Additional Clinical Trial Site Opens in UK
Clementia Pharmaceuticals, Inc. today announced the initiation of enrollment of children as young as 6 years old in the company's ongoing Phase 2 study of palovarotene for the treatment of fibrodysplasia ossificans progressiva (FOP). The company also announced the addition of a clinical trial site in the United Kingdom that should ease the travel burden for affected individuals living in the European Union. FOP is an extremely rare and severely disabling congenital myopathy characterized by heterotopic ossification (HO), the abnormal growth of bone in muscles, tendons and ligaments, causing significant morbidities and progressive disability. There are currently no approved treatments for FOP.
Clementia expanded enrollment to include children with FOP in the ongoing Phase 2 clinical trial following completion of the appropriate nonclinical studies. In addition to the 24 patients already enrolled in this adaptive-design, dose-ranging study, an additional 16 patients will be enrolled, with at least eight being between the ages of 6 and 14. The company anticipates completion of enrollment by the end of 2015.
"The inclusion of children as young as 6 years old in the ongoing Phase 2 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," commented Donna Grogan, M.D., Chief Medical Officer of Clementia. "Once the Phase 2 study is complete, we will analyze the data as quickly as possible to inform the design of our planned Phase 3 study, which we anticipate starting in the second half of 2016."
Clementia also announced the opening of a fourth clinical trial site at Royal National Orthopaedic Hospital in London, United Kingdom for the ongoing Phase 2 clinical trial, with Dr. Richard Keen as the principal investigator.
"We are pleased to participate in this important research to further serve patients living in Europe," noted Dr. Richard Keen, Royal National Orthopaedic Hospital. Chris Bedford-Gay, father of a young child with FOP and founder of FOP Friends® the UK Charity and FOP support group, commented, "We appreciate Clementia's commitment in accelerating the development of a potential treatment for FOP, one that cannot come quickly enough to families like ours."
At this time, 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. UCSF is the first site to open enrollment to children with FOP age 6 and older who meet eligibility criteria. All other sites plan to initiate enrollment of younger children as they receive approvals from their respective institutions.
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