臨床研究等提出・公開システム

Date of registration	Mar. 30, 2026
Last modified on	Mar. 30, 2026
Trial ID	jRCT2031250857

Scientific Title	A phase 1 extension study of NS-035 in patients with Fukuyama-type congenital muscular dystrophy (FCMD) (NS035-P1-OE)
Public Title	A phase 1 extension study of NS-035 in patients with Fukuyama-type congenital muscular dystrophy (FCMD) (NS035-P1-OE)

Contact for Scientific Queries	Name	Higashioka Masaya
	Affiliation	Nippon Shinyaku Co., Ltd.
	Address	14, Nishinosho-Monguchi-cho, Kisshoin, Minami-ku, Kyoto
	Telephone	+81-120-40-8930
	E-mail	zz_mail_clinical-trials@po.nippon-shinyaku.co.jp
Contact for Public Queries	Name	Clinical Development Operations
	Affiliation	Nippon Shinyaku Co., Ltd.
	Address	14, Nishinosho-Monguchi-cho, Kisshoin, Minami-ku, Kyoto
	Telephone	+81-120-40-8930
	E-mail	zz_mail_clinical-trials@po.nippon-shinyaku.co.jp

Recruitment status	Recruiting

Anticipated date of first enrollment		April. 01, 2026
Target sample size		12
Study Type		Interventional
Study Design	allocation	single arm study
	masking	open(masking not used)
	control	uncontrolled control
	assignment	single assignment
	purpose	treatment purpose
Key inclusion & exclusion criteria	Inclusion Criteria	1) Patients with FCMD who completed the TKY-TWMU-2021 trial 2) Patients whose legal guardian capable of providing informed consent has provided written informed consent upon thorough understanding of the study procedure. Efforts should be made so that the subjects themselves give voluntary assent after having been provided with an explanation according to their ability to understand.
	Exclusion Criteria	1) Patients in whom the principal investigator (or sub-investigator) determined that a safety-related adverse event occurred during the TKY-TWMU-2021 trial 2) Patients who underwent treatment aimed at producing fukutin or fukutin-related proteins after completion of the TKY-TWMU-2021 trial 3) Patients who are pregnant, or who cannot agree to use an appropriate contraceptive method from the first dose of the investigational drug until 6 months after the final dose(including cases where menarche or spermarche occurs during the clinical trial period) 4) Patients with a history of epilepsy who have had status epilepticus within the past year since the consent was obtained.
	Age Minimum	7age old over
	Age Maximum	No limit
	Gender	Both
Health Condition(s) or Problem(s) Studied		Fukuyama-type congenital muscular dystrophy (FCMD)
Intervention(s)		NS-035 (40 mg/kg) and D-mannitol (500 mg/kg) are administered intravenously once a week.
Primary Outcome(s)		- Adverse events - Physical and neurological examination - Vital signs (Blood pressure, Pulse rate, Body temperature) - Standard 12-lead electrocardiogram - Echocardiogram, Clinical examination (Hematology tests, blod biochemistry tests, urinalysis) - Immunological tests (Anti-NS-035 antibody) - Endocrinological tests, Cytokines - Ultrasonography (Kidney, Ureter, Bladder) - SpO2 - Blood gas analysis - Urinary cytology
Secondary Outcome(s)		- Gross motor function measure-88(GMFM-88) - Urinary cytology

Primary Sponsor	Nippon Shinyaku Co., Ltd.

Name of Certified Review Board	Tokyo Medical University Hospital Institutional Review Board
Address	6-7-1 Nishishinjuku, Shinjuku-ku, Tokyo
Telephone	+81-3-3342-6111
Approval Status	Not approval

Plan to share IPD	No

Countries of Recruitment（Except Japan）	none