RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMAS
Condition: Nonmalignant Neoplasm; Tuberous Sclerosis; Lymphangioleimyomatosis; Kidney Angiomyolipoma
MeSH terms: Tuberous Sclerosis; Angiomyolipoma
Study type: Interventional
Phase: Phase 2

Summary: 
      This research study is evaluating a drug called rapamycin as a possible treatment for the
      lumps (or tumors) that form in the kidneys, called angiomyolipomas, in people who have either
      TSC or LAM. Kidney angiomyolipomas are tumors that are made up of blood vessels, muscle and
      fat. Rapamycin has been approved to treat other diseases, but it is investigational for
      treating kidney angiomyolipomas.

      Investigational means that it is being as a possible treatment for kidney angiomyolipomas but
      is not currently approved by the U.S. Food and Drug Administration (FDA) for treating this
      disease.
    

Detailed description: 
      This research study is a Phase ll clinical trial. Phase II clinical trials test the
      effectiveness of an investigational drug to learn whether the drug works in treating a
      specific disease. "Investigational" means that the drug is still being studied and that
      research doctors are trying to find out more about it-such as the safest dose to use, the
      side effects it may cause, and if the drug is effective for treating different types of
      diseases. It also means that the FDA (the U.S. Food and Drug Administration) has not yet
      approved this drug for this type of disease.

      The purpose of this research study is to see if the drug, rapamycin, is effective in treating
      kidney angiomyolipomas. Rapamycin is a drug that may decrease the size of tumors. This drug
      has been used in treating other diseases and cancers in humans and information from those
      other research studies suggests that rapamycin may help to shrink tumors in this research
      study.

      The investigators will also be looking at the safety of this drug in people with TSC or LAM.
      In addition, the investigators will also look at whether this drug is effective for treating
      other conditions that occur in people that have TSC or LAM (tubers, subependymal giant cell
      astrocytomos, facial angiofibromas, kidney cysts in TSC, or symptoms of cough, breathing
      trouble or other symptoms of lung problems in people with LAM).

      Although these tumors (kidney angiomyolipomas) may not change for many years, they do not go
      away on their own. They often grow larger and may bleed. The standard of care for treating
      these tumors is to take pictures of them using ultrasound, CT or MRI (called kidney imaging)
      every 1-3 years to see if they are getting larger or if they are bleeding. If they do grow
      larger or bleed, removing them by surgery or cutting off the blood supply to the tumor
      (vascular embolization) may be recommended.

      This research study is being done because there are no medicines available at this time that
      can treat kidney angiomyolipomas. However, recent studies in the laboratory have shown that
      the drug, rapamycin, maybe effective in treating these tumors. Because rapamycin is approved
      by the FDA for treating other medical conditions, we know what doses are safe to use based on
      guidelines that have been approved by the FDA.
    

Intervention name(s): Rapamycin
Intervention type(s): Drug
Other intervention name(s): Rapamune; Sirolimus
Intervention MeSH terms: Sirolimus
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: angiomyolipoma