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.

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.

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2016-002977-37-HU

Enrollment

216

Registered

2017-01-18

Start date

2017-02-24

Completion date

Unknown

Last updated

2024-09-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

Interventions

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 des

Eligibility

Sex/Gender

All

Inclusion 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

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).

Design outcomes

Primary

Measure	Time frame
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)	—

Measure

Time frame

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

Measure	Time frame
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)	—

Countries

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

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026