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

被引：26

作者：

Hsiao, Edward C. ^{[1
,2
]}

Di Rocco, Maja ^{[3
]}

Cali, Amanda ^{[4
,5
]}

Zasloff, Michael ^{[6
,7
,8
]}

Al Mukaddam, Mona ^{[9
,10
]}

Pignolo, Robert J. ^{[11
]}

Grunwald, Zvi ^{[12
]}

Netelenbos, Coen ^{[13
]}

Keen, Richard ^{[14
]}

Baujat, Genevieve ^{[15
]}

Brown, Matthew A. ^{[16
]}

Cho, Tae-Joon ^{[17
]}

De Cunto, Carmen ^{[18
]}

Delai, Patricia ^{[19
]}

Haga, Nobuhiko ^{[20
]}

Morhart, Rolf ^{[21
]}

Scott, Christiaan ^{[22
]}

Zhang, Keqin ^{[23
]}

Diecidue, Robert J. ^{[24
]}

Friedman, Clive S. ^{[25
]}

Kaplan, Fredrick S. ^{[26
,27
]}

Eekhoff, Elisabeth M. W. ^{[13
]}

机构：

[1] Univ Calif San Francisco, Div Endocrinol & Metab, San Francisco, CA 94143 USA

[2] Univ Calif San Francisco, Dept Med, Inst Human Genet, San Francisco, CA USA

[3] IRCCS Giannina Gaslini Inst, Dept Pediat, Unit Rare Dis, Genoa, Italy

[4] Radiant Hope Fdn, Philadelphia, PA USA

[5] PENN Med, Ian Cali FOP Res Fund, Ctr Res FOP & Related Disorders, Philadelphia, PA USA

[6] Univ Penn, Sch Med, Ctr Res FOP & Related Disorders, Dept Orthopaed Surg, Philadelphia, PA 19104 USA

[7] Univ Penn, Sch Med, Ctr Res FOP & Related Disorders, Dept Genet, Philadelphia, PA 19104 USA

[8] Georgetown Univ, Sch Med, MedStar Georgetown Transplant Inst, Washington, DC USA

[9] Univ Penn, Sch Med, Div Endocrinol Diabet & Metab, Dept Med, Philadelphia, PA 19104 USA

[10] Univ Penn, Sch Med, Div Endocrinol Diabet & Metab, Dept Orthopaed Surg, Philadelphia, PA 19104 USA

[11] Mayo Clin, Dept Med, Rochester, MN USA

[12] Thomas Jefferson Univ, Dept Anesthesiol, Philadelphia, PA 19107 USA

[13] Univ Amsterdam, Med Ctr, Amsterdam Bone Ctr, Dept Internal Med,Sect Endocrinol,VUmc, Amsterdam, Netherlands

[14] Royal Natl Orthopaed Hosp, Stanmore, Middx, England

[15] Hop Necker Enfants Malad, Inst Imagine, Ctr Reference Malad Osseuses Constitut, Dept Genet, Paris, France

[16] Queensland Univ Technol, Translat Res Inst, Inst Hlth & Biomed Innovat, Woolloongabba, Qld, Australia

[17] Seoul Natl Univ, Childrens Hosp, Div Pediat Orthopaed, Seoul, South Korea

[18] Hosp Italiano Buenos Aires, Dept Pediat, Pediat Rheumatol Sect, Buenos Aires, DF, Argentina

[19] Hosp Israelita Albert Einstein, Inst Ensino & Pesquisa, Sao Paulo, SP, Brazil

[20] Univ Tokyo, Grad Sch Med, Dept Rehabil Med, Tokyo, Japan

[21] Klinikum Garmisch Partenkirchen GmbH, Dept Pediat, Garmisch Partenkirchen, Germany

[22] Univ Cape Town, Red Cross Childrens Hosp, Paediat Rheumatol, Cape Town, South Africa

[23] Shanghai Tongji Univ, Tongji Hosp, Dept Endocrinol, Shanghai, Peoples R China

[24] Thomas Jefferson Univ, Sidney Kimmel Med Coll, Philadelphia, PA 19107 USA

[25] Schulich Sch Med & Dent, Pediat Oral Hlth & Dent, London, ON, Canada

[26] Univ Penn, Perelman Sch Med, Ctr Res FOP & Related Disorders, Dept Med, Philadelphia, PA 19104 USA

[27] Univ Penn, Perelman Sch Med, Ctr Res FOP & Related Disorders, Dept Orthopaed Surg, Philadelphia, PA 19104 USA

来源：

BRITISH JOURNAL OF CLINICAL PHARMACOLOGY | 2019年 / 85卷 / 06期

基金：

日本学术振兴会;

关键词：

Fibrodysplasia ossificans progressiva (FOP); patient safety; rare disease clinical trials; HETEROTOPIC OSSIFICATION; PET/CT;

D O I：

10.1111/bcp.13777

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

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.

引用

页码：1199 / 1207

页数：9

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