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

Edward C. Hsiao, Maja Di Rocco, Amanda Cali, Michael Zasloff, Mona Al Mukaddam, Robert 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 journalArticle

2 Citations (Scopus)

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)
JournalBritish Journal of Clinical Pharmacology
DOIs
StateAccepted/In press - Jan 1 2018

Fingerprint

Myositis Ossificans
Clinical Trials
Heterotopic Ossification
Rare Diseases
Practice Guidelines
Research
Safety

Keywords

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

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)

Cite this

Special considerations for clinical trials in fibrodysplasia ossificans progressiva (FOP). / Hsiao, Edward C.; Di Rocco, Maja; Cali, Amanda; Zasloff, Michael; Al Mukaddam, Mona; Pignolo, Robert; 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.

In: British Journal of Clinical Pharmacology, 01.01.2018.

Research output: Contribution to journalArticle

Hsiao, EC, Di Rocco, M, Cali, A, Zasloff, M, Al Mukaddam, M, Pignolo, R, Grunwald, Z, Netelenbos, C, Keen, R, Baujat, G, Brown, MA, Cho, TJ, De Cunto, C, Delai, P, Haga, N, Morhart, R, Scott, C, Zhang, K, Diecidue, RJ, Friedman, CS, Kaplan, FS & Eekhoff, EMW 2018, 'Special considerations for clinical trials in fibrodysplasia ossificans progressiva (FOP)', British Journal of Clinical Pharmacology. https://doi.org/10.1111/bcp.13777
Hsiao, Edward C. ; Di Rocco, Maja ; Cali, Amanda ; Zasloff, Michael ; Al Mukaddam, Mona ; Pignolo, Robert ; 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. / Special considerations for clinical trials in fibrodysplasia ossificans progressiva (FOP). In: British Journal of Clinical Pharmacology. 2018.
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