Clinical trial · Interventional
Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)
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)
NCT00790400CI-TRIAL-00026016EXIST-2completedPhase 3Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This study will evaluate the safety and efficacy of RAD001 in treating patients with Angiomyolipoma associated with Tuberous Sclerosis Complex or Sporadic Lymphangioleiomyomatosis.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Lymphangioleiomyomatosis (LAM) | Lymphangioleiomyomatosis | ONTOLOGY_EXACT | 0.85 |
| Tuberous Sclerosis Complex (TSC) | — | UNRESOLVED | — |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Everolimus Placebo | Drug | — | UNRESOLVED |
| Everolimus (RAD001) | Drug | Everolimus | ALIAS |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Everolimus
- description
- Study drug was given by continuous oral daily dosing of two 5 mg tablets.
- interventionNames
- Drug: Everolimus (RAD001)
- type
- PLACEBO_COMPARATOR
- label
- Placebo
- description
- Placebo was given by continuous oral daily dosing of two 5 mg tablets.
- interventionNames
- Drug: Everolimus Placebo
Primary outcomes (1)
- measure
- Angiomyolipoma Response Rate as Per Central Radiology Review
- timeFrame
- 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 up to 5.7 years
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Male or Female 18 years or older * Clinically definite diagnosis of Tuberous Sclerosis Complex according to the modified Gomez criteria or sporadic LAM (biopsy-proven or compatible chest CT scan) * Clinically definite diagnosis of renal angiomyolipoma * At least one Angiomyolipoma of ≥ 3 cm in its longest diameter using CT or MRI * Females of child bearing potential must use birth control and have documentation of negative pregnancy test * Written informed consent according to local guidelines Exclusion Criteria: * Recent heart attack, cardiac related chest pain or stroke * Severely impaired lung function * Bleeding related to angiomyolipoma or embolization during 6 months prior to randomization * Clinically significant chylous ascites * Clinically significant hematological or hepatic abnormality * 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 * Recent use of an investigational drug * Bleeding diathesis or on oral anti-vitamin K medication * Uncontrolled high cholesterol * Uncontrolled diabetes * HIV * Inability to attend scheduled clinic visits * Patients with metal implants thus prohibiting MRI evaluations * Angiomyolipoma which requires surgery at the time of randomization * History of malignancy * Severe or uncontrolled medical conditions which would cause an unacceptable safety risk or compromise compliance with the protocol
References
Publications (5)
- BACKGROUNDBissler JJ, Kingswood JC, Radzikowska E, Zonnenberg BA, Frost M, Belousova E, Sauter M, Nonomura N, Brakemeier S, de Vries PJ, Whittemore VH, Chen D, Sahmoud T, Shah G, Lincy J, Lebwohl D, Budde K. Everolimus for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis (EXIST-2): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet. 2013 Mar 9;381(9869):817-24. doi: 10.1016/S0140-6736(12)61767-X. PMID 23312829
- DERIVEDBissler JJ, Nonomura N, Budde K, Zonnenberg BA, Fischereder M, Voi M, Louveau AL, Herbst F, Bebin EM, Curatolo P, Zonta A, Belousova E. Angiomyolipoma rebound tumor growth after discontinuation of everolimus in patients with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis. PLoS One. 2018 Sep 7;13(9):e0201005. doi: 10.1371/journal.pone.0201005. eCollection 2018. PMID 30192751
- DERIVEDBissler JJ, Budde K, Sauter M, Franz DN, Zonnenberg BA, Frost MD, Belousova E, Berkowitz N, Ridolfi A, Christopher Kingswood J. Effect of everolimus on renal function in patients with tuberous sclerosis complex: evidence from EXIST-1 and EXIST-2. Nephrol Dial Transplant. 2019 Jun 1;34(6):1000-1008. doi: 10.1093/ndt/gfy132. PMID 30053159
- DERIVEDSparagana S, Franz DN, Krueger DA, Bissler JJ, Berkowitz N, Burock K, Kingswood JC. Pooled analysis of menstrual irregularities from three major clinical studies evaluating everolimus for the treatment of tuberous sclerosis complex. PLoS One. 2017 Oct 12;12(10):e0186235. doi: 10.1371/journal.pone.0186235. eCollection 2017. PMID 29023494
- DERIVEDBissler JJ, Kingswood JC, Radzikowska E, Zonnenberg BA, Frost M, Belousova E, Sauter M, Nonomura N, Brakemeier S, de Vries PJ, Berkowitz N, Miao S, Segal S, Peyrard S, Budde K. Everolimus for renal angiomyolipoma in patients with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis: extension of a randomized controlled trial. Nephrol Dial Transplant. 2016 Jan;31(1):111-9. doi: 10.1093/ndt/gfv249. Epub 2015 Jul 8. PMID 26156073