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Clementia begins natural history trial in FOP patients

Clementia Pharmaceuticals has started a natural history trial in patients with fibrodysplasia ossificans progressiva (FOP), a rare, severely disabling genetic disease.

FOP is characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) and new abnormal bone formation.

The process called heterotopic ossification (HO) occurs in muscles, tendons and ligaments, and causes significant morbidities and progressive disability.

The multi-center, non-interventional, longitudinal trial will measure disease progression over three years in FOP patients.

The trial is designed to evaluate the relationship between abnormal bone formation and physical function as assessed by range of motion and quality of life.

The prospective design of the trial will allow researchers to monitor flare-up progression and determine the annual rate of flare-ups over three years.

A total of 50 children and adults with FOP will be enrolled in the trial at seven international sites.

The University of California and the Hospital Italiano de Buenos Aires are currently recruiting patients for the trial.

International FOP Association (IFOPA) Global Research Development director Betsy Bogard said: "The global FOP community is eager to gain new prospective data and insight that may lead to the improved understanding and treatment of this extremely rare, debilitating, and difficult-to-study disease."

The natural history trial is being carried out in parallel with the company’s interventional trials with palovarotene, an investigational retinoic acid receptor gamma agonist.

Clementia chief medical officer Donna Grogan said: "We are also continuing our collaboration with the IFOPA by providing data from this study for its patient registry."