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)
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Tracking Information | |||||
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First Received Date ICMJE | November 10, 2008 | ||||
Last Updated Date | July 25, 2012 | ||||
Start Date ICMJE | April 2009 | ||||
Primary Completion Date | June 2011 (final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
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). ] [ Designated as safety issue: No ] Angiomyolipoma response defined as the combination of the following criteria: reduction in angiomyolipoma volume of ≥ 50% relative to baseline, where angiomyolipoma volume was sum of volumes of all target lesions identified at baseline, and with a confirmatory scan performed approximately 12 weeks later (no sooner than 8 weeks later)• No new angiomyolipoma lesions ≥ 1.0 cm in longest diameter were identified.• There were no kidney increases in volume > 20% from nadir. The patient did not have any angiomyolipoma-related bleeding of ≥ grade 2 |
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Original Primary Outcome Measures ICMJE |
• 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 ] | ||||
Change History | Complete list of historical versions of study NCT00790400 on ClinicalTrials.gov Archive Site | ||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Outcome Measures ICMJE | |||||
Original Other Outcome Measures ICMJE | |||||
Descriptive Information | |||||
Brief Title ICMJE | Efficacy 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 ICMJE | A 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 Angiomyolipoma associated with Tuberous Sclerosis Complex or Sporadic Lymphangioleiomyomatosis. |
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Detailed Description | |||||
Study Type ICMJE | Interventional | ||||
Study Phase | Phase 3 | ||||
Study Design ICMJE | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
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Condition ICMJE |
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Intervention ICMJE |
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Study Arm (s) |
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Publications * | |||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Active, not recruiting | ||||
Enrollment ICMJE | 118 | ||||
Estimated Completion Date | December 2014 | ||||
Primary Completion Date | June 2011 (final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply |
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Gender | Both | ||||
Ages | 18 Years and older | ||||
Accepts Healthy Volunteers | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Location Countries ICMJE | United States, Canada, France, Germany, Italy, Japan, Netherlands, Poland, Russian Federation, Spain, United Kingdom | ||||
Administrative Information | |||||
NCT Number ICMJE | NCT00790400 | ||||
Other Study ID Numbers ICMJE | CRAD001M2302 | ||||
Has Data Monitoring Committee | |||||
Responsible Party | Novartis ( Novartis Pharmaceuticals ) | ||||
Study Sponsor ICMJE | Novartis Pharmaceuticals | ||||
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Investigators ICMJE |
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Information Provided By | Novartis | ||||
Verification Date | July 2012 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |