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

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Brief Title

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

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.

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
    

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 Phase

Phase 3

Study Type

Interventional


Primary Outcome

Occurances of adverse events and serious adverse events

Secondary Outcome

 Percentage of patients with clinical benefit

Condition

Tuberous Sclerosis Complex

Intervention

everolimus

Study Arms / Comparison Groups

 everolimus
Description:  everolimus, 2mg dispersible tablets

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

206

Start Date

April 2, 2017

Completion Date

August 4, 2027

Primary Completion Date

August 4, 2027

Eligibility Criteria

        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.
      

Gender

All

Ages

2 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT02962414

Organization ID

CRAD001M2X02B


Responsible Party

Sponsor

Study Sponsor

Novartis Pharmaceuticals


Study Sponsor

, , 


Verification Date

June 2021