A Study of Tadalafil for Duchenne Muscular Dystrophy

This study has been terminated.
(The study is being terminated for lack of efficacy)
Information provided by (Responsible Party):
Eli Lilly and Company
ClinicalTrials.gov Identifier:
First received: May 24, 2013
Last updated: April 15, 2016
Last verified: April 2016
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.

Condition Intervention Phase
Muscular Dystrophy, Duchenne
Drug: Tadalafil
Drug: Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy

Resource links provided by NLM:

Further study details as provided by Eli Lilly and Company:

Primary Outcome Measures:
  • Change from Baseline in Six Minute Walk Distance (6MWD) in Meters [ Time Frame: Baseline, Week 48 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change from Baseline in the North Star Ambulatory Assessment (NSAA) Global Score [ Time Frame: Baseline, Week 48 ] [ Designated as safety issue: No ]
  • Change from Baseline in Timed Function Tests in Seconds [ Time Frame: Baseline, Week 48 ] [ Designated as safety issue: No ]
  • Time to Persistent 10% Worsening in 6MWD [ Time Frame: Baseline through Week 48 ] [ Designated as safety issue: No ]
  • Time to Persistent 10% Worsening in Timed Function Tests [ Time Frame: Baseline through Week 48 ] [ Designated as safety issue: No ]
  • Change from Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Scores [ Time Frame: Baseline, Week 48 ] [ Designated as safety issue: No ]
  • Pharmacokinetics (PK): Apparent Clearance (CL/F) of Tadalafil [ Time Frame: 1 Hour up to 24 Hours Postdose; Weeks 4, 12, 24 and 36 ] [ Designated as safety issue: No ]

Enrollment: 227
Study Start Date: September 2013
Study Completion Date: April 2016
Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Placebo
Placebo taken orally once daily.
Drug: Placebo
Other Name: Administered orally
Experimental: Tadalafil 0.3 mg/kg
Tadalafil 0.3 milligram per kilogram (mg/kg) taken orally once daily.
Drug: Tadalafil
Administered orally
Other Names:
  • LY450190
  • Cialis
Experimental: Tadalafil 0.6 mg/kg
Tadalafil 0.6 mg/kg taken orally once daily.
Drug: Tadalafil
Administered orally
Other Names:
  • LY450190
  • Cialis


Ages Eligible for Study:   7 Years to 14 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01865084

  Show 62 Study Locations
Sponsors and Collaborators
Eli Lilly and Company
Study Director: Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST) Eli Lilly and Company
  More Information

Responsible Party: Eli Lilly and Company
ClinicalTrials.gov Identifier: NCT01865084     History of Changes
Other Study ID Numbers: 15122  H6D-MC-LVJJ 
Study First Received: May 24, 2013
Last Updated: April 15, 2016
Health Authority: United States: Food and Drug Administration
Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica
Belgium: Federal Agency for Medicinal Products and Health Products
Canada: Health Canada
France: Agence Nationale de Sécurité du Médicament et des produits de santé
Germany: Federal Institute for Drugs and Medical Devices
Italy: The Italian Medicines Agency
Korea: Ministry of Food and Drug Safety
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Russia: Ministry of Health of the Russian Federation
Spain: Agencia Española de Medicamentos y Productos Sanitarios
Taiwan : Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Muscular Diseases
Muscular Disorders, Atrophic
Musculoskeletal Diseases
Nervous System Diseases
Neuromuscular Diseases
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Phosphodiesterase 5 Inhibitors
Phosphodiesterase Inhibitors
Urological Agents
Vasodilator Agents

ClinicalTrials.gov processed this record on May 26, 2016