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news.Canada-based Clementia Pharmaceuticals has started enrolling children aged six years in its ongoing Phase II trial of palovarotene to treat fibrodysplasia ossificans progressiva (FOP), an extremely rare and severely disabling congenital myopathy.
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An additional clinical trial site has been added by the company in the UK to ease travel burden for affected individuals living in the European Union (EU).
FOP is characterized by heterotopic ossification (HO), the abnormal growth of bone in muscles, tendons and ligaments, causing significant morbidities and progressive disability. Currently, there are no approved treatments for FOP.
The addition of children with FOP in the ongoing Phase II trial follows the completion of the appropriate nonclinical studies.
Apart from the 24 patients already enrolled in this adaptive-design, dose-ranging trial, an additional 16 patients will be enrolled, with at least eight being between the ages of 6 and 14.
The company intends to complete patient enrollment in this trial by the end of this year.
Clementia chief medical officer Donna Grogan said: "The inclusion of children as young as six years old in the ongoing Phase II clinical trial will enable a preliminary evaluation of the safety and efficacy of palovarotene in a younger population, a particularly important consideration given the early onset of FOP flare-ups.
"Once the Phase II study is complete, we will analyze the data as quickly as possible to inform the design of our planned Phase III study, which we anticipate starting in the second half of 2016."
The company has also announced the opening of a fourth clinical trial site at Royal National Orthopaedic Hospital in London, UK for the ongoing Phase II trial, with Dr Richard Keen as the principal investigator.
Currently, individuals with FOP age 15 or older can enroll at the UK site as well as all other sites, which include the University of California San Francisco (UCSF), University of Pennsylvania in Philadelphia, and Hopital Necker-Enfants Malades in Paris, France.