Roll-over study in patients with TSC and refractory seizures who are judged by the Investigator to benefit from continued treatment with everolimus after completion of study CRAD001M2304.
- Conditions
- Refractory seizures associated with tuberous sclerosis complexMedDRA version: 20.0Level: PTClassification code 10045138Term: Tuberous sclerosisSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2016-002977-37-BE
- Lead Sponsor
- ovartis Pharma AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 216
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: 173
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
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 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 visits)
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Proportion of patients with clinical benefit as assessed by the Investigator at scheduled visits;Timepoint(s) of evaluation of this end point: Continuous monitoring and reporting every 3 months (during scheduled visits)