Resumen

El propósito principal de este estudio es determinar si tadalafil puede enlentecer el deterioro de la capacidad para caminar en niños con distrofia muscular de Duchenne (DMD). El estudio también evaluará la seguridad de tadalafil y cualquier evento adverso que pudiera estar asociado con su uso en niños con DMD. Los participantes recibirán el tratamiento del estudio (tadalafil o placebo) durante las primeras 48 semanas del estudio, y luego podrán continuar en una extensión abierta (OLE) que consta de dos períodos durante los cuales todos los participantes recibirán tadalafil. En el período 1 de OLE, todos los participantes recibirán tadalafil durante 48 semanas. Los participantes que completen el período 1 de OLE continuarán en el período 2 de OLE y recibirán tadalafil durante al menos otras 48 semanas.

Elegibilidad

Edad mínima: 7 YearsEdad máxima: 14 YearsSexo: MALE

Criterios de inclusión

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.

Criterios de exclusión

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

Ensayo clínico de fase 3, aleatorizado, doble ciego, controlado con placebo de tadalafil para la distrofia muscular de Duchenne

Resumen

Elegibilidad

Criterios de inclusión

Criterios de exclusión

Intervenciones

Tadalafil

Placebo

Ubicaciones

For additional information regarding investigative sites for this trial, contact 1-877-CTLILLY (1-877-285-4559, 1-317-615-4559) Mon - Fri from 9 AM to 5 PM Eastern Time (UTC/GMT - 5 hours, EST), or speak with your personal physician.

Patrocinadores