Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1)

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Brief Title

Efficacy and Safety of Everolimus (RAD001) in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)(EXIST-1)

Official Title

A Randomized, Double-blind, Placebo-controlled Study of Everolimus in the Treatment of Patients With Subependymal Giant Cell Astrocytomas (SEGA) Associated With Tuberous Sclerosis Complex (TSC)

Brief Summary

      This study evaluated the efficacy and safety of Everolimus in treating patients with
      Subependymal Giant Cell Astrocytomas associated with Tuberous Sclerosis Complex.
    


Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Percentage of Participants With Best Overall Subependymal Giant Cell Astrocytomas (SEGA) Response

Secondary Outcome

 Change From Baseline in Frequency of Total Seizure Events Per 24 Hours at Week 24 in Both Core and Extension Period

Condition

Tuberous Sclerosis

Intervention

Everolimus

Study Arms / Comparison Groups

 Everolimus
Description:  Everolimus was administered orally at a starting dose of 4.5mg/m^2 daily and subsequently titrated to attain whole blood trough concentration of 5 to 15 ng/mL. Dose adjustments were permitted based on safety and whole blood trough concentrations.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

117

Start Date

August 2009

Completion Date

October 2014

Primary Completion Date

March 2011

Eligibility Criteria

        Inclusion Criteria:

          -  All Ages

          -  Definite diagnosis of Tuberous Sclerosis according to the modified Gomez criteria

          -  At least one Subependymal Giant Cell Astrocytoma of at least 1 cm in diameter

          -  Evidence of SEGA worsening as compared to prior MRI scans

          -  Females of child bearing potential must use birth control

          -  Written informed consent

        Exclusion Criteria:

          -  SEGA related surgery is likely to be required in the opinion of the investigator

          -  Recent heart attack, cardiac related chest pain or stroke

          -  Severely impaired lung function

          -  Severe liver dysfunction

          -  Severe kidney dysfunction

          -  Pregnancy or breast feeding

          -  Current infection

          -  History of organ transplant

          -  Surgery within two months prior to study enrollment

          -  Prior therapy with a medication in the same class as Everolimus

          -  Uncontrolled high cholesterol

          -  Uncontrolled diabetes

          -  HIV

          -  Patients with metal implants thus prohibiting MRI evaluations

        Other protocol-defined inclusion/exclusion criteria may apply
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Novartis Pharmaceuticlas, , 

Location Countries

Australia

Location Countries

Australia

Administrative Informations


NCT ID

NCT00789828

Organization ID

CRAD001M2301

Secondary IDs

2007-006997-27

Responsible Party

Sponsor

Study Sponsor

Novartis Pharmaceuticals


Study Sponsor

Novartis Pharmaceuticlas, Study Director, Novartis Pharmaceuticals


Verification Date

January 2016