Tokyo, Aug. 18 -- UMIN Clinical Trials Registry (UMIN-CTR) received information related to the study (UMIN000062551) titled 'Side effects and their management associated with DMD gene therapy: A study based on real-world clinical data in Japan' on Aug. 18.

Study Type: Observational

Primary Sponsor: Institute - National Center of Neurology and Psychiatry

Condition: Condition - Duchenne Muscular Dystrophy (DMD) Classification by malignancy - Others Genomic information - YES

Objective: Narrative objectives1 - This study aims to clarify the real-world profile of side effects following the administration of a novel gene therapy (delandistrogene moxeparvovec) for Duchenne Muscular Dystrophy (DMD). By retrospectively analyzing anonymized real-world clinical data collected through the online consultation platform "Medii E-Consult" as part of the safety management project by the Japanese Society of Child Neurology, we will systematically evaluate the incidence, severity, and clinical course of side effects. This study aims to generate objective evidence, such as establishing side effect management guidelines, to contribute to the safety and health protection of future patients receiving this treatment. Basic objectives2 - Others

Eligibility: Age-lower limit - 3 years-old

Gender - Male and Female Key inclusion criteria - Patients diagnosed with DMD. Patients who received gene therapy with delandistrogene moxeparvovec. Cases for which expert consultation was conducted via Medii E-Consult after the treatment. Patients aged 3 years to less than 8 years (within the indicated age range). Key exclusion criteria - Cases with incomplete data judged unsuitable for analysis. Cases without data after the initiation of gene therapy. Cases where written informed consent for participation in this study could not be obtained. Target Size - 200

Recruitment Status: Recruitment status - Enrolling by invitation Date of protocol fixation - 2026 Year 07 Month 01 Day Date of IRB - 2026 Year 07 Month 03 Day Anticipated trial start date - 2026 Year 07 Month 04 Day Last follow-up date - 2030 Year 03 Month 31 Day

To know more, visit https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000071504

Disclaimer: Curated by HT Syndication.