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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

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ClinicalTrials.gov Identifier: NCT02962414
Recruitment Status : Recruiting
First Posted : November 11, 2016
Last Update Posted : October 8, 2018
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 218 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 : February 27, 2019
Estimated Study Completion Date : July 8, 2027


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




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.



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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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02962414


Contacts
Contact: Novartis Pharmaceuticals 1-888-669-6682 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals +41613241111

  Show 68 Study Locations
Sponsors and Collaborators
Novartis Pharmaceuticals

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02962414     History of Changes
Other Study ID Numbers: CRAD001M2X02B
First Posted: November 11, 2016    Key Record Dates
Last Update Posted: October 8, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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:
Sclerosis
Seizures
Tuberous Sclerosis
Pathologic Processes
Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurologic Manifestations
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
Everolimus
Sirolimus
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Anti-Bacterial Agents
Anti-Infective Agents