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

Edward C. Hsiao, Maja Di Rocco, Amanda Cali, Michael Zasloff, Mona Al Mukaddam, Robert J. Pignolo, Zvi Grunwald, Coen Netelenbos, Richard Keen, Genevieve Baujat, Matthew A. Brown, Tae Joon Cho, Carmen De Cunto, Patricia Delai, Nobuhiko Haga, Rolf Morhart, Christiaan Scott, Keqin Zhang, Robert J. Diecidue, Clive S. FriedmanFredrick S. Kaplan, Elisabeth M.W. Eekhoff

Research output: Contribution to journalReview article

7 Scopus citations

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.

Original languageEnglish (US)
Pages (from-to)1199-1207
Number of pages9
JournalBritish Journal of Clinical Pharmacology
Volume85
Issue number6
DOIs
StatePublished - Jun 2019

Keywords

  • Fibrodysplasia ossificans progressiva (FOP)
  • patient safety
  • rare disease clinical trials

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)

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    Hsiao, E. C., Di Rocco, M., Cali, A., Zasloff, M., Al Mukaddam, M., Pignolo, R. J., Grunwald, Z., Netelenbos, C., Keen, R., Baujat, G., Brown, M. A., Cho, T. J., De Cunto, C., Delai, P., Haga, N., Morhart, R., Scott, C., Zhang, K., Diecidue, R. J., ... Eekhoff, E. M. W. (2019). Special considerations for clinical trials in fibrodysplasia ossificans progressiva (FOP). British Journal of Clinical Pharmacology, 85(6), 1199-1207. https://doi.org/10.1111/bcp.13777