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


Tracking Information

Start Date  ICMJEApril 2009
Estimated Primary Completion DateFebruary 2014   (final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE 
 (submitted: November 12, 2008)		• Subependymal Giant Cell Astrocytoma response rate through MRIs at screening, 12, 24 and 48 weeks and annually thereafter [ Time Frame: Screening, 12, 24, 48 weeks and annually for 4-5 years ] [ Designated as safety issue: No ]
Original Primary Outcome Measures  ICMJESame as current
Change HistoryComplete list of historical versions of study NCT00789828 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE 
 (submitted: November 12, 2008)
  • • Time to Subependymal Giant Cell Astrocytoma progression through MRIs at screening, 12, 24 and 48 weeks and annually thereafter [ Time Frame: Biomarkers assessed throughout the first year of the study only. All other outcomes are assessed through the duration of the study (4-5 years). ] [ Designated as safety issue: Yes ]
  • • Skin lesion response rate tracked by digital photographs [ Time Frame: Biomarkers assessed throughout the first year of the study only. All other outcomes are assessed through the duration of the study (4-5 years). ] [ Designated as safety issue: Yes ]
  • • Change from baseline in biomarkers collected during the first years of study [ Time Frame: Biomarkers assessed throughout the first year of the study only. All other outcomes are assessed through the duration of the study (4-5 years). ] [ Designated as safety issue: Yes ]
  • • Changes in renal function by assessing creatinine clearance levels throughout the study [ Time Frame: Biomarkers assessed throughout the first year of the study only. All other outcomes are assessed through the duration of the study (4-5 years). ] [ Designated as safety issue: Yes ]
  • • Safety assessed on a continuous basis throughout study [ Time Frame: Biomarkers assessed throughout the first year of the study only. All other outcomes are assessed through the duration of the study (4-5 years). ] [ Designated as safety issue: Yes ]
  • • Change from baseline in frequency of epileptiform events monitored by 24 hour EEG at baseline and 6 months [ Time Frame: Biomarkers assessed throughout the first year of the study only. All other outcomes are assessed through the duration of the study (4-5 years). ] [ Designated as safety issue: Yes ]
Original Secondary Outcome Measures  ICMJESame as current

Descriptive Information

Brief Title  ICMJEEfficacy and Safety of RAD001 in Patients of All Ages With Subependymal Giant Cell Astrocytoma Associated With Tuberous Sclerosis Complex (TSC)
Official Title  ICMJEA Randomized, Double-blind, Placebo-controlled Study of RAD001 in the Treatment of Patients With Subependymal Giant Cell Astrocytomas (SEGA) Associated With Tuberous Sclerosis Complex (TSC)
Brief Summary

This study will evaluate the safety and efficacy of RAD001 in treating patients with Subependymal Giant Cell Astrocytomas associated with Tuberous Sclerosis Complex.

Detailed Description 
Study PhasePhase III
Study Type  ICMJEInterventional
Study Design  ICMJETreatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Crossover Assignment, Safety/Efficacy Study
Condition  ICMJETuberous Sclerosis
Intervention  ICMJE
  • Drug: RAD001 (Everolimus)
    1 mg in tablet form
    Other Name: RAD001
  • Drug: Placebo
    placebo
Study Arms / Comparison Groups
  • RAD001: Experimental
    Intervention: Drug: RAD001 (Everolimus)
  • RAD001 Placebo: Placebo Comparator
    Intervention: Drug: Placebo

Recruitment Information

Estimated Enrollment  ICMJE99
Completion Date 
Estimated Primary Completion DateFebruary 2014   (final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • All Ages
  • Definite diagnosis of Tuberous Sclerosis
  • At least one Subependymal Giant Cell Astrocytoma of at least 1 cm in diameter
  • Evidence of SEGA progression as compared to prior MRI scans
  • Females of child bearing potential must use birth control

Exclusion Criteria:

  • 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

GenderBoth
Ages 
Accepts Healthy VolunteersNo
Contacts  ICMJE
Contact: Novartis Pharmaceuticals+1 800-340-6843
Location Countries  ICMJEUnited States,   Australia,   Belgium,   Canada,   Italy,   Netherlands,   Poland,   Russian Federation,   United Kingdom

Administrative Information

NCT ID  ICMJENCT00789828
Responsible PartyExternal Affairs, Novartis
Study ID Numbers  ICMJECRAD001M2301
Study Sponsor  ICMJENovartis Pharmaceuticals
Collaborators  ICMJE 
Investigators  ICMJE
Study Director:Novartis PharmaceuticlasNovartis Pharmaceuticals
Information Provided ByNovartis