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

• ClinicalTrials.gov Identifier: NCT02962414
• Recruitment Status: Active, not recruiting
• First Posted: November 11, 2016
• Last Update Posted: September 6, 2019
• Sponsor: Novartis Pharmaceuticals
• Information provided by (Responsible Party): Novartis ( Novartis Pharmaceuticals )

Study Description

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

Condition or disease	Intervention/treatment	Phase
Tuberous Sclerosis Complex	Drug: everolimus	Phase 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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 206 participants
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: 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.
• Actual Study Start Date: April 2, 2017
• Estimated Primary Completion Date: August 4, 2027
• Estimated Study Completion Date: August 4, 2027

Arms and Interventions

Arm	Intervention/treatment
Experimental: everolimus; everolimus, 2mg dispersible tablets	Drug: everolimus; everolimus, 2mg dispersible tablets; Other Name: RAD001

Outcome Measures

Primary Outcome Measures :

1. Occurances of adverse events and serious adverse events [ Time Frame: Day 1 up to approximately 10 years ]
   The patients will be followed continuously for tolerability (i.e adverse events, serious adverse events)

Secondary Outcome Measures :

1. Percentage of patients with clinical benefit [ Time Frame: Day 1 up to approximately 10 years, assessed every 12 weeks, ]
   At every quarterly visit (every 12 weeks ± 2 weeks), the Investigator is required to confirm that the patients continue to have clinical benefit as per Investigator's assessment.

Eligibility Criteria

• Ages Eligible for Study: 2 Years to 65 Years (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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.

Contacts and Locations

  Show 68 Study Locations

Sponsors and Collaborators

Novartis Pharmaceuticals

More Information

• Responsible Party: Novartis Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT02962414 History of Changes
• Other Study ID Numbers: CRAD001M2X02B
• First Posted: November 11, 2016
• Last Update Posted: September 6, 2019
• Last Verified: September 2019

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Undecided

Plan Description

Novartis is committed to sharing access to patient-level data and supporting clinical documents from eligible studies with qualified external researchers. Requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to protect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Novartis ( Novartis Pharmaceuticals ):

Everolimus; Tuberous sclerosis complex; TSC; Refractory seizure; Roll over study; Long term safety; PASS

Additional relevant MeSH terms:

Tuberous Sclerosis; Seizures; Sclerosis; Pathologic Processes; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Hamartoma; Neoplasms; Neoplasms, Multiple Primary; Neoplastic Syndromes, Hereditary; Malformations of Cortical Development, Group I; Malformations of Cortical Development; Nervous System Malformations; Neurocutaneous Syndromes; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Sirolimus; Everolimus; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Anti-Bacterial Agents; Anti-Infective Agents; Antibiotics, Antineoplastic; Antifungal Agents