Status:
TERMINATED
A Study of Tadalafil for Duchenne Muscular Dystrophy
Lead Sponsor:
Eli Lilly and Company
Conditions:
Muscular Dystrophy, Duchenne
Eligibility:
MALE
7-14 years
Phase:
PHASE3
Brief Summary
The main purpose of this study is to determine if tadalafil can slow the decline in walking ability of boys who have Duchenne muscular dystrophy (DMD). The study will also assess the safety of tadalaf...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
September 1 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2016
Estimated Enrollment :
331 Patients enrolled
Trial Details
Trial ID
NCT01865084
Start Date
September 1 2013
End Date
March 1 2016
Last Update
October 9 2019
Active Locations (62)
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1
Cedars Sinai Medical Center
Los Angeles, California, United States, 90048
2
University of California, Davis - Health Systems
Sacramento, California, United States, 95817
3
Children's Hospital
Aurora, Colorado, United States, 80045
4
University of Florida Health Science Center
Gainesville, Florida, United States, 32610