Special considerations for clinical trials in fibrodysplasia ossificans progressiva (FOP)

• Published in: British journal of clinical pharmacology Vol. 85; no. 6; pp. 1199 - 1207
• Main Authors: Hsiao, Edward C., Di Rocco, Maja, Cali, Amanda, Zasloff, Michael, Al Mukaddam, Mona, Pignolo, Robert J., Grunwald, Zvi, Netelenbos, Coen, Keen, Richard, Baujat, Genevieve, Brown, Matthew A., Cho, Tae‐Joon, De Cunto, Carmen, Delai, Patricia, Haga, Nobuhiko, Morhart, Rolf, Scott, Christiaan, Zhang, Keqin, Diecidue, Robert J., Friedman, Clive S., Kaplan, Fredrick S., Eekhoff, Elisabeth M.W.
• Format: Journal Article
• Language: English
• Published: England John Wiley and Sons Inc 01.06.2019
• Subjects: Bone Remodeling - drug effects; Clinical Trials as Topic - methods; Consensus; Fibrodysplasia ossificans progressiva (FOP); Humans; Myositis Ossificans - diagnosis; Myositis Ossificans - drug therapy; Myositis Ossificans - physiopathology; Ossification, Heterotopic - diagnosis; Ossification, Heterotopic - drug therapy; Ossification, Heterotopic - physiopathology; Patient Safety; Patient Selection; rare disease clinical trials; Research Design; Review‐themed Issue; Stakeholder Participation; rare disease clinical trials; patient safety; Fibrodysplasia ossificans progressiva (FOP)
• ISSN: 0306-5251; 1365-2125; 1365-2125
• DOI: 10.1111/bcp.13777

Clinical trials for orphan diseases are critical for developing effective therapies. One such condition, fibrodysplasia ossificans progressiva (FOP; MIM#135100), is characterized by progressive heterotopic ossification (HO) that leads to severe disability. Individuals with FOP are extremely sensitive to even minor traumatic events. There has been substantial recent interest in clinical trials for novel and urgently‐needed treatments for FOP. The International Clinical Council on FOP (ICC) was established in 2016 to provide consolidated and coordinated advice on the best practices for clinical care and clinical research for individuals who suffer from FOP. The Clinical Trials Committee of the ICC developed a focused list of key considerations that encompass the specific and unique needs of the FOP community – considerations that are endorsed by the entire ICC. These considerations complement established protocols for developing and executing robust clinical trials by providing a foundation for helping to ensure the safety of subjects with FOP in clinical research trials.