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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT02962414
Ethics application status
Date submitted
9/11/2016
Date registered
11/11/2016

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
Timepoint [1] 0 0
Day 1 up to approximately 10 years
Secondary outcome [1] 0 0
Percentage of patients with clinical benefit
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
Sex
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
NA
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
8/06/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
13/08/2027
Actual
Sample size
Target
Accrual to date
Final
206
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
Country [7] 0 0
United States of America
State/province [7] 0 0
New Jersey
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Oregon
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United States of America
State/province [10] 0 0
Pennsylvania
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United States of America
State/province [11] 0 0
Texas
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Belgium
State/province [12] 0 0
Brussel
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Belgium
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Bruxelles
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Belgium
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Gent
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Belgium
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Leuven
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Canada
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British Columbia
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Colombia
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Valle Del Cauca
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France
State/province [18] 0 0
Amiens
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France
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Bron Cedex
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France
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Lille Cedex
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France
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Marseille Cedex 05
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France
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Strasbourg
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Hungary
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Budapest
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Italy
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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
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Okayama
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Japan
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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
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Warszawa
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Russian Federation
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Samara Region
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Russian Federation
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Voronezh Region
Country [37] 0 0
Russian Federation
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Moscow
Country [38] 0 0
Spain
State/province [38] 0 0
Andalucia
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Spain
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Catalunya
Country [40] 0 0
Spain
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Pais Vasco
Country [41] 0 0
Spain
State/province [41] 0 0
Madrid
Country [42] 0 0
Spain
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Valencia
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Taiwan
State/province [43] 0 0
Kaohsiung
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Taiwan
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Tainan
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Taiwan
State/province [45] 0 0
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
Country [53] 0 0
United Kingdom
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London
Country [54] 0 0
United Kingdom
State/province [54] 0 0
Sheffield
Country [55] 0 0
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
Trial website
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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
No/undecided IPD sharing reason/comment
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


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT02962414