Clinical trial · Interventional
Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM
A Trial of the Efficacy and Safety of Sirolimus(Rapamycin)Therapy for Renal Angiomyolipmoas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
NCT00490789CI-TRIAL-00001431TESSTALunknownPhase 2ClinicalTrials.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 this study is to determine the safety and efficacy of the mTOR inhibitor sirolimus as a treatment for renal angiomyolipomas in patients with tyberous 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 | Lymphangioleiomyomatosis | ONTOLOGY_EXACT | 0.98 |
| Tuberous Sclerosis | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| sirolimus | Drug | Sirolimus | ALIAS |
Design
Arms and outcomes
Arms (0)
[]Primary outcomes (2)
- measure
- longest diameter of renal angiomyolipomas assessed by MRI scan, toxicity graded by National Cancer Institute's Common Terminology Criteria for Adverse Events v3.0
- timeFrame
- assessments at baseline and 2,6,12 and 24 months
- measure
- toxicity graded by National Cancer Institute's Common Terminology Criteria for Adverse Events
- timeFrame
- throughout study
Secondary outcomes (1)
- measure
- respiratory function tests (FEV1, FVC, DLCO), cognitive function (memory, executive skills)
- timeFrame
- 2 years
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
- Maximum age
- 65 Years
Show eligibility criteria text
Inclusion Criteria: * If female, documentation of negative pregnancy test prior to enrolment. * Participants, including males, must use an effective form of contraception, whilst taking sirolimus and for twelve weeks after stopping the drug * One or more renal angiomyolipomata of at least two centimetres or greater in largest diameter * Adequate renal function :glomerular filtration rate \> 40 ml/min * Clinically definite diagnosis of tuberous sclerosis (modified Gomez criteria) or sporadic LAM (biopsy-proven or compatible high resolution chest CT scan and respiratory function tests.) * Signed and dated informed consent Exclusion Criteria: * History of non-compliance or inability to give informed consent * Significant haematological or hepatic abnormality (i.e. transaminase levels \> 150 i.u./L serum albumin \< 30 g/L, haematocrit\< 30%, platelets \< 100,000/ mm3, adjusted absolute neutrophil count \< 1,500/mm3, total WBC \< 3,000/ mm3) * Greater than 1 g proteinuria daily * Multiple bilateral AMLs, where individual lesions cannot be distinguished * Renal haemorrhage within preceding year * In those who have had a renal haemorrhage, known conservatively managed renal aneurysm(s) greater than 10mm * Patients who have had embolisation for AML(s) within the preceding 6 months * Patients who are unable to walk 100 metres on the flat * Continuous requirement for supplemental oxygen * Patients who have had or are being considered for organ transplant * Uncontrolled hyperlipidaemia * Intercurrent infection at initiation of Sirolimus * Surgery within last 2 months * Pregnant or lactating women * Use of an investigational drug within the last 30 days * Change in anti epileptic drug medication within the last 3 months * Likely to need vaccination e.g. for travel during the course of the trial (except for influenza vaccine in patients with LAM) * Current usage of strong inhibitors of CYP3AE ( such as ketoconazole, voriconazole, itraconazole, tilithromycin or clarithromycin) or strong inducers (such as rifampicin or rifabutin)
References
Publications (1)
- DERIVEDDavies DM, de Vries PJ, Johnson SR, McCartney DL, Cox JA, Serra AL, Watson PC, Howe CJ, Doyle T, Pointon K, Cross JJ, Tattersfield AE, Kingswood JC, Sampson JR. Sirolimus therapy for angiomyolipoma in tuberous sclerosis and sporadic lymphangioleiomyomatosis: a phase 2 trial. Clin Cancer Res. 2011 Jun 15;17(12):4071-81. doi: 10.1158/1078-0432.CCR-11-0445. Epub 2011 Apr 27. PMID 21525172