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.

• Register: EUCTR
• Last refreshed on: 13 March 2017
• Main ID: EUCTR2016-002977-37-HU
• Date of registration: 18/01/2017
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma AG
• Public title: 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.
• Scientific 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.
• Date of first enrolment: 24/02/2017
• Target sample size: 216
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-002977-37
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: yes Other trial design description: Roll-over study If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

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

Key inclusion & exclusion criteria

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: 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

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).


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Refractory seizures associated with tuberous sclerosis complex
MedDRA version: 19.1 Level: PT Classification code 10045138 Term: Tuberous sclerosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Intervention(s)

Trade Name: Votubia
Product Name: Everolimus
Product Code: RAD001
Pharmaceutical Form: Dispersible tablet
INN or Proposed INN: everolimus
CAS Number: 159351-69-6
Current Sponsor code: RAD001
Other descriptive name: EVEROLIMUS
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 2-

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: Continuous monitoring and reporting every 3 months (during scheduled visits)

Main Objective: To evaluate the long term safety data with everolimus treatment i.e adverse events (AEs) and serious adverse events (SAEs).

Primary end point(s): Frequency and severity of AEs/SAEs

Secondary Objective: To evaluate clinical benefit as assessed by the Investigator

Secondary Outcome(s)

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)

Secondary ID(s)

CRAD001M2X02B

Source(s) of Monetary Support

Novartis Pharma AG