Special considerations for clinical trials in fibrodysplasia ossificans progressiva (FOP)
| Autor: | Robert J. Diecidue, Edward C. Hsiao, Clive S. Friedman, Keqin Zhang, Elisabeth M.W. Eekhoff, Tae Joon Cho, Mona Al Mukaddam, Richard Keen, Zvi Grunwald, Maja Di Rocco, Coen Netelenbos, Matthew A. Brown, Amanda Cali, F.S. Kaplan, Patricia Delai, Geneviève Baujat, Michael Zasloff, Carmen De Cunto, Robert J. Pignolo, Rolf Morhart, Nobuhiko Haga, Christiaan Scott |
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| Přispěvatelé: | Internal medicine, ACS - Diabetes & metabolism, AGEM - Endocrinology, metabolism and nutrition, Amsterdam Movement Sciences - Rehabilitation & Development |
| Jazyk: | angličtina |
| Rok vydání: | 2018 |
| Předmět: |
Research design
medicine.medical_specialty rare disease clinical trials Consensus Ossification Orphan diseases 030226 pharmacology & pharmacy 03 medical and health sciences Patient safety 0302 clinical medicine Stakeholder Participation patient safety Medicine Humans Pharmacology (medical) 030212 general & internal medicine Pharmacology & Pharmacy Intensive care medicine Pharmacology Clinical Trials as Topic business.industry Ossification Heterotopic Patient Selection Review‐themed Issue Myositis ossificans Pharmacology and Pharmaceutical Sciences medicine.disease Fibrodysplasia ossificans progressiva (FOP) Clinical trial Clinical research Myositis Ossificans Research Design Fibrodysplasia ossificans progressiva Heterotopic ossification Heterotopic Bone Remodeling business |
| Zdroj: | British Journal of Clinical Pharmacology British Journal of Clinical Pharmacology, 85(6), 1199-1207. Wiley-Blackwell British journal of clinical pharmacology, vol 85, iss 6 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, Friedman, C S, Kaplan, F S & Eekhoff, E M W 2019, ' Special considerations for clinical trials in fibrodysplasia ossificans progressiva (FOP) ', British Journal of Clinical Pharmacology, vol. 85, no. 6, pp. 1199-1207 . https://doi.org/10.1111/bcp.13777 |
| ISSN: | 1365-2125 0306-5251 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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