A Phase II Study of Everolimus, Leuprolide/Letrozole, and Everolimus/Leuprolide/Letrozole in Patients with Inoperable Fibrolamellar Hepatocellular Carcinoma

Full Title
A Randomized Three Arm Phase II Study of (1) Everolimus, (2) Estrogen Deprivation Therapy (EDT) with Leuprolide + Letrozole and (3) Everolimus + EDT in Patients with Unresectable Fibrolamellar Hepatocellular Carcinoma (FLL-HCC)
Disease Status
Newly Diagnosed & Relapsed/Refractory

There is no effective treatment for fibrolamellar liver cancer that cannot be removed surgically. In this study, researchers are evaluating different combinations of the drugs everolimus, letrozole, and leuprolide to treat patients with inoperable fibrolamellar liver cancer.

Leuprolide and letrozole inhibit the production of the hormone estrogen, which can fuel the growth of fibrolamellar liver cancer. Everolimus works by inhibiting mTOR, a protein that drives cancer growth.

In this study, patients will be randomly assigned to one of three treatment groups: (1) everolimus alone, (2) leuprolide and letrozole, or (3) everolimus, leuprolide, and letrozole. Patients in groups 1 and 2 who do not do well on those regimens have the option of receiving all three drugs together.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have a confirmed diagnosis of fibrolamellar hepatocellular carcinoma that cannot be surgically removed.
  • Prior therapy is allowed, but all previous treatments must have been completed at least 4 weeks before entering the study, and patients must have recovered from any serious side effects.
  • Patients must be able to be ambulatory for more than half of their normal waking hours.
  • Patients must be age 12 or older.

For more information and to inquire about eligibility for this study, please contact Dr. Ghassan Abou-Alfa at 646-888-4184.

Hepatobiliary: Liver Cancer
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