A Study of Tadalafil for Duchenne Muscular Dystrophy

Launched by ELI LILLY AND COMPANY · May 24, 2013

Keywords

ClinConnect Summary

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Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • Ambulant males with Duchenne muscular dystrophy (DMD) confirmed by typical clinical presentation (onset of clinical signs or symptoms before 6 years of age supported by an elevated serum creatinine kinase level, and ongoing difficulty with walking) together with either a record of a genetic confirmation of the DMD diagnosis, or a record of muscle biopsy showing near-complete dystrophin deficiency (excluding revertant fibers)
• • 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
• • Able to complete the six minute walk distance (6MWD) test with results within 20% of each other at a minimum of 2 pre-randomization assessments
• • Left ventricular ejection fraction (LVEF) ≥50% as determined by echocardiogram
• • Written informed consent from parents/legal guardian will be obtained prior to any study procedure being performed. In addition, the child may be required to give documented assent, if capable.
• Exclusion Criteria:
• • Symptomatic cardiomyopathy or heart failure
• • Change in prophylactic treatment for heart failure within 3 months prior to start of study treatment
• • Cardiac rhythm disorder
• • History of participation in gene or cell-based therapy , or antisense oligonucleotide or stop codon read-through therapy
• • Unable to take orally administered tablets
• • Use of any pharmacologic treatment, other than corticosteroids, that might have an effect on muscle strength within 3 months prior to the start of study treatment (for example, growth hormone, anabolic steroids including testosterone)
• • 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
• • Surgery that might have an effect on muscle strength or function within 3 months before study entry or planned surgery at any time during the study
• • Evidence of a lower limb injury that may affect performance on the 6MWD
• • Severe behavioral problems, including severe autism or attention deficit disorders, that may interfere with completion of the 6MWD
• • Any contraindication to tadalafil (use of any form of organic nitrate, either regularly and/or intermittently, or known serious hypersensitivity to tadalafil)
• • History of significant renal insufficiency or clinical evidence of cirrhosis
• • Have known allergy to any of the excipients in tadalafil tablets, notably lactose
• • Current Phosphodiesterase Type 5 (PDE5) inhibitor therapy or treatment within the past 6 months