イシグロ　クミコ   ISHIGURO Kumiko   石黒　久美子    所属   医学部 医学科（東京女子医科大学病院）    職種   助教
論文種別	原著
言語種別	英語
査読の有無	査読なし
表題	Nonrandomized Allocation of Steroid Therapy in Patients With Fukuyama Congenital Muscular Dystrophy: Study Protocol for a Phase II Clinical Trial
掲載誌名	正式名：Neuropsychopharmacology Repts 略　 称：Neuropsychopharmacol Rep ISSNコード：2574173X
掲載区分	国外
巻・号・頁	45(3),pp.e70043
著者・共著者	MURAKAMI Terumi†, SATOU Takatoshi, ISHIZUKA Takami , NAKANURA Harumasa, TACHIMORI Hisateru , HARADA Hiroko, OI Hideki, HATANO Kenji, OBA Mari S, ISHIGURO Kumiko, SHICHIJI Minobu, KIHARA Yuuki, TAKESHIMA Yasuhiro, TANIGUCHI-IKED Mariko, HATTORI Ayako, SHIMIZU-MOTOHASHI Yuko, AWAN Hiroyuki, BO Ryosuke, ISHIGAKI Keiko*, NAGATA Satoru
発行年月	2025/09
概要	Aim: This study aimed to evaluate the efficacy and safety of steroid therapy in patients with Fukuyama congenital muscular dystrophy (FCMD). Methods: This is a noncontrolled, nonblinded, multicenter collaborative phase 2 trial in patients with a definite diagnosis of 3-kb insertion mutation homozygous or compound heterozygous FCMD based on genetic testing. The first part of the study evaluates efficacy; patients with homozygous FCMD were given oral prednisolone at a dose of 1 mg/kg on alternate days (0.5 mg/kg/day) for 24 weeks. The second part of the study evaluates safety; patients with homozygous and heterozygous FCMD were given oral prednisolone at a dose of 1 mg/kg on alternate days (0.5 mg/kg/day) for 48 weeks. Homozygous patients will be evaluated in the first part of the study for up to 24 weeks after prednisolone administration, after which they will continue receiving prednisolone in the second part for an additional 24 weeks. The primary endpoints were the changes in motor function evaluated using the gross motor function measure after treatment with prednisolone in the first part and the safety profiles based on the results of physical examination, vital signs, 12-lead electrocardiography (ECG), echocardiography, ophthalmic testing, SpO2, laboratory tests, immunological tests, and adverse events in the second part. Discussion: Based on previous clinical research, prednisolone shows great potential as a therapeutic drug in patients with FCMD. To achieve this goal, we planned an investigator-initiated trial to confirm the effectiveness and safety of prednisolone. Keywords: Fukuyama congenital muscular dystrophy; drug repositioning; investigator‐initiated clinical trial; prednisolone; steroid therapy.
DOI	10.1002/npr2.70043
PMID	40814256