Duchenne muscular dystrophy
Conditions
Interventions
None listed
Sponsors
National Center of Neurology and Psychiatry (NCNP)
Eligibility
Sex/Gender
Male
Inclusion criteria
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
Exclusion criteria
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).)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Motor function test | — |
Secondary
| Measure | Time frame |
|---|---|
| Subject demographics | — |
Countries
Japan
Contacts
Public ContactMikiko Shigemori
CareCure NMD Secretariat Office
Outcome results
None listed