Start of saracatinib trial in myositis ossificans

Partager sur

Since the discovery of the causative gene (ACVR1) in myositis ossificans or fibrodysplasia ossificans progressiva (FOP), several therapeutic avenues have been explored, including the potent ALK2/ACVR1 kinase inhibitor saracatinib:

  • this drug has the advantage of having been developed for the treatment of ovarian cancers with a good safety profile;
  • the current trial, codenamed STOPFOP, is a double-blind, randomized phase IIa study and is being conducted in Europe (but not in France);
  • the investigators hope to decrease the volume of heterotopic bone formations as evaluated by a bone scan;
  • twenty patients with FOP are being enrolled in the study, which will last six months.

 

Protocol paper: a multi-center, double-blinded, randomized, 6-month, placebo-controlled study followed by 12-month open label extension to evaluate the safety and efficacy of Saracatinib in Fibrodysplasia Ossificans Progressiva (STOPFOP). Smilde BJ, Stockklausner C, Keen R et al. BMC Musculoskelet Disord. 2022 Jun 1;23(1):519.

On the same topic