Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM) (EXIST-2)


Tracking Information

Start Date  ICMJEMarch 2009
Estimated Primary Completion DateJanuary 2014   (final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE 
 (submitted: November 12, 2008)		• Angiomyolipoma 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 years ] [ Designated as safety issue: No ]
Original Primary Outcome Measures  ICMJESame as current
Change HistoryComplete list of historical versions of study NCT00790400 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE 
 (submitted: November 12, 2008)
  • • Time to angiomyolipoma 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 (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 (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 (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 (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 (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 Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)
Official Title  ICMJEA Randomized, Double-blind, Placebo-controlled Study of RAD0001 in the Treatment of Angiomyolipoma in Patients With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)
Brief Summary

This study will evaluate the safety and efficacy of RAD001 in treating patients with Angiomyolipomas associated with Tuberous Sclerosis Complex or Sporadic Lymphangioleiomyomatosis.

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  ICMJE
  • Tuberous Sclerosis
  • Subependymal Giant Cell Astrocytoma
Intervention  ICMJE
  • Drug: Everolimus (RAD001)
    5 mg in tablet form. 10mg daily dosing throughout the trial.
    Other Name: RAd001
  • Other: Placebo
    Placebo
Study Arms / Comparison Groups
  • 1: RAD001 5mg: Experimental
    Intervention: Drug: Everolimus (RAD001)
  • 2: RAD001 Placebo: Placebo Comparator
    Intervention: Other: Placebo

Recruitment Information

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

Inclusion Criteria:

  • 18 years or older
  • Definite diagnosis of Tuberous Sclerosis or Sporadic Lymphangioleiomyomatosis
  • At least one Angiomyolipoma of at least 3 cm in diameter
  • Females of child bearing potential must use birth control

Exclusion Criteria:

  • Recent heart attack, cardiac related chest pain or stroke
  • Severely impaired lung function
  • Bleeding related to angiomyolipoma
  • Severe liver dysfunction
  • Severe kidney disfunction
  • 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
Ages18 Years and older
Accepts Healthy VolunteersNo
Contacts  ICMJE
Contact: Novartis Pharmaceuticlas+1-800-340-6843
Location Countries  ICMJEUnited States,   Canada,   France,   Germany,   Italy,   Netherlands,   Poland,   Russian Federation,   United Kingdom

Administrative Information

NCT ID  ICMJENCT00790400
Responsible Partyexternal Affairs, Novartis
Study ID Numbers  ICMJECRAD001M2302
Study Sponsor  ICMJENovartis Pharmaceuticals
Collaborators  ICMJE 
Investigators  ICMJE
Study Director:Novartis PharmaceuticalsNovartis Pharmaceuticals
Information Provided ByNovartis