Tokyo, June 13 -- UMIN Clinical Trials Registry (UMIN-CTR) received information related to the study (UMIN000061891) titled 'A multicenter observational study for the higher dose regimen of Nusinersen' on June 12.

Study Type: Observational

Primary Sponsor: Institute - Kagoshima University Graduate School of Medical and Dental Sciences

Condition: Condition - Participants with later-onset 5q spinal muscular atrophy (SMA) Classification by malignancy - Others Genomic information - YES

Objective: Narrative objectives1 - To evaluate the therapeutic effects of higher dose nusinersen on patient-reported health status and physical function in individuals with later-onset SMA Basic objectives2 - Others

Eligibility: Age-lower limit - 18 years-old <= Age-upper limit - Not applicable Gender - Male and Female Key inclusion criteria - Patients must meet all of the following criteria: (1) Patients who are scheduled to initiate or have already initiated high-dose nusinersen under routine clinical practice. (2) Patients who are able to provide written informed consent by themselves. Patients must be able to understand the purpose and risks of the study, and written informed consent must be obtained in accordance with national and local data protection laws. If the patient has sufficient decision-making capacity but has difficulty writing due to progression of the underlying disease, written informed consent may be obtained by proxy writing, provided that the patient's willingness to participate is confirmed and a witness is present. (3) Patients with a genetically confirmed diagnosis of late-onset 5q spinal muscular atrophy (SMA) (Type II, Type III, or Type IV). (4) Patients who are 18 years of age or older at the time of obtaining informed consent. (5) Patients who are able to attend follow-up visits at least once per year during the study period. Key exclusion criteria - Candidates will be excluded from study entry if any of the following criteria apply at screening: (1) Medical or psychiatric conditions that may interfere with the ability to comply with study procedures or assessments. (2) Current enrollment in ASCEND or any other investigational nusinersen trial. (3) Current or planned enrollment in an investigational trial for SMN-directed therapy or SMA gene therapy. (4) Patients who are deemed inappropriate as observational study participants by the principal investigator or a sub-investigator. Target Size - 40

Recruitment Status: Recruitment status - Open public recruiting Date of protocol fixation - 2026 Year 02 Month 25 Day Date of IRB - 2026 Year 03 Month 19 Day Anticipated trial start date - 2026 Year 06 Month 12 Day Last follow-up date - 2027 Year 12 Month 31 Day

To know more, visit https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000069804

Disclaimer: Curated by HT Syndication.