The metformin in tuberous sclerosis (MiTS) study: A randomised double-blind placebo-controlled trial.

ABSTRACT:

Background

Tuberous Sclerosis Complex (TSC) is a genetic disorder characterised by the development of benign tumours secondary to loss of inhibitory regulation of the mTOR (mechanistic Target of Rapamycin) intracellular growth pathway. Metformin inhibits the mTOR pathway. We investigated whether metformin would reduce growth of hamartomas associated with tuberous sclerosis complex.

Methods

In this multicentre randomized, double-blind, placebo-controlled trial, patients with a clinical diagnosis of tuberous sclerosis, aged over 10 years and with at least one renal angiomyolipoma of greater than 1 cm in diameter were enrolled. Participants were randomly allocated (1:1) by a secure website to receive metformin or placebo for 12 months. The primary outcome was percentage volume

SUBMITTER: Amin S 

PROVIDER: S-EPMC7910694 | biostudies-literature | 2021 Feb

REPOSITORIES: biostudies-literature