PEComa Clinical Trials and Treatments

PEComa stands for perivascular epithelioid cell tumor. The tumor cells sit around small blood vessels and carry markers of both smooth muscle and pigment-making cells. That mix is how a pathologist recognizes a PEComa under the microscope, usually after the tumor has been removed or biopsied.

The most common symptoms of PEComa include A lump or mass, which may or may not hurt, Vaginal bleeding, when the tumor grows in the uterus, Belly pain, blood in the stool or a blocked bowel, when it grows in the digestive tract, Weight loss and low red blood cell counts (anemia), No symptoms at all, since some PEComas are found on a scan done for another reason. Malignant PEComa is diagnosed in about 0.12 to 0.24 people per million (NORD). The wider PEComa family includes kidney angiomyolipomas, which are far more common and are found in about 0.44% of people. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for PEComa from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. PEComa is so rare that most trials include it alongside other rare sarcomas or tumors. Dana-Farber in Boston is running a Phase 2 trial of elacestrant, an estrogen-blocking pill (NCT07467772).

Yes, Fyarro (sirolimus protein-bound particles) is the only FDA-approved drug for malignant PEComa. It was approved on November 22, 2021 for adults whose tumor has spread or cannot be removed by surgery. It is given by IV on days 1 and 8 of every 21-day cycle. Oral sirolimus and everolimus are sometimes used off label. Related tumors in the same family have their own approvals: everolimus for kidney angiomyolipomas in tuberous sclerosis complex and oral sirolimus for LAM.

PEComa arises from acquired (somatic) genetic changes in the affected cells, rather than from an inherited variant. The useful test is usually of the tumor: in the AMPECT trial, 8 of 9 people whose tumors had a TSC2 change responded to Fyarro. A few PEComas occur with tuberous sclerosis complex, which a blood test for TSC1 and TSC2 can check. Malignant PEComa is diagnosed in about 0.12 to 0.24 people per million (NORD). The wider PEComa family includes kidney angiomyolipomas, which are far more common and are found in about 0.44% of people.

For PEComa the genetic testing that matters is usually of the tumor or affected blood cells rather than inherited DNA. The useful test is usually of the tumor: in the AMPECT trial, 8 of 9 people whose tumors had a TSC2 change responded to Fyarro. A few PEComas occur with tuberous sclerosis complex, which a blood test for TSC1 and TSC2 can check.

Those results guide treatment choice and are often the entry requirement for targeted-therapy trials, so ask whether the biopsy was sent for molecular testing and request the report. The Genetic Testing section of this page lists laboratories in the NIH Genetic Testing Registry.

Eligibility for PEComa clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a PEComa clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.