Last reviewed · How we verify
Rapamycin
At a glance
| Generic name | Rapamycin |
|---|---|
| Also known as | Sirolimus, Rapamune, X-Rapamune, Rapacan, Siromus |
| Sponsor | Institut National de la Santé Et de la Recherche Médicale, France |
| Target | Peptidyl-prolyl cis-trans isomerase FKBP3, Sodium/nucleoside cotransporter 1, ATP-binding cassette sub-family G member 2 |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
Approved indications
- Gorham's disease
- Lymphangiectasis
- Lymphangioma
- Lymphangiomatosis
- Prevention of Kidney Transplant Rejection
- Pulmonary lymphangioleiomyomatosis
- Renal cell carcinoma
- Tuberous sclerosis syndrome
Common side effects
Key clinical trials
- A Phase 2, Baseline-Controlled Study Evaluating the Safety and Efficacy of PTX-022 (Sirolimus) Topical Gel 3.9% in the Treatment of Cutaneous Venous Malformations (PHASE2)
- Testing Lutetium Lu 177 Dotatate in Patients With Somatostatin Receptor Positive Advanced Bronchial Neuroendocrine Tumors (PHASE2)
- Comparing Retreatment of 177Lu-DOTATATE PRRT Versus the Usual Treatment in Patients With Metastatic Unresectable Gastroenteropancreatic Neuroendocrine Tumors, NET RETREAT Trial (PHASE2)
- Combination Chemotherapy With or Without Temsirolimus in Treating Patients With Intermediate Risk Rhabdomyosarcoma (PHASE3)
- SELVA: A Phase 3 Study Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations (PHASE3)
- Risk-Based Therapy in Treating Younger Patients With Newly Diagnosed Liver Cancer (PHASE3)
- Immunotherapy in Combination With Prednisone and Sirolimus for Kidney Transplant Recipients With Unresectable or Metastatic Skin Cancer (PHASE1, PHASE2)
- A Phase II Study of Sunitinib or Temsirolimus in Patients With Advanced Rare Tumours (PHASE2)
Primary sources
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| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |