Clinical trial · Interventional
Everolimus (RAD001) Therapy of Giant Cell Astrocytoma in Patients With Tuberous Sclerosis Complex
Everolimus (RAD001)Therapy of Giant Cell Astrocytomas in Patients With Tuberous Sclerosis Complex
NCT00411619CI-TRIAL-00015860completedPhase 1 / Phase 2Results 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)
The purpose of the study is to evaluate the safety and potential side effects of everolimus (an experimental drug) on a person with Tuberous Sclerosis Complex who also has been diagnosed with a brain tumor (astrocytoma) The hypothesis is that the drug will cause the tumor size to decrease, and may have beneficial activity separate from effects on tumors in patients.
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 |
|---|---|---|---|
| Subependymal Giant Cell Astrocytoma | Subependymal Giant Cell Astrocytoma | ONTOLOGY_EXACT | 0.90 |
| Tuberous Sclerosis | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Everolimus | Drug | Everolimus | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Everolimus
- description
- As this was a non-randomized, open-label, single arm study, all patients in the study received treatment with everolilmus
- interventionNames
- Drug: Everolimus
Primary outcomes (1)
- measure
- Number With Observed Adverse Side Effects
- timeFrame
- During the entire study
Secondary outcomes (1)
- measure
- Overall Reduction in SEGA Tumor Volume.
- timeFrame
- During the entire study
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 3 Years
Show eligibility criteria text
Inclusion Criteria: * Age three years and older * If female and of child bearing potential, documentation of negative pregnancy test prior to enrollment. In the extension phase, all females of child bearing potential will be required to take monthly home pregnancy tests and record results on a provided diary card. * Clinically definite diagnosis of tuberous sclerosis (modified Gomez criteria or positive genetic test) * Presence of giant cell astrocytoma as defined by imaging characteristics and serial increase in size of lesion on 2 or more MRI scans * Adequate renal function (creatinine \< 1.5 mg/dl) Exclusion Criteria: * Serious intercurrent medical illness or other uncontrolled medical disease which could compromise participation in the study * Significant hematologic or hepatic abnormality * Continuous requirement for supplemental oxygen * Intercurrent infection at initiation of everolimus * Embolization of angiomyolipoma within one month; any other recent surgery within 2 months of initiation of everolimus * Pregnant or lactating women * Use of an investigational drug within the last 30 days * Must be adequately recovered from the acute toxicities of any prior therapy * Clinical evidence of impending herniation or focal neurologic deficit related to the subject's astrocytoma * Unwilling or unable to use highly effective contraception
References
Publications (6)
- DERIVEDPeters JM, Prohl A, Kapur K, Nath A, Scherrer B, Clancy S, Prabhu SP, Sahin M, Franz DN, Warfield SK, Krueger DA. Longitudinal Effects of Everolimus on White Matter Diffusion in Tuberous Sclerosis Complex. Pediatr Neurol. 2019 Jan;90:24-30. doi: 10.1016/j.pediatrneurol.2018.10.005. Epub 2018 Oct 18. PMID 30424962
- 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
- DERIVEDFranz DN, Agricola K, Mays M, Tudor C, Care MM, Holland-Bouley K, Berkowitz N, Miao S, Peyrard S, Krueger DA. Everolimus for subependymal giant cell astrocytoma: 5-year final analysis. Ann Neurol. 2015 Dec;78(6):929-38. doi: 10.1002/ana.24523. Epub 2015 Nov 9. PMID 26381530
- DERIVEDKrueger DA, Care MM, Agricola K, Tudor C, Mays M, Franz DN. Everolimus long-term safety and efficacy in subependymal giant cell astrocytoma. Neurology. 2013 Feb 5;80(6):574-80. doi: 10.1212/WNL.0b013e3182815428. Epub 2013 Jan 16. PMID 23325902
- DERIVEDTillema JM, Leach JL, Krueger DA, Franz DN. Everolimus alters white matter diffusion in tuberous sclerosis complex. Neurology. 2012 Feb 21;78(8):526-31. doi: 10.1212/WNL.0b013e318247ca8d. Epub 2012 Jan 18. PMID 22262746
- DERIVEDKrueger DA, Care MM, Holland K, Agricola K, Tudor C, Mangeshkar P, Wilson KA, Byars A, Sahmoud T, Franz DN. Everolimus for subependymal giant-cell astrocytomas in tuberous sclerosis. N Engl J Med. 2010 Nov 4;363(19):1801-11. doi: 10.1056/NEJMoa1001671. PMID 21047224