Clementia Pharmaceuticals commences multi-center study of patients with fibrodysplasia ossificans progressiva

Clementia Pharmaceuticals, Inc.May 28 2015

Study Now Enrolling Patients Ages Two through 65 Years Old

Clementia Pharmaceuticals, Inc. today announced that it has commenced enrollment in the second part (Part B) of its natural history study in patients with fibrodysplasia ossificans progressiva (FOP), a rare, severely disabling congenital myopathy characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in the formation of new, abnormal (heterotopic) bone in muscles, tendons and ligaments.

The multi-center, non-interventional, two-part longitudinal study is designed to measure disease progression over three years in patients with FOP. The study will evaluate the relationship between abnormal bone formation, which progressively restricts movement, and physical function as assessed by range of motion and patient-reported outcomes. Part B will enroll 40 patients from two to 65 years of age.

A committee comprised of FOP and imaging experts reviewed data from ten patients in the first part of the study (Part A), and determined that low-dose whole body CT scan (excluding head) provided better assessment of presence and amount of heterotopic bone throughout the body than bone density (DEXA) scans and CT scout scans. Based on these findings, low-dose whole body CT scan (excluding head) will be used in Part B to detect the presence and progression of heterotopic bone.

"The commencement of Part B of the natural history study brings us closer to our goal of advancing the global community's understanding of FOP and developing a treatment for this debilitating disease," said Donna Grogan, M.D., Chief Medical Officer of Clementia. "The enrollment of children will enable us to provide a more complete picture of FOP's natural progression."

The natural history study, which was designed in collaboration with investigators and with the assistance of the International FOP Association (IFOPA), is being conducted in parallel with Clementia's interventional trials with palovarotene, an investigational agent for the prevention of abnormal bone formation following a flare-up. Importantly, data from the natural history study will be shared with the IFOPA to advance its mission of finding a cure for FOP.