Clementia Pharmaceuticals announced the initiation of a natural history study in patients with fibrodysplasia ossificans progressiva (FOP), a rare, severely disabling genetic disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) and new abnormal bone formation. This process, known as heterotopic ossification (HO), occurs in muscles, tendons and ligaments, and causes significant morbidities and progressive disability.
The multi-center, non-interventional, 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 and physical function as assessed by range of motion and quality of life. The prospective design of the study will allow researchers to monitor flare-up progression and determine the annual rate of flare-ups over three years. Seven international sites will enroll a total of 50 children and adults with FOP. The University of California, San Francisco, and the Hospital Italiano de Buenos Aires are currently recruiting patients for this study.
“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,” said Betsy Bogard, Global Research Development Director for the International FOP Association (IFOPA). “Clementia has agreed to share their natural history study data with the IFOPA’s planned patient registry. We applaud and thank Clementia for undertaking this important study and for their collaborative and patient-centered approach in sharing it with us. We urge patients and families affected by FOP to learn more about this research.”
The natural history study is being conducted in parallel with Clementia’s interventional trials with palovarotene, an investigational retinoic acid receptor gamma agonist.
“Until now, investigators studying FOP have relied primarily on basic research, case reports, and results from patient surveys to understand this disease,” noted Robert Pignolo, M.D., Ph.D., Associate Professor of Medicine and Orthopaedic Surgery at the University of Pennsylvania Perelman School of Medicine. “This is the first prospective, longitudinal study to perform total body imaging and other functional assessments in patients with FOP in a systematic manner. The standardized data generated by the natural history study will guide ongoing drug development in FOP, including clinical trials of palovarotene.”
“We are grateful to the investigators and the IFOPA for their collaboration on the design of the natural history study, which includes the first patient-reported outcomes questionnaire developed specifically for FOP,” commented Donna Grogan, M.D., chief medical officer of Clementia. “With this input from the community, we are confident the study will yield meaningful results that will advance the understanding of FOP and inform our ongoing development of palovarotene. We are also continuing our collaboration with the IFOPA by providing data from this study for its patient registry.”
Source: Clementia Pharmaceuticals