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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02962414




Registration number
NCT02962414
Ethics application status
Date submitted
9/11/2016
Date registered
11/11/2016
Date last updated
21/04/2020

Titles & IDs
Public title
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
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.
Secondary ID [1] 0 0
CRAD001M2X02B
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Tuberous Sclerosis Complex 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Neurological 0 0 0 0
Epilepsy

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - everolimus

Experimental: everolimus - everolimus, 2mg dispersible tablets


Treatment: Drugs: everolimus
everolimus, 2mg dispersible tablets

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Occurances of adverse events and serious adverse events - The patients will be followed continuously for tolerability (i.e adverse events, serious adverse events)
Timepoint [1] 0 0
Day 1 up to approximately 10 years
Secondary outcome [1] 0 0
Percentage of patients with clinical benefit - 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.
Timepoint [1] 0 0
Day 1 up to approximately 10 years, assessed every 12 weeks,

Eligibility
Key inclusion criteria
Key

- 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
Minimum age
2 Years
Maximum age
65 Years
Gender
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,WA
Recruitment hospital [1] 0 0
Novartis Investigative Site - Randwick
Recruitment hospital [2] 0 0
Novartis Investigative Site - Nedlands
Recruitment postcode(s) [1] 0 0
2130 - Randwick
Recruitment postcode(s) [2] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
Connecticut
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Minnesota
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United States of America
State/province [7] 0 0
New Jersey
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United States of America
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Ohio
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United States of America
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Oregon
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United States of America
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Pennsylvania
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United States of America
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Texas
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Belgium
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Brussel
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Belgium
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Bruxelles
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Belgium
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Gent
Country [15] 0 0
Belgium
State/province [15] 0 0
Leuven
Country [16] 0 0
Canada
State/province [16] 0 0
British Columbia
Country [17] 0 0
Colombia
State/province [17] 0 0
Valle Del Cauca
Country [18] 0 0
France
State/province [18] 0 0
Amiens Cedex 1
Country [19] 0 0
France
State/province [19] 0 0
Bron Cedex
Country [20] 0 0
France
State/province [20] 0 0
Lille Cedex
Country [21] 0 0
France
State/province [21] 0 0
Marseille Cedex 5
Country [22] 0 0
France
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Strasbourg Cedex
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Hungary
State/province [23] 0 0
Budapest
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Italy
State/province [24] 0 0
BA
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Italy
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BO
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Italy
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PV
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Italy
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RM
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Italy
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SI
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Japan
State/province [29] 0 0
Okayama
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Japan
State/province [30] 0 0
Osaka
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Japan
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Shizuoka
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Korea, Republic of
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Seoul
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Mexico
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Jalisco
Country [34] 0 0
Poland
State/province [34] 0 0
Warszawa
Country [35] 0 0
Russian Federation
State/province [35] 0 0
Samara Region
Country [36] 0 0
Russian Federation
State/province [36] 0 0
Voronezh Region
Country [37] 0 0
Russian Federation
State/province [37] 0 0
Moscow
Country [38] 0 0
Spain
State/province [38] 0 0
Andalucia
Country [39] 0 0
Spain
State/province [39] 0 0
Catalunya
Country [40] 0 0
Spain
State/province [40] 0 0
Pais Vasco
Country [41] 0 0
Spain
State/province [41] 0 0
Madrid
Country [42] 0 0
Spain
State/province [42] 0 0
Valencia
Country [43] 0 0
Taiwan
State/province [43] 0 0
Kaohsiung City
Country [44] 0 0
Taiwan
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Tainan
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Taiwan
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Taipei
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Thailand
State/province [46] 0 0
Bangkok
Country [47] 0 0
Turkey
State/province [47] 0 0
Ankara
Country [48] 0 0
Turkey
State/province [48] 0 0
Istanbul
Country [49] 0 0
United Kingdom
State/province [49] 0 0
Birmingham
Country [50] 0 0
United Kingdom
State/province [50] 0 0
Cambrigdeshire
Country [51] 0 0
United Kingdom
State/province [51] 0 0
Buckinghamshire
Country [52] 0 0
United Kingdom
State/province [52] 0 0
Liverpool
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United Kingdom
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London
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United Kingdom
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Sheffield
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United Kingdom
State/province [55] 0 0
York

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
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
Trial website
https://clinicaltrials.gov/show/NCT02962414
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications