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

Tuberous Sclerosis Complex Clinical Trial

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.

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02962414
• Other study ID #: CRAD001M2X02B
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: June 8, 2017
• Est. completion date: August 13, 2027

Study information

• Verified date: May 2024
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical 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

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 206
• Est. completion date: August 13, 2027
• Est. primary completion date: August 4, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 65 Years

Eligibility

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.

Related Conditions & MeSH terms

• Sclerosis
• Seizures
• Tuberous Sclerosis
• Tuberous Sclerosis Complex

Intervention

Drug:
• everolimus
everolimus, 2mg dispersible tablets

Locations

Country	Name	City	State
Australia	Novartis Investigative Site	Nedlands	Western Australia
Australia	Novartis Investigative Site	Randwick	New South Wales
Belgium	Novartis Investigative Site	Bruxelles
Belgium	Novartis Investigative Site	Gent
Belgium	Novartis Investigative Site	Jette	Brussel
Belgium	Novartis Investigative Site	Leuven
Canada	Novartis Investigative Site	Vancouver	British Columbia
Colombia	Novartis Investigative Site	Cali	Valle Del Cauca
France	Novartis Investigative Site	Amiens
France	Novartis Investigative Site	Bron Cedex
France	Novartis Investigative Site	Lille Cedex
France	Novartis Investigative Site	Marseille Cedex 05
France	Novartis Investigative Site	Strasbourg
Hungary	Novartis Investigative Site	Budapest
Italy	Novartis Investigative Site	Bari	BA
Italy	Novartis Investigative Site	Bologna	BO
Italy	Novartis Investigative Site	Pavia	PV
Italy	Novartis Investigative Site	Roma	RM
Italy	Novartis Investigative Site	Siena	SI
Japan	Novartis Investigative Site	Okayama-city	Okayama
Japan	Novartis Investigative Site	Osaka
Japan	Novartis Investigative Site	Shizuoka-city	Shizuoka
Japan	Novartis Investigative Site	Suita	Osaka
Korea, Republic of	Novartis Investigative Site	Seoul
Korea, Republic of	Novartis Investigative Site	Seoul
Korea, Republic of	Novartis Investigative Site	Seoul
Mexico	Novartis Investigative Site	Guadalajara	Jalisco
Poland	Novartis Investigative Site	Warszawa
Russian Federation	Novartis Investigative Site	Moscow
Russian Federation	Novartis Investigative Site	Moscow
Russian Federation	Novartis Investigative Site	Samara	Samara Region
Russian Federation	Novartis Investigative Site	Voronezh	Voronezh Region
Spain	Novartis Investigative Site	Barcelona	Catalunya
Spain	Novartis Investigative Site	Madrid
Spain	Novartis Investigative Site	Madrid
Spain	Novartis Investigative Site	San Sebastian	Pais Vasco
Spain	Novartis Investigative Site	Sevilla	Andalucia
Spain	Novartis Investigative Site	Valencia
Taiwan	Novartis Investigative Site	Kaohsiung
Taiwan	Novartis Investigative Site	Tainan
Taiwan	Novartis Investigative Site	Taipei
Thailand	Novartis Investigative Site	Bangkok
Thailand	Novartis Investigative Site	Bangkok
Thailand	Novartis Investigative Site	Bangkok
Turkey	Novartis Investigative Site	Ankara
Turkey	Novartis Investigative Site	Istanbul
United Kingdom	Novartis Investigative Site	Buckinghamshire
United Kingdom	Novartis Investigative Site	Cambridge	Cambrigdeshire
United Kingdom	Novartis Investigative Site	Edgbaston	Birmingham
United Kingdom	Novartis Investigative Site	Liverpool
United Kingdom	Novartis Investigative Site	London
United Kingdom	Novartis Investigative Site	London
United Kingdom	Novartis Investigative Site	Sheffield
United Kingdom	Novartis Investigative Site	York
United States	Novartis Investigative Site	Aurora	Colorado
United States	Novartis Investigative Site	Chicago	Illinois
United States	Novartis Investigative Site	Cincinnati	Ohio
United States	Novartis Investigative Site	Dallas	Texas
United States	Novartis Investigative Site	Hartford	Connecticut
United States	Novartis Investigative Site	Houston	Texas
United States	Novartis Investigative Site	Los Angeles	California
United States	Novartis Investigative Site	Morristown	New Jersey
United States	Novartis Investigative Site	Oakland	California
United States	Novartis Investigative Site	Philadelphia	Pennsylvania
United States	Novartis Investigative Site	Phoenix	Arizona
United States	Novartis Investigative Site	Portland	Oregon
United States	Novartis Investigative Site	Saint Paul	Minnesota
United States	Novartis Investigative Site	San Diego	California

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  Australia,  Belgium,  Canada,  Colombia,  France,  Hungary,  Italy,  Japan,  Korea, Republic of,  Mexico,  Poland,  Russian Federation,  Spain,  Taiwan,  Thailand,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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.	Day 1 up to approximately 10 years, assessed every 12 weeks,