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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

Primary Purpose

Tuberous Sclerosis Complex

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
everolimus
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Tuberous Sclerosis Complex focused on measuring Everolimus, Tuberous sclerosis complex, TSC, Refractory seizure, Roll over study, Long term safety, PASS

Eligibility Criteria

2 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • TGen/APNNA SC
  • David Geffen School of Medicine at UCLA
  • UCSF Benioff Children s Hospital
  • Rady Children s Hospital SC
  • University of Colorado School of Medicine
  • Connecticut Childrens Medical Center
  • University of Chicago Medical Center
  • Minnesota Epilepsy Group
  • Atlantic Health Systems
  • Cinn Children Hosp Medical Center SC-2
  • Oregon Health and Science Univ
  • Childrens Hospital of Philadelphia
  • Texas Scottish Rite Hospital for Children SC
  • Texas Children s Hospital SC-2
  • Novartis Investigative Site
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Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

everolimus

Arm Description

everolimus, 2mg dispersible tablets

Outcomes

Primary Outcome Measures

Occurances of adverse events and serious adverse events
The patients will be followed continuously for tolerability (i.e adverse events, serious adverse events)

Secondary Outcome Measures

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.

Full Information

First Posted
November 9, 2016
Last Updated
May 9, 2023
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT02962414
Brief 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
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.
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
June 8, 2017 (Actual)
Primary Completion Date
August 4, 2027 (Anticipated)
Study Completion Date
August 13, 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. 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
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Tuberous Sclerosis Complex
Keywords
Everolimus, Tuberous sclerosis complex, TSC, Refractory seizure, Roll over study, Long term safety, PASS

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
206 (Actual)

8. Arms, Groups, and Interventions

Arm Title
everolimus
Arm Type
Experimental
Arm Description
everolimus, 2mg dispersible tablets
Intervention Type
Drug
Intervention Name(s)
everolimus
Other Intervention Name(s)
RAD001
Intervention Description
everolimus, 2mg dispersible tablets
Primary Outcome Measure Information:
Title
Occurances of adverse events and serious adverse events
Description
The patients will be followed continuously for tolerability (i.e adverse events, serious adverse events)
Time Frame
Day 1 up to approximately 10 years
Secondary Outcome Measure Information:
Title
Percentage of patients with clinical benefit
Description
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.
Time Frame
Day 1 up to approximately 10 years, assessed every 12 weeks,

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Facility Information:
Facility Name
TGen/APNNA SC
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85012
Country
United States
Facility Name
David Geffen School of Medicine at UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90005-1752
Country
United States
Facility Name
UCSF Benioff Children s Hospital
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
Facility Name
Rady Children s Hospital SC
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
University of Colorado School of Medicine
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Connecticut Childrens Medical Center
City
Hartford
State/Province
Connecticut
ZIP/Postal Code
06106
Country
United States
Facility Name
University of Chicago Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60637
Country
United States
Facility Name
Minnesota Epilepsy Group
City
Saint Paul
State/Province
Minnesota
ZIP/Postal Code
55102
Country
United States
Facility Name
Atlantic Health Systems
City
Morristown
State/Province
New Jersey
ZIP/Postal Code
07962
Country
United States
Facility Name
Cinn Children Hosp Medical Center SC-2
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229-3039
Country
United States
Facility Name
Oregon Health and Science Univ
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Childrens Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Texas Scottish Rite Hospital for Children SC
City
Dallas
State/Province
Texas
ZIP/Postal Code
75219
Country
United States
Facility Name
Texas Children s Hospital SC-2
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Novartis Investigative Site
City
Randwick
State/Province
New South Wales
ZIP/Postal Code
2130
Country
Australia
Facility Name
Novartis Investigative Site
City
Nedlands
State/Province
Western Australia
ZIP/Postal Code
6009
Country
Australia
Facility Name
Novartis Investigative Site
City
Jette
State/Province
Brussel
ZIP/Postal Code
1090
Country
Belgium
Facility Name
Novartis Investigative Site
City
Bruxelles
ZIP/Postal Code
1200
Country
Belgium
Facility Name
Novartis Investigative Site
City
Gent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
Novartis Investigative Site
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
Novartis Investigative Site
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6H 3V4
Country
Canada
Facility Name
Novartis Investigative Site
City
Cali
State/Province
Valle Del Cauca
ZIP/Postal Code
760012
Country
Colombia
Facility Name
Novartis Investigative Site
City
Amiens
ZIP/Postal Code
80054
Country
France
Facility Name
Novartis Investigative Site
City
Bron Cedex
ZIP/Postal Code
69677
Country
France
Facility Name
Novartis Investigative Site
City
Lille Cedex
ZIP/Postal Code
59037
Country
France
Facility Name
Novartis Investigative Site
City
Marseille Cedex 05
ZIP/Postal Code
13885
Country
France
Facility Name
Novartis Investigative Site
City
Strasbourg Cedex
ZIP/Postal Code
F 67098
Country
France
Facility Name
Novartis Investigative Site
City
Budapest
ZIP/Postal Code
1145
Country
Hungary
Facility Name
Novartis Investigative Site
City
Bari
State/Province
BA
ZIP/Postal Code
70124
Country
Italy
Facility Name
Novartis Investigative Site
City
Bologna
State/Province
BO
ZIP/Postal Code
40139
Country
Italy
Facility Name
Novartis Investigative Site
City
Pavia
State/Province
PV
ZIP/Postal Code
27100
Country
Italy
Facility Name
Novartis Investigative Site
City
Roma
State/Province
RM
ZIP/Postal Code
00161
Country
Italy
Facility Name
Novartis Investigative Site
City
Siena
State/Province
SI
ZIP/Postal Code
53100
Country
Italy
Facility Name
Novartis Investigative Site
City
Okayama-city
State/Province
Okayama
ZIP/Postal Code
700-8558
Country
Japan
Facility Name
Novartis Investigative Site
City
Suita
State/Province
Osaka
ZIP/Postal Code
565 0871
Country
Japan
Facility Name
Novartis Investigative Site
City
Shizuoka-city
State/Province
Shizuoka
ZIP/Postal Code
420-8688
Country
Japan
Facility Name
Novartis Investigative Site
City
Osaka
ZIP/Postal Code
534-0021
Country
Japan
Facility Name
Novartis Investigative Site
City
Seoul
ZIP/Postal Code
03080
Country
Korea, Republic of
Facility Name
Novartis Investigative Site
City
Seoul
ZIP/Postal Code
03722
Country
Korea, Republic of
Facility Name
Novartis Investigative Site
City
Seoul
ZIP/Postal Code
06351
Country
Korea, Republic of
Facility Name
Novartis Investigative Site
City
Guadalajara
State/Province
Jalisco
ZIP/Postal Code
44280
Country
Mexico
Facility Name
Novartis Investigative Site
City
Warszawa
ZIP/Postal Code
04 730
Country
Poland
Facility Name
Novartis Investigative Site
City
Samara
State/Province
Samara Region
ZIP/Postal Code
443095
Country
Russian Federation
Facility Name
Novartis Investigative Site
City
Voronezh
State/Province
Voronezh Region
ZIP/Postal Code
394024
Country
Russian Federation
Facility Name
Novartis Investigative Site
City
Moscow
ZIP/Postal Code
119991
Country
Russian Federation
Facility Name
Novartis Investigative Site
City
Moscow
ZIP/Postal Code
127412
Country
Russian Federation
Facility Name
Novartis Investigative Site
City
Sevilla
State/Province
Andalucia
ZIP/Postal Code
41013
Country
Spain
Facility Name
Novartis Investigative Site
City
Barcelona
State/Province
Catalunya
ZIP/Postal Code
08035
Country
Spain
Facility Name
Novartis Investigative Site
City
San Sebastian
State/Province
Pais Vasco
ZIP/Postal Code
20080
Country
Spain
Facility Name
Novartis Investigative Site
City
Madrid
ZIP/Postal Code
28009
Country
Spain
Facility Name
Novartis Investigative Site
City
Madrid
ZIP/Postal Code
28041
Country
Spain
Facility Name
Novartis Investigative Site
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Facility Name
Novartis Investigative Site
City
Kaohsiung City
ZIP/Postal Code
83301
Country
Taiwan
Facility Name
Novartis Investigative Site
City
Tainan
ZIP/Postal Code
70403
Country
Taiwan
Facility Name
Novartis Investigative Site
City
Taipei
ZIP/Postal Code
10002
Country
Taiwan
Facility Name
Novartis Investigative Site
City
Bangkok
ZIP/Postal Code
10330
Country
Thailand
Facility Name
Novartis Investigative Site
City
Bangkok
ZIP/Postal Code
10400
Country
Thailand
Facility Name
Novartis Investigative Site
City
Bangkok
ZIP/Postal Code
10700
Country
Thailand
Facility Name
Novartis Investigative Site
City
Ankara
ZIP/Postal Code
06500
Country
Turkey
Facility Name
Novartis Investigative Site
City
Istanbul
ZIP/Postal Code
34093
Country
Turkey
Facility Name
Novartis Investigative Site
City
Edgbaston
State/Province
Birmingham
ZIP/Postal Code
B15 2TH
Country
United Kingdom
Facility Name
Novartis Investigative Site
City
Cambridge
State/Province
Cambrigdeshire
ZIP/Postal Code
CB2 0QQ
Country
United Kingdom
Facility Name
Novartis Investigative Site
City
Buckinghamshire
ZIP/Postal Code
SL9 0RJ
Country
United Kingdom
Facility Name
Novartis Investigative Site
City
Liverpool
ZIP/Postal Code
L12 2AP
Country
United Kingdom
Facility Name
Novartis Investigative Site
City
London
ZIP/Postal Code
SW17 0QT
Country
United Kingdom
Facility Name
Novartis Investigative Site
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
Facility Name
Novartis Investigative Site
City
Sheffield
ZIP/Postal Code
S10 2TH
Country
United Kingdom
Facility Name
Novartis Investigative Site
City
York
ZIP/Postal Code
YO31 7EX
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Undecided
IPD Sharing 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

Learn more about this trial

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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