ODP103 IPN60130 for the Treatment of Fibrodysplasia Ossificans Progressiva: Methodology of the Randomized, Double-Blind, Placebo-Controlled Phase II FALKON Trial

ABSTRACT: Abstract

Objectives

Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder caused by activin receptor-like kinase-2/activin A receptor type 1(ALK2/ACVR1) mutation and characterized by heterotopic ossification (HO) inducing progressive restriction of mobility. IPN60130 is a selective ALK2/ACVR1 inhibitor, currently being investigated for the treatment of FOP. 1 Here, we describe methodology of the FALKON trial (NCT05039515) designed to compare efficacy and safety of IPN60130 with placebo in patients with FOP.

Methods

Patients will be randomized to oral placebo, or low or high dose IPN60130 for 12 months; patients receiving placebo will then transition to IPN60130 for 12 months. Patients will be randomized to receive placebo, or low or high dose IPN60

SUBMITTER:  

PROVIDER: S-EPMC9624752 | biostudies-literature | 2022 Nov

REPOSITORIES: biostudies-literature