Roll-over study in patients with TSC and refractory seizures who arejudged by the Investigator to benefit from continued treatment witheverolimus after completion of study CRAD001M2304.

Phase 1

• Conditions: Refractory seizures associated with tuberous sclerosis complex; MedDRA version: 21.0Level: LLTClassification code 10045138Term: Tuberous sclerosisSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2016-002977-37-IT
• Lead Sponsor: OVARTIS PHARMA AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-06-01
• Last Update Posted: 2 August 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 216

Inclusion Criteria

1. Patient is currently enrolled in the Novartis-sponsored study
CRAD001M2304 receiving everolimus and has fulfilled all its
requirements.
2. Patient is currently benefiting from treatment with everolimus, as
determined by the Investigator.
3. Patient has demonstrated compliance, as assessed by the
Investigator, with the parent study protocol requirements.
4. Patient is willing and able to comply with scheduled visits and
treatment plans.
5. Written informed consent/adolescent assent obtained prior to
enrolling into the roll-over study.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 43
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Patient has been permanently discontinued from everolimus study
treatment in CRAD001M2304.
2. Everolimus is approved for patients with TSC with refractory seizures
3. 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 & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the long term safety data with everolimus treatment i.e<br>adverse events (AEs) and serious adverse events (SAEs).;Secondary Objective: To evaluate clinical benefit as assessed by the Investigator;Primary end point(s): Frequency and severity of AEs/SAEs;Timepoint(s) of evaluation of this end point: Continuous monitoring and reporting every 3 months (during scheduled<br>visits)

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Proportion of patients with clinical benefit as assessed by the<br>Investigator at scheduled visits;Timepoint(s) of evaluation of this end point: Continuous monitoring and reporting every 3 months (during scheduled<br>visits)