Clinical trial · Interventional
Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM
Rapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis
NCT00457808CI-TRIAL-00000666completedPhase 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 was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam.
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 |
|---|---|---|---|
| Rapamycin, sirolimus | Drug | Sirolimus | ALIAS |
Design
Arms and outcomes
Arms (0)
[]Primary outcomes (1)
- measure
- Volume of Angiomyolipomata
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
- Maximum age
- 65 Years
Show eligibility criteria text
Inclusion Criteria: * Diagnosed with angiomyolipomas and have either Tuberous Sclerosis complex or Lymphangioleiomyomatosis * Between the gaes of 18 and 65 years * Competency to voluntarily consent * Clinically definite diagnosis of tuberous sclerosis or S-LAM * Adequate contraception * At least one angiomyolipoma of 1 cm or greater in largest diameter Exclusion Criteria: * Use of continuous supplemental oxygen * Concurrent infection * Recent surgery * Ongoing or planned pregnancy * Lactation * Use of an investigational drug within the last 30 days of study entrance
References
Publications (1)
- DERIVEDBissler JJ, McCormack FX, Young LR, Elwing JM, Chuck G, Leonard JM, Schmithorst VJ, Laor T, Brody AS, Bean J, Salisbury S, Franz DN. Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis. N Engl J Med. 2008 Jan 10;358(2):140-51. doi: 10.1056/NEJMoa063564. PMID 18184959