Special considerations for clinical trials in fibrodysplasia ossificans progressiva (FOP). Issue 6 (6th November 2018)

• Record Type: Journal Article
• 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.
• Is Part Of: British journal of clinical pharmacology. Volume 85:Issue 6(2019)
• Page Start: 1199
• Page End: 1207
• Publication Date: 2018-11-06
• Subjects: Fibrodysplasia ossificans progressiva (FOP) -- patient safety -- rare disease clinical trials; Pharmacology -- Periodicals; Drugs -- Periodicals; 615.1
• DOI: 10.1111/bcp.13777
• Languages: English
• ISSNs: 0306-5251

Abstract

Abstract : 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. Abstract :