Shizuoka, Japan, 4208688
        • Novartis Investigative Site
  • Mexico
    • Jalisco
      • Guadalajara, Jalisco, Mexico, 44280
        • Novartis Investigative Site
  • Poland
      • Warsaw, Poland, 04-730
        • Novartis Investigative Site
  • Russia
      • Moscow, Russia, 119991
        • Novartis Investigative Site
      • Moscow, Russia, 127412
        • Novartis Investigative Site
      • Voronezh, Russia, 394024
        • Novartis Investigative Site
    • Samara Oblast
      • Samara, Samara Oblast, Russia, 443095
        • Novartis Investigative Site
  • South Korea
      • Seoul, South Korea, 03080
        • Novartis Investigative Site
      • Seoul, South Korea, 03722
        • Novartis Investigative Site
      • Seoul, South Korea, 06351
        • Novartis Investigative Site
  • Spain
      • Barcelona, Spain, 08035
        • Novartis Investigative Site
      • Madrid, Spain, 28041
        • Novartis Investigative Site
      • Madrid, Spain, 28009
        • Novartis Investigative Site
      • Seville, Spain, 41013
        • Novartis Investigative Site
      • Valencia, Spain, 46026
        • Novartis Investigative Site
    • Gipuzkoa
      • Donostia / San Sebastian, Gipuzkoa, Spain, 20014
        • Novartis Investigative Site
  • Taiwan
      • Kaohsiung City, Taiwan, 83301
        • Novartis Investigative Site
      • Tainan, Taiwan, 704302
        • Novartis Investigative Site
      • Taipei, Taiwan, 10002
        • Novartis Investigative Site
  • Thailand
      • Bangkok, Thailand, 10330
        • Novartis Investigative Site
      • Bangkok, Thailand, 10700
        • Novartis Investigative Site
      • Bangkok, Thailand, 10400
        • Novartis Investigative Site
  • Turkey (Türkiye)
    • Fatih
      • Istanbul, Fatih, Turkey (Türkiye), 34093
        • Novartis Investigative Site
    • Yenimahalle
      • Ankara, Yenimahalle, Turkey (Türkiye), 06500
        • Novartis Investigative Site
  • United Kingdom
      • Buckinghamshire, United Kingdom, SL9 0RJ
        • Novartis Investigative Site
      • Cambridge, United Kingdom, CB2 0QQ
        • Novartis Investigative Site
      • Liverpool, United Kingdom, L12 2AP
        • Novartis Investigative Site
      • London, United Kingdom, SW17 0QT
        • Novartis Investigative Site
      • London, United Kingdom, WC1N 3JH
        • Novartis Investigative Site
      • Sheffield, United Kingdom, S10 2TH
        • Novartis Investigative Site
      • York, United Kingdom, YO31 7EX
        • Novartis Investigative Site
    • West Midlands
      • Birmingham, West Midlands, United Kingdom, B15 2TH
        • Novartis Investigative Site
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85012
        • TGen APNNA
    • California
      • Los Angeles, California, United States, 90005-1752
        • David Geffen School of Medicine at UCLA
      • Oakland, California, United States, 94609
        • UCSF Benioff Children s Hospital
      • San Diego, California, United States, 92123
        • Rady Children s Hospital
    • Colorado
      • Aurora, Colorado, United States, 80045
        • University of Colorado School of Medicine
    • Connecticut
      • Hartford, Connecticut, United States, 06106
        • Connecticut Childrens Medical Cntr
    • Illinois
      • Chicago, Illinois, United States, 60637
        • University of Chicago Medical Center
    • Minnesota
      • Saint Paul, Minnesota, United States, 55102
        • Minnesota Epilepsy Group
    • New Jersey
      • Morristown, New Jersey, United States, 07962
        • Atlantic Health Systems
    • Ohio
      • Cincinnati, Ohio, United States, 45229-3039
        • Cinn Children Hosp Medical Center
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health Sciences University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Childrens Hospital Of Philadelphia
    • Texas
      • Dallas, Texas, United States, 75219
        • Texas Scottish Rite Hos for Child
      • Houston, Texas, United States, 77030
        • Texas Childrens Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 65 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key 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.

Key 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: everolimus
everolimus, 2mg dispersible tablets
Drug: everolimus
everolimus, 2mg dispersible tablets
Other Names:
  • RAD001

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Occurances of adverse events and serious adverse events
Time Frame: Day 1 up to approximately 10 years
The patients will be followed continuously for tolerability (i.e adverse events, serious adverse events)
Day 1 up to approximately 10 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of patients with clinical benefit
Time Frame: Day 1 up to approximately 10 years, assessed every 12 weeks,
At every quarterly visit (every 12 weeks ± 2 weeks), the Investigator is required to confirm that the patients continue to have clinical benefit as per Investigator's assessment.
Day 1 up to approximately 10 years, assessed every 12 weeks,

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Investigators

  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 8, 2017

Primary Completion (Estimated)

August 4, 2027

Study Completion (Estimated)

August 13, 2027

Study Registration Dates

First Submitted

November 9, 2016

First Submitted That Met QC Criteria

November 9, 2016

First Posted (Estimated)

November 11, 2016

Study Record Updates

Last Update Posted (Actual)

April 2, 2026

Last Update Submitted That Met QC Criteria

April 1, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CRAD001M2X02B
  • 2024-516746-19-00 (Registry Identifier: EU CT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

IPD Plan Description

Novartis is committed to sharing access to patient-level data and supporting clinical documents from eligible studies with qualified external researchers. Requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to protect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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