Muscular Dystrophy, DuchennePhase 3NCT01865084

A Study of Tadalafil for Duchenne Muscular Dystrophy

A Phase 3 study testing Tadalafil in people with Muscular Dystrophy, Duchenne, run by Eli Lilly and Company at 62 sites in 16 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

Who is it for?: Ages 7 to 14 years, men only
Conditions the study is for: Muscular Dystrophy, Duchenne
What is being tested?: Tadalafil
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 331 participants
Where: 62 sitesin 16 countries
Study dates: Sep 2013to Mar 2016
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 7 to 14 years
Men only
No healthy volunteers
Child

Who may be able to join

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.

Who cannot join

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)

Show all 14Show fewer

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

Trial locations and contacts

62 sites in 16 countries.

Country

City, postcode or hospital

62 sites in 16 countries

United States23 sites

Children's Hospital
Aurora, Colorado, 80045
Carolinas Healthcare System
Charlotte, North Carolina, 28203
Ann and Robert Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Childrens Hospital Medical Center
Cincinnati, Ohio, 45229
Children's Medical Center Dallas
Dallas, Texas, 75207
Duke University Medical Center
Durham, North Carolina, 27705
University of Florida Health Science Center
Gainesville, Florida, 32610
NW Florida Clinical Research Group
Gulf Breeze, Florida, 32561
Pennsylvania State University College of Medicine
Hershey, Pennsylvania, 17033
University of Iowa
Iowa City, Iowa, 52242
University of Kansas Medical Center
Kansas City, Kansas, 66160
Cedars Sinai Medical Center
Los Angeles, California, 90048
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Children of the King's Daughters
Norfolk, Virginia, 23510
Nemours Children's Hospital
Orlando, Florida, 32827
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Childrens Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Oregon Health and Science University
Portland, Oregon, 97239
University of California, Davis - Health Systems
Sacramento, California, 95817
University of Utah School of Medicine
Salt Lake City, Utah, 84132
Univ of Texas Health Science Center at San Antonio
San Antonio, Texas, 78229
Seattle Children's Hospital Research Foundation
Seattle, Washington, 98105
Washington University Medical Center
St Louis, Missouri, 63110

Other trials in United States

Canada6 sites

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Calgary, Alberta, T3B 6A8
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Halifax, Nova Scotia, B3K 6R8
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London, Ontario, N6C 2V5
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Ottawa, Ontario, K1H 8L1
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Vancouver, British Columbia, V6H 3V4
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Winnipeg, Manitoba, R3A 1R9

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Germany5 sites

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Dresden, 01307
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Essen, 45122
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Freiburg im Breisgau, 79106
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Göttingen, 37075
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Munich, 80337

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Italy4 sites

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Genova, 16147
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Milan, 20122
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Padova, 35128
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Rome, 00165

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Japan4 sites

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Aichi, 467-8602
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Nagano, 390-8621
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Saitama, 349-0196
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Tokyo, 187-8551

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Spain4 sites

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Barcelona, 08025
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Donostia / San Sebastian, 20014
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Madrid, 28046
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Valencia, 46026

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Belgium3 sites

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Ghent, 9000
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Leuven, 3000
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Liège, 4000

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France2 sites

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Angers, 49933
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Nantes, 44093

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Netherlands2 sites

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Leiden, 2300 RC
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Nijmegen, 6500 HB

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Taiwan2 sites

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Kaohsiung City, 807
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Taipei, 100

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Turkey (Türkiye)2 sites

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Adana, 01330
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Ankara, 06100

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Argentina1 site

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Caba, C1204AAD

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Puerto Rico1 site

University of Puerto Rico, Medical Sciences Campus
San Juan, 000935

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Russia1 site

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Moscow, 125412

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South Korea1 site

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Seoul, 110-744

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United Kingdom1 site

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Oxford, Oxfordshire, OX3 9DU

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About this study

Official title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy

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.

Conditions: Muscular Dystrophy, Duchenne
Being tested: Tadalafil, Placebo
Medical subject headings: Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

TadalafilDrug
Also called LY450190, Cialis
Administered orally
PlaceboDrug
Also called Administered orally

Study groups in NCT01865084
Study group	Role	What they receive
Placebo	Experimental	Placebo taken orally once daily. Receives: Drug: Placebo
0.3 mg/kg Tadalafil	Experimental	0.3 milligram per kilogram (mg/kg) tadalafil taken orally once daily. Receives: Drug: Tadalafil
0.6 mg/kg Tadalafil	Experimental	0.6 mg/kg tadalafil taken orally once daily. Receives: Drug: Tadalafil

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: Baseline, Week 48

Secondary outcomes (6)

Change From Baseline in the North Star Ambulatory Assessment (NSAA) Global Score
The NSAA is a functional scale specifically designed for ambulant boys with DMD that can provide additional information on motor functions important in maintaining normal ambulation and other activities important to everyday life. The NSAA is a 17-item evaluation of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0, 1, or 2, with higher scores reflecting better performance on the assessment, for a total maximum score of 34. This score was transformed to a 0 to 100 scale for the key analysis (referred to as linearized), with higher transformed scores reflecting better performance.The LS mean (LSM) change from baseline standard error was derived using mixed model repeated measures methodology (MMRM) with factors for pooled country, treatment, visit, treatment-by-visit interaction and Day 1 value as baseline covariate.
Measured: Baseline, Week 48
Change From Baseline in Timed Function Tests in Seconds
Timed function tests included time it took to rise from floor, walk 10 meters, ascend 4 stairs, and descend 4 stairs.The lower the time in seconds taken, the better the performance. The LS mean change from baseline, standard error, was derived using mixed model repeated measures methodology (MMRM) with factors for pooled country, treatment, visit, treatment-by-visit interaction and Day 1 value as baseline covariate.
Measured: Baseline, Week 48
Time to Persistent 10% Worsening in 6MWD
Time on study until the 6MWD becomes 10% less than the baseline 6MWD and continues at that level or lower until the end of study.
Measured: Baseline through Week 48
Time to Persistent 10% Worsening in Timed Function Tests (TFT)
Time on study until the TFT becomes 10% worse than the baseline TFT and continues at that level or lower until the end of study. The time to persistent 10% worsening is the observed time after baseline until the first observed timepoint where their time used for the TFTs is >110% of the baseline time and all the time values observed afterward are also >110% of baseline. If the participant discontinues prior to experiencing persistent worsening, this outcome for the participant is censored at the date of discontinuation of the double-blind period.
Only participants with complete evaluable data were analyzed. Complete evaluable data was defined as having baseline measurement, complete dates at evaluable visits and a post-baseline measurement at each evaluable visit.
Measured: Baseline through Week 48
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Scores
PODCI includes a Global Functioning Scale and 5 core scales:Upper Extremity and Physical Function,Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort,and Happiness.The Global Functioning Scale is the mean of the mean scores from 4 of the 5 core scales (all except the happiness core scale).The following PODCI scores were prespecified in the protocol for analysis: Global Functioning, Upper Extremity and Physical Function,Transfer/Basic Mobility, and Sports/Physical Functioning. The Global Functioning Scale and each of the core scales were standardized so that a score of "0" represents a poor outcome/worse health, while "100" is the best possible outcome/best health (i.e., complete range of each score is 0 to 100, with higher scores representing better functioning). The LS mean (LSM) change from baseline,standard error was derived using MMRM with factors for pooled country, treatment, visit, treatment-by-visit interaction and baseline PODC scale as covariate.
Measured: Baseline, Week 48
Pharmacokinetics (PK): Apparent Clearance (CL/F) of Tadalafil
The data reported are population estimate and inter-patient variability.
Measured: Weeks 4, 12, 24 and 36: -1 Hour up to 24 Hours Postdose

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Sep 2013Mar 2016

First posted30 May 2013 (estimated)
Study startSep 2013
Primary completionDec 2015
Study completionMar 2016

Why it stopped: The study is being terminated for lack of efficacy

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (3)Papers the sponsor has linked to this study.
Other trials for Muscular Dystrophy, DuchenneStudies of the same condition, open and under way.
More trials run by Eli Lilly and CompanyThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 331 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01865084
Sponsor's protocol ID: 15122
Other IDs: H6D-MC-LVJJ
Status last verified: Sep 2019
First submitted: 24 May 2013
Last updated: 9 Oct 2019
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Individual data sharing: Yes: Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.

Publications

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.
PubMed 19941337
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.
PubMed 40217158
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.
PubMed 28972192

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
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Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
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Duchenne Muscular Dystrophy

All Muscular Dystrophy, Duchenne trials

Study at a glance

From the registry record, last updated by the sponsor on 9 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy
ClinicalTrials.gov ID: NCT01865084
Acronym / sponsor ID: 15122
Conditions: Muscular Dystrophy, Duchenne
Phase: Phase 3
Study type: Interventional
Recruitment status: TerminatedReason stopped: The study is being terminated for lack of efficacy
Enrolment: 331 participants (actual)
Lead sponsor: Eli Lilly and CompanyIndustry (pharma, biotech, devices)
Study start: Sep 2013
Primary completion: Dec 2015 (actual)
Study completion: Mar 2016 (actual)
First posted: 30 May 2013 (estimated)
Results: Posted 1 Mar 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 3

Arm	Type	Receives
Placebo	Experimental	Drug: PlaceboPlacebo taken orally once daily.
0.3 mg/kg Tadalafil	Experimental	Drug: Tadalafil0.3 milligram per kilogram (mg/kg) tadalafil taken orally once daily.
0.6 mg/kg Tadalafil	Experimental	Drug: Tadalafil0.6 mg/kg tadalafil taken orally once daily.

Who may qualify

Ages: 7 to 14 years
Sex: Male only
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (5 listed)

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.

Key exclusion criteria (14 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
TadalafilAlso: LY450190, CialisAdministered orally	Drug	0.3 mg/kg Tadalafil, 0.6 mg/kg Tadalafil
PlaceboAlso: Administered orally	Drug	Placebo

Primary outcome

Change From Baseline in Six Minute Walk Distance (6MWD) in Meters
Time frame: Baseline, Week 48
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.

6 secondary outcomes

Change From Baseline in the North Star Ambulatory Assessment (NSAA) Global Score
Time frame: Baseline, Week 48
Change From Baseline in Timed Function Tests in Seconds
Time frame: Baseline, Week 48
Time to Persistent 10% Worsening in 6MWD
Time frame: Baseline through Week 48
Time to Persistent 10% Worsening in Timed Function Tests (TFT)
Time frame: Baseline through Week 48
Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Scores
Time frame: Baseline, Week 48
Pharmacokinetics (PK): Apparent Clearance (CL/F) of Tadalafil
Time frame: Weeks 4, 12, 24 and 36: -1 Hour up to 24 Hours Postdose

Results and safety

Results posted to the registry on 1 Mar 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Placebo	116	111
0.3 mg/kg Tadalafil	102	98
0.6 mg/kg Tadalafil	113	107

Primary outcome: Change From Baseline in Six Minute Walk Distance (6MWD) in Meters

Time frame: Baseline, Week 48. Least squares mean (Standard Error). Unit: Meters

Measure	Placebo n = 113	0.3 mg/kg Tadalafil n = 101	0.6 mg/kg Tadalafil n = 111
Value	-50.99 (9.316)	-64.71 (9.809)	-59.08 (9.397)

Placebo vs 0.3 mg/kg Tadalafil: p = 0.307, Mixed Models Analysis
Placebo vs 0.6 mg/kg Tadalafil: p = 0.538, Mixed Models Analysis

Population analysed: All randomized participants who received at least one dose of study drug who had a baseline and at least one post-baseline measurement.

6 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Placebo - DB	5 of 116 (4.3%)	83 of 116 (71.6%)	Not reported
0.3 mg/kg Tadalafil -DB	4 of 102 (3.9%)	82 of 102 (80.4%)	Not reported
0.6 mg/kg Tadalafil - DB	6 of 112 (5.4%)	92 of 112 (82.1%)	Not reported
0.3 mg/kg Tadalafil - OLE	6 of 150 (4%)	68 of 150 (45.3%)	Not reported
0.6 mg/kg Tadalafil - OLE	9 of 165 (5.5%)	84 of 165 (50.9%)	Not reported

Limitations and caveats (from the sponsor): 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.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2019.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 19941337McDonald 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.
Derived from this study: PubMed 40217158Cox 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.
Derived from this study: PubMed 28972192Victor 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.
Individual participant dataSharing planned: Yes. Anonymized individual patient level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

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) study director

Eli Lilly and Company

62 sites in 16 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Tadalafil in people with Muscular Dystrophy, Duchenne, run by Eli Lilly and Company at 62 sites in 16 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 9 Oct 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 62 sites in United States, Canada, Germany, Italy, Japan and 11 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 1 Mar 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in Six Minute Walk Distance (6MWD) in Meters (time frame: Baseline, Week 48). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01865084, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2019.