Tuberous Sclerosis ComplexPhase 3NCT02962414
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
A Phase 3 study testing everolimus in people with Tuberous Sclerosis Complex, run by Novartis Pharmaceuticals at 67 sites in 18 countries.
Trial 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 Read more about this study
- Who is it for?
- Ages 2 to 65 years
- Conditions the study is for
- Tuberous Sclerosis Complex
- What is being tested?
- everolimus
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 206 participants
- Where
- 67 sitesin 18 countries
- Study dates
- 23 Aug 2017to 30 Dec 2026
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
67 sites in 18 countries.
67 sites in 18 countries
United States14 sites
University of Colorado School of Medicine
Aurora, Colorado, 80045
University of Chicago Medical Center
Chicago, Illinois, 60637
Cinn Children Hosp Medical Center
Cincinnati, Ohio, 45229-3039
Texas Scottish Rite Hos for Child
Dallas, Texas, 75219
Connecticut Childrens Medical Cntr
Hartford, Connecticut, 06106
Texas Childrens Hospital
Houston, Texas, 77030
David Geffen School of Medicine at UCLA
Los Angeles, California, 90005-1752
Atlantic Health Morristown MC
Morristown, New Jersey, 07962
UCSF Benioff Children s Hospital
Oakland, California, 94609
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
TGen APNNA
Phoenix, Arizona, 85012
Oregon Health Sciences University
Portland, Oregon, 97239
Minnesota Epilepsy Group
Saint Paul, Minnesota, 55102
Rady Children s Hospital
San Diego, California, 92123
United Kingdom7 sites
Novartis Investigative Site
Birmingham, West Midlands, B15 2TH
Novartis Investigative Site
Buckinghamshire, SL9 0RJ
Novartis Investigative Site
Cambridge, CB2 0QQ
Novartis Investigative Site
Liverpool, L12 2AP
Novartis Investigative Site
London, SW17 0QT
Novartis Investigative Site
London, WC1N 3JH
Novartis Investigative Site
Sheffield, S10 2TH
Spain6 sites
Novartis Investigative Site
Barcelona, 08035
Novartis Investigative Site
Donostia / San Sebastian, Gipuzkoa, 20014
Novartis Investigative Site
Madrid, 28009
Novartis Investigative Site
Madrid, 28041
Novartis Investigative Site
Seville, 41013
Novartis Investigative Site
Valencia, 46026
France5 sites
Novartis Investigative Site
Amiens, 80054
Novartis Investigative Site
Bron, 69677
Novartis Investigative Site
Lille, 59037
Novartis Investigative Site
Marseille, 13885
Novartis Investigative Site
Strasbourg, 67000
Italy5 sites
Novartis Investigative Site
Bari, BA, 70124
Novartis Investigative Site
Bologna, BO, 40139
Novartis Investigative Site
Pavia, PV, 27100
Novartis Investigative Site
Roma, RM, 00161
Novartis Investigative Site
Siena, SI, 53100
Belgium4 sites
Novartis Investigative Site
Brussels, 1090
Novartis Investigative Site
Brussels, 1200
Novartis Investigative Site
Ghent, 9000
Novartis Investigative Site
Leuven, 3000
Japan4 sites
Novartis Investigative Site
Okayama, 7008558
Novartis Investigative Site
Osaka, 5340021
Novartis Investigative Site
Shizuoka, Shizuoka, 4208688
Novartis Investigative Site
Suita, Osaka, 5650871
Russia4 sites
Novartis Investigative Site
Moscow, 119991
Novartis Investigative Site
Moscow, 127412
Novartis Investigative Site
Samara, Samara Oblast, 443095
Novartis Investigative Site
Voronezh, 394024
South Korea3 sites
Novartis Investigative Site
Seoul, 03080
Novartis Investigative Site
Seoul, 03722
Novartis Investigative Site
Seoul, 06351
Taiwan3 sites
Novartis Investigative Site
Kaohsiung City, 833
Novartis Investigative Site
Tainan, 704302
Novartis Investigative Site
Taipei, 10002
Thailand3 sites
Novartis Investigative Site
Bangkok, 10330
Novartis Investigative Site
Bangkok, 10400
Novartis Investigative Site
Bangkok, 10700
Australia2 sites
Novartis Investigative Site
Nedlands, Western Australia, 6009
Novartis Investigative Site
Sydney, New South Wales, 2031
Turkey (Türkiye)2 sites
Novartis Investigative Site
Ankara, Yenimahalle, 06500
Novartis Investigative Site
Istanbul, Fatih, 34093
Colombia1 site
Novartis Investigative Site
Cali, Valle del Cauca Department, 760032
About this study
Official title: 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
Read the 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.
- Conditions
- Tuberous Sclerosis Complex
- Being tested
- everolimus
- Keywords
- Everolimus, Tuberous sclerosis complex, TSC, Refractory seizure, Roll over study, Long term safety, PASS
- Medical subject headings
- Tuberous Sclerosis, Seizures
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
everolimusDrug
Also called RAD001
everolimus, 2mg dispersible tablets
| Study group | Role | What they receive |
|---|---|---|
| everolimus | Experimental | everolimus, 2mg dispersible tablets Receives: Drug: everolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurances of adverse events and serious adverse events
The patients will be followed continuously for tolerability (i.e adverse events, serious adverse events)
Measured: Day 1 up to approximately 10 years
Secondary outcomes (1)
Percentage of patients with clinical benefit
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.
Measured: Day 1 up to approximately 10 years, assessed every 12 weeks,
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Nov 2016 (estimated)
- Study start23 Aug 2017
- Primary completion30 Dec 2026 (estimated)
- Study completion30 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Tuberous Sclerosis ComplexStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 206 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02962414
- Sponsor's protocol ID
- CRAD001M2X02B
- Other IDs
- 2024-516746-19-00
- Status last verified
- Aug 2026
- First submitted
- 9 Nov 2016
- Last updated
- 21 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided: 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
Study at a glance
From the registry record, last updated by the sponsor on 21 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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.
- ClinicalTrials.gov ID
- NCT02962414
- Acronym / sponsor ID
- CRAD001M2X02B
- Conditions
- Tuberous Sclerosis Complex
- Keywords
- Everolimus, Tuberous sclerosis complex, TSC, Refractory seizure, Roll over study, Long term safety, PASS
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 206 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 23 Aug 2017 (actual)
- Primary completion
- 30 Dec 2026 (estimated)
- Study completion
- 30 Dec 2026 (estimated)
- First posted
- 11 Nov 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| everolimus | Experimental | Drug: everolimuseverolimus, 2mg dispersible tablets |
Who may qualify
- Ages
- 2 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- 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 (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| everolimusAlso: RAD001everolimus, 2mg dispersible tablets | Drug | everolimus |
Primary outcome
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)
1 secondary outcome
Percentage of patients with clinical benefit
Time frame: Day 1 up to approximately 10 years, assessed every 12 weeks,
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Aug 2026.
- Individual participant dataSharing planned: Undecided. 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
67 sites in 18 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing everolimus in people with Tuberous Sclerosis Complex, run by Novartis Pharmaceuticals at 67 sites in 18 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 21 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 67 sites in United States, United Kingdom, Spain, France, Italy and 13 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Occurances of adverse events and serious adverse events (time frame: Day 1 up to approximately 10 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Aug 2026.