A Study of Tadalafil for Duchenne Muscular Dystrophy
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 tadalafil and any side effects that might be associated with it in boys who have DMD. Participants will receive study treatment (tadalafil or placebo) for the first 48 weeks of the study, and can then continue into an open label extension (OLE) that consists of two periods during which all participants will receive tadalafil. In OLE period 1, all participants will receive tadalafil for 48 weeks. Participants completing OLE period 1 will continue into OLE period 2 and will receive tadalafil for at least another 48 weeks.
Timeline
- Start
- 2013-09
- Primary completion
- 2015-12
- Completion
- 2016-03
Outcome
Missed primary endpoint
Stopped: “The study is being terminated for lack of efficacy”
paper Tadalafil had no effect on the primary outcome: PMID 28972192 ↗
paper Tadalafil did not lessen the decline in ambulatory ability in boys with DMD. PMID 28972192 ↗
registry analysis (unspecified test); Placebo vs 0.3 mg/kg Tadalafil; p = 0.307; Mixed Models Analysis NCT01865084 ↗
registry analysis (unspecified test); Placebo vs 0.6 mg/kg Tadalafil; p = 0.538; Mixed Models Analysis NCT01865084 ↗
Publications
- Background McDonald CM, Henricson EK, Han JJ, Abresch RT, Nicorici A, Elfring GL, Atkinson L, Reha A, Hirawat S, Miller LL. The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy. Muscle Nerve. 2010 Apr;41(4):500-10. doi: 10.1002/mus.21544.
- Cox D, Byrne B, Hammers DW, Landry J, Sweeney HL. Effect of Tadalafil on cardiac function and left ventricular dimensions in Duchenne muscular dystrophy: safety and cardiac MRI substudy results from a randomized, placebo-controlled trial. BMC Cardiovasc Disord. 2025 Apr 11;25(1):276. doi: 10.1186/s12872-025-04727-3.
- Victor RG, Sweeney HL, Finkel R, McDonald CM, Byrne B, Eagle M, Goemans N, Vandenborne K, Dubrovsky AL, Topaloglu H, Miceli MC, Furlong P, Landry J, Elashoff R, Cox D; Tadalafil DMD Study Group. A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy. Neurology. 2017 Oct 24;89(17):1811-1820. doi: 10.1212/WNL.0000000000004570. Epub 2017 Sep 29.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Tadalafil | Small molecule | 0.3 mg/kg | Oral |
| Subject | Tadalafil | Small molecule | 0.6 mg/kg | Oral |