A phase 1 extension study of NS-035 in patients with Fukuyama-type congenital muscular dystrophy (FCMD)

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

Status

Recruiting

Phases

Phase 1

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2031250857

Enrollment

Registered

2026-03-30

Start date

2026-04-01

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Fukuyama-type congenital muscular dystrophy (FCMD)

Interventions

NS-035 (40 mg/kg) and D-mannitol (500 mg/kg) are administered intravenously once a week.

Eligibility

Sex/Gender

All

Inclusion 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

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.

Design outcomes

Primary

Measure	Time frame
- 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	—

Measure

Time frame

- 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

Measure	Time frame
- Gross motor function measure-88(GMFM-88) - Urinary cytology	—

Contacts

Public ContactOperations Clinical

Nippon Shinyaku Co., Ltd.

zz_mail_clinical-trials@po.nippon-shinyaku.co.jp+81-120-40-8930

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026