None listed
Conditions
Interventions
Drug : Two doses of tadalafil (0.3 mg/kg and 0.6 mg/kg) will be compared to placebo. up to 48 weeks, once daily, oral.
Sponsors
Eli Lilly Korea
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Males with proven DMD 2. Ages 7-14 years inclusive 3. Ambulant, defined as 6MWD between 200 and 400 meters inclusive at screening and baseline 4. Baseline 6MWD measurement within 20% of the screening 6MWD 5. Left ventricular ejection fraction (LVEF) =50% as determined by echocardiogram obtained at screening, or based on record of an echocardiogram performed within 30 days of Visit 1 if a copy of the record can be obtained as source documentation for the screening LVEF 6. Receiving systemic corticosteroids for a minimum of 6 months immediately prior to screening, with no significant change in total daily dosage or dosing regimen (except those adjusting for weight changes) for a minimum of 3 months immediately prior to screening and a reasonable expectation that total daily dosage and dosing regimen will not change significantly (except for adjustments for weight) for the duration of the study. 7. Written informed consent from parents/legal guardian and written assent from patients will be obtained prior to any study procedure being performed
Exclusion criteria
Exclusion criteria: 1. Symptomatic cardiomyopathy or heart failure (New York Heart Association Class III or IV) 2. Change in prophylactic treatment for heart failure within 3 months prior to start of study treatment 3. Cardiac rhythm disorder defined as sinus rhythm with ectopic contractions or conductance disturbances, or any rhythm other than sinus, observed on screening ECG 4. Use of continuous mechanical ventilator assistance. [Evening use of bi-level positive airway pressure (BPAP) or continuous positive airway pressure (CPAP) therapy is allowed] 5. Any contraindication to tadalafil (use of any form of organic nitrate, either regularly and/or intermittently, or known serious hypersensitivity to tadalafil) 6. History of significant renal insufficiency, defined as receiving renal dialysis or having a screening serum cystatin C level = 2.35 mg/L 7. Clinical evidence of cirrhosis 8. Diagnosed with a retinal disorder (for example, hereditary retinal disorders, retinopathy of prematurity) 9. New or changed treatment with herbal or dietary supplements being taken with an expectation of an effect on muscle strength or function during 1 month prior to first dose of study drug.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| lessens the decline in ambulatory ability as measured by the 6MWD compared to placebo in boys with Duchenne muscular dystrophy (DMD) | — |
Secondary
| Measure | Time frame |
|---|---|
| lessens the decline in North Star Ambulatory Assessment (NSAA) global score | — |
Countries
Korea, Republic of
Contacts
Public ContactJong Hee Chae
Seoul National University Hospital
Outcome results
None listed