Tokyo, Nov. 24 -- UMIN Clinical Trials Registry (UMIN-CTR) received information related to the study (UMIN000059862) titled 'The long-term natural history study of Duchenne muscular dystrophy' on Nov. 23.
Study Type:
Observational
Primary Sponsor:
Institute - National Center of Neurology and Psychiatry (NCNP)
Condition:
Condition - Duchenne muscular dystrophy
Classification by malignancy - Others
Genomic information - NO
Objective:
Narrative objectives1 - To investigate the long-term natural history data of motor function in ambulatory patients with Duchenne muscular dystrophy (DMD) receiving standard care in Japan until they become non-ambulatory.
Basic objectives2 - Others
Eligibility:
Age-lower limit - 4
years-old
<=
Age-upper limit - Not applicable
Gender - Male
Key inclusion criteria - 1) Patients with a dystrophin abnormality proven by dystrophin gene testing or muscle pathology and a definitive diagnosis of DMD
2) Sex: male
3) Aged 4 or older (at the time of informed consent)
4) Able to rise from the floor and walk independently at baseline
5) Patients receiving oral glucocorticosteroids for at least 6 months before baseline without change in dosage or dosing regimen within 3 months and having stable symptoms
6) If participating in another clinical study, at least 6 months has passed since starting this study at baseline
7) Patients who participated in the previous study (UMIN000030562) and are able to walk at baseline in this study
Key exclusion criteria - 1) Patients with clinically apparent symptoms of cardiac failure and/or respiratory failure as determined by the investigators
2) Patients continuously using a ventilator (use of non-invasive positive-pressure ventilation [NPPV] during sleep is allowed)
3) Patients with behavior abnormalities including intellectual disability, autistic tendency, and attention deficit hyperactivity disorder that, in the opinion of the investigators, could prevent to conduct the assessment of motor function
4) Other patients as determined by the investigators to be ineligible to participate in this study
5) Patients participating in another clinical trial at baseline (This restriction does not apply to patients who have continued participation from the previous study (UMIN000030562).)
Target Size - 80
Recruitment Status:
Recruitment status - Enrolling by invitation
Date of protocol fixation - 2024 Year 09 Month 20 Day
Date of IRB - 2024 Year 10 Month 04 Day
Anticipated trial start date - 2025 Year 05 Month 01 Day
Last follow-up date - 2029 Year 08 Month 31 Day
To know more, visit https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000068454
Disclaimer: Curated by HT Syndication.
