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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)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT00790400
First received: November 10, 2008
Last updated: January 26, 2016
Last verified: January 2016
Results First Received: May 23, 2012  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions: Tuberous Sclerosis Complex (TSC)
Lymphangioleiomyomatosis (LAM)
Interventions: Drug: Everolimus (RAD001)
Drug: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Everolimus Study drug was given by continuous oral daily dosing of two 5 mg tablets.
Placebo Placebo was given by continuous oral daily dosing of two 5 mg tablets.

Participant Flow:   Overall Study
    Everolimus   Placebo
STARTED   79   39 
COMPLETED   7 [1]   13 
NOT COMPLETED   72   26 
Ongoing in Double-blind                72                26 
[1] Completed means discontinued the double-blind treatment period



  Baseline Characteristics


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Angiomyolipoma Response Rate as Per Central Radiology Review (Double-blind Period)   [ Time Frame: From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or analysis cut-off date (30-June-2011). ]

2.  Secondary:   Time to Angiomyolipoma Progression as Per Central Radiology Review (Double-blind Period)   [ Time Frame: From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or analysis cut-off date (30-June-2011). ]

3.  Secondary:   Skin Lesion Response Rate as Per Investigator (Only Patients With at Least One Skin Lesion at Baseline)   [ Time Frame: From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or analysis cut-off date (30-June-2011). ]

4.  Secondary:   Percentage of Participants With Renal Impairment   [ Time Frame: From date of randomization until the earliest date of first documented AML progression, date of further anti-AML medication (including open-label Everolimus)/surgery or analysis cut-off date (30-June-2011). ]

5.  Secondary:   Change From Baseline in Plasma Angiogenic Molecules   [ Time Frame: Baseline, 12 Months ]
Results not yet reported.   Anticipated Reporting Date:   09/2016   Safety Issue:   No


  Serious Adverse Events
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Reporting Groups
  Description
Everolimus Everolimus
Placebo Placebo

Serious Adverse Events
    Everolimus   Placebo
Total, serious adverse events     
# participants affected / at risk   15/79 (18.99%)   7/39 (17.95%) 
Blood and lymphatic system disorders     
Bone marrow oedema † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Cardiac disorders     
Cardiovascular insufficiency † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Gastrointestinal disorders     
Caecitis † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Constipation † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Volvulus † 1     
# participants affected / at risk   0/79 (0.00%)   1/39 (2.56%) 
General disorders     
Fatigue † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Hepatobiliary disorders     
Bile duct stenosis † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Immune system disorders     
Hypersensitivity † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Infections and infestations     
Appendicitis † 1     
# participants affected / at risk   2/79 (2.53%)   0/39 (0.00%) 
Bronchitis † 1     
# participants affected / at risk   0/79 (0.00%)   1/39 (2.56%) 
Gastrointestinal infection † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Pneumonia † 1     
# participants affected / at risk   1/79 (1.27%)   1/39 (2.56%) 
Pyelonephritis † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Tonsillitis † 1     
# participants affected / at risk   0/79 (0.00%)   1/39 (2.56%) 
Wound infection † 1     
# participants affected / at risk   0/79 (0.00%)   1/39 (2.56%) 
Injury, poisoning and procedural complications     
Fall † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Metabolism and nutrition disorders     
Decreased appetite † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Gout † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Musculoskeletal and connective tissue disorders     
Back pain † 1     
# participants affected / at risk   0/79 (0.00%)   1/39 (2.56%) 
Joint effusion † 1     
# participants affected / at risk   2/79 (2.53%)   0/39 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Angiomyolipoma † 1     
# participants affected / at risk   0/79 (0.00%)   1/39 (2.56%) 
Nervous system disorders     
Complex regional pain syndrome † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Convulsion † 1     
# participants affected / at risk   2/79 (2.53%)   0/39 (0.00%) 
Grand mal convulsion † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Psychiatric disorders     
Affective disorder † 1     
# participants affected / at risk   0/79 (0.00%)   1/39 (2.56%) 
Hallucination † 1     
# participants affected / at risk   0/79 (0.00%)   1/39 (2.56%) 
Renal and urinary disorders     
Renal failure acute † 1     
# participants affected / at risk   2/79 (2.53%)   0/39 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Bronchospasm † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Pleuritic pain † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Skin and subcutaneous tissue disorders     
Angioedema † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Vascular disorders     
Hypertensive crisis † 1     
# participants affected / at risk   1/79 (1.27%)   0/39 (0.00%) 
Events were collected by systematic assessment
1 Term from vocabulary, 14.0




  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information