Tokyo, July 9 -- UMIN Clinical Trials Registry (UMIN-CTR) received information related to the study (UMIN000062178) titled 'Impact of Tofersen treatment on Neurofilament light chain level in Japanese patients with SOD1-ALS' on July 8.

Study Type: Observational

Primary Sponsor: Institute - Tohoku University School of Medicine

Condition: Condition - Patients with SOD1-ALS Classification by malignancy - Others Genomic information - YES

Objective: Narrative objectives1 - To investigate the effects of tofersen treatment on neurofilament light chain levels and clinical outcomes in Japanese patients with SOD1-ALS. Basic objectives2 - Others

Eligibility: Age-lower limit - 15 years-old <= Age-upper limit - Not applicable Gender - Male and Female Key inclusion criteria - Patients who meet all of the following criteria will be eligible for participation in this study: (1)Patients aged 15 years or older at the time of obtaining informed consent. (2)Patients diagnosed with superoxide dismutase 1 (SOD1)-associated amyotrophic lateral sclerosis (ALS) based on genetic testing. (3)Patients who are receiving treatment with tofersen under routine clinical practice, including those who have discontinued tofersen at the time of informed consent. (4)Patients capable of providing written informed consent. -Patients who are able to understand the purpose and potential risks of the study and from whom written informed consent can be obtained in accordance with national and local personal data protection laws. -For patients aged 15 years or older and under 18 years at the time of consent, written informed consent must be obtained from both the patient and an appropriate legally authorized representative. -If a patient has sufficient decision-making capacity but has difficulty writing due to progression of the underlying disease or other reasons, written informed consent may be obtained by proxy writing, provided that the patient's intention to participate in the study is confirmed and the consent process is conducted in the presence of an impartial witness. (5)Patients who are able to provide cerebrospinal fluid (CSF) as their own biological specimens for this observational study, including blood samples when feasible. Key exclusion criteria - Patients who meet any of the following criteria will be excluded from the study: (1)Patients with medical or psychiatric conditions that may interfere with their ability to comply with study procedures or assessments. (2)Patients who have previously received ALS treatments with expected long-lasting or sustained effects, such as viral gene therapies. (3)Patients with comorbid conditions known to be associated with elevated neurofilament light chain (NfL)levels, including multiple sclerosis, dementia, Parkinson's disease or other neurodegenerative diseases, traumatic brain injury, or stroke. (4)Patients who are currently participating in, or have previously participated in, other clinical trials of tofersen, including the VALOR trial and its extension study, or the ATLAS trial. Expanded access programs and compassionate use programs are not considered clinical trials. (5)Patients who are currently participating in, or are planning to participate in, another clinical trial for amyotrophic lateral sclerosis (ALS). (6)Patients who are deemed inappropriate for participation in this observational study by the principal investigator or sub-investigators. Target Size - 40

Recruitment Status: Recruitment status - Open public recruiting Date of protocol fixation - 2026 Year 03 Month 10 Day Date of IRB - 2026 Year 03 Month 18 Day Anticipated trial start date - 2026 Year 07 Month 08 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=R000069802

Disclaimer: Curated by HT Syndication.