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 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.

Trial Health

Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

331

participants targeted

Target at P50-P75 for phase_3

Timeline

Completed

Started Sep 2013

Geographic Reach

16 countries

62 active sites

Status

terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

2.5 years study duration

First Submitted

Initial submission to the registry

May 24, 2013

Completed

6 days until next milestone

First Posted

Study publicly available on registry

May 30, 2013

Completed

3 months until next milestone

Study Start

First participant enrolled

September 1, 2013

Completed

2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2015

Completed

3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2016

Completed

1 year until next milestone

Results Posted

Study results publicly available

March 1, 2017

Completed

Last Updated

October 9, 2019

Status Verified

September 1, 2019

Enrollment Period

2.2 years

First QC Date

May 24, 2013

Results QC Date

September 30, 2016

Last Update Submit

September 25, 2019

Conditions

Muscular Dystrophy, Duchenne

Outcome Measures

Primary Outcomes (1)

Change From Baseline in Six Minute Walk Distance (6MWD) in Meters
6MWD measured the distance in meters a participant was able to walk in 6 minutes. The study used 6MWD procedure modified specifically for use in boys with Duchenne muscular dystrophy (DMD), including standardized verbal encouragement at specific intervals to maintain attention to the test, and use of a "safety chaser" to walk behind the participant during testing (McDonald et al., 2010a). The LS mean (LSM) change from baseline, standard error was derived using mixed model repeated measures (MMRM) methodology with factors for pooled country, treatment, visit, treatment-by-visit interaction and baseline 6MWD as a covariate.
Baseline, Week 48

Secondary Outcomes (6)

Change From Baseline in the North Star Ambulatory Assessment (NSAA) Global Score
Baseline, Week 48
Change From Baseline in Timed Function Tests in Seconds
Baseline, Week 48
Time to Persistent 10% Worsening in 6MWD
Baseline through Week 48
Time to Persistent 10% Worsening in Timed Function Tests (TFT)
Baseline through Week 48
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Scores
Baseline, Week 48
+1 more secondary outcomes

Study Arms (3)

Placebo

EXPERIMENTAL

Placebo taken orally once daily.

Drug: Placebo

0.3 mg/kg Tadalafil

EXPERIMENTAL

0.3 milligram per kilogram (mg/kg) tadalafil taken orally once daily.

Drug: Tadalafil

0.6 mg/kg Tadalafil

EXPERIMENTAL

0.6 mg/kg tadalafil taken orally once daily.

Drug: Tadalafil

Interventions

TadalafilDRUG

Administered orally

Also known as: LY450190, Cialis

0.3 mg/kg Tadalafil0.6 mg/kg Tadalafil

PlaceboDRUG

Also known as: Administered orally

Placebo

Eligibility Criteria

Age7 Years - 14 Years

Sexmale

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

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.

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Eli Lilly and Companylead

Study Sites (62)

Cedars Sinai Medical Center

Los Angeles, California, 90048, United States

Location

University of California, Davis - Health Systems

Sacramento, California, 95817, United States

Location

Children's Hospital

Aurora, Colorado, 80045, United States

Location

University of Florida Health Science Center

Gainesville, Florida, 32610, United States

Location

NW Florida Clinical Research Group

Gulf Breeze, Florida, 32561, United States

Location

Nemours Children's Hospital

Orlando, Florida, 32827, United States

Location

Ann and Robert Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Washington University Medical Center

St Louis, Missouri, 63110, United States

Location

Carolinas Healthcare System

Charlotte, North Carolina, 28203, United States

Location

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Childrens Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Pennsylvania State University College of Medicine

Hershey, Pennsylvania, 17033, United States

Location

Childrens Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Childrens Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Children's Medical Center Dallas

Dallas, Texas, 75207, United States

Location

Univ of Texas Health Science Center at San Antonio

San Antonio, Texas, 78229, United States

Location

University of Utah School of Medicine

Salt Lake City, Utah, 84132, United States

Location

Children of the King's Daughters

Norfolk, Virginia, 23510, United States

Location

Seattle Children's Hospital Research Foundation

Seattle, Washington, 98105, United States

Location

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.

Caba, C1204AAD, Argentina

Location

Ghent, 9000, Belgium

Location

Leuven, 3000, Belgium

Location

Liège, 4000, Belgium

Location

Calgary, Alberta, T3B 6A8, Canada

Location

Vancouver, British Columbia, V6H 3V4, Canada

Location

Winnipeg, Manitoba, R3A 1R9, Canada

Location

Halifax, Nova Scotia, B3K 6R8, Canada

Location

London, Ontario, N6C 2V5, Canada

Location

Ottawa, Ontario, K1H 8L1, Canada

Location

Angers, 49933, France

Location

Nantes, 44093, France

Location

Dresden, 01307, Germany

Location

Essen, 45122, Germany

Location

Freiburg im Breisgau, 79106, Germany

Location

Göttingen, 37075, Germany

Location

Munich, 80337, Germany

Location

Genova, 16147, Italy

Location

Milan, 20122, Italy

Location

Padua, 35128, Italy

Location

Rome, 00165, Italy

Location

Aichi, 467-8602, Japan

Location

Nagano, 390-8621, Japan

Location

Saitama, 349-0196, Japan

Location

Tokyo, 187-8551, Japan

Location

Leiden, 2300 RC, Netherlands

Location

Nijmegen, 6500 HB, Netherlands

Location

University of Puerto Rico, Medical Sciences Campus

San Juan, 000935, Puerto Rico

Location

Moscow, 125412, Russia

Location

Seoul, 110-744, South Korea

Location

Barcelona, 08025, Spain

Location

Donostia / San Sebastian, 20014, Spain

Location

Madrid, 28046, Spain

Location

Valencia, 46026, Spain

Location

Kaohsiung City, 807, Taiwan

Location

Taipei, 100, Taiwan

Location

Adana, 01330, Turkey (Türkiye)

Location

Ankara, 06100, Turkey (Türkiye)

Location

Oxford, Oxfordshire, OX3 9DU, United Kingdom

Location

Related Publications (3)

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.
PMID: 19941337BACKGROUND
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.
PMID: 40217158DERIVED
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.
PMID: 28972192DERIVED

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

Tadalafil

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

CarbolinesPyridinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsIndole AlkaloidsIndolesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingHeterocyclic Compounds, 3-Ring

Limitations and Caveats

The Sponsor concluded that the efficacy results do not provide sufficient justification for continuance of the open-label extension (OLE) period of the study, where all participants were receiving daily treatment with tadalafil.

Results Point of Contact

Title: Chief Medical Officer
Organization: Eli Lilly and Company

Study Officials

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
STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee: No
Restriction Type: GT60
Restrictive Agreement: Yes

Study Design

Study Type: interventional
Phase: phase 3
Allocation: RANDOMIZED
Masking: QUADRUPLE
Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose: TREATMENT
Intervention Model: PARALLEL
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

May 24, 2013

First Posted

May 30, 2013

Study Start

September 1, 2013

Primary Completion

December 1, 2015

Study Completion

March 1, 2016

Last Updated

October 9, 2019

Results First Posted

March 1, 2017

Record last verified: 2019-09

Data Sharing

IPD Sharing: Will share
Shared Documents: STUDY PROTOCOL, SAP, CSR
Time Frame: Data are available 6 months after the primary publication and approval of the indication studied in the US and EU, whichever is later. Data will be indefinitely available for requesting.
Access Criteria: A research proposal must be approved by an independent review panel and researchers must sign a data sharing agreement.

Locations

AR(1)DE(5)KR(1)CA(6)FR(2)IT(4)TW(2)BE(3)TU(2)GB(1)RU(1)ES(4)US(23)NL(2)JP(4)PR(1)

Brief Summary

Trial Health

Trial Health Score

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Results Posted

Conditions

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (6)

Study Arms (3)

Placebo

0.3 mg/kg Tadalafil

0.6 mg/kg Tadalafil

Interventions

Eligibility Criteria

You may qualify if:

You may not qualify if:

Sponsors & Collaborators

Study Sites (62)

Related Publications (3)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Intervention Hierarchy (Ancestors)

Limitations and Caveats

Results Point of Contact

Study Officials

Publication Agreements

Study Design

Study Record Dates

Data Sharing

Locations