Trial · NCT01865084
A Study of Tadalafil for Duchenne Muscular Dystrophy
NCT01865084, Phase 3, Terminated, Eli Lilly and Company; results first posted 2017-03-01
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Tadalafil for Duchenne Muscular Dystrophy. Trials, record NCT01865084. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01865084
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Tadalafil for Duchenne Muscular Dystrophy
- NCT number
- NCT01865084
- Status
- Terminated
- Phases
- Phase 3
- Enrollment
- 331 participants, actual
- Lead sponsor
- Eli Lilly and Company
- Conditions
- Muscular Dystrophy, Duchenne
- Interventions
- Tadalafil, Placebo
- First posted
- 2013-05-30
- Last updated
- 2019-10-09
- Results first posted
- 2017-03-01
- 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.
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in Six Minute Walk Distance (6MWD) in Meters | 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. |
| Secondary | Change From Baseline in the North Star Ambulatory Assessment (NSAA) Global Score | Baseline, Week 48 | 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. |
| Secondary | Change From Baseline in Timed Function Tests in Seconds | Baseline, Week 48 | 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. |
| Secondary | Time to Persistent 10% Worsening in 6MWD | Baseline through Week 48 | 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. |
| Secondary | Time to Persistent 10% Worsening in Timed Function Tests (TFT) | Baseline through Week 48 | 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. |
| Secondary | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Scores | Baseline, Week 48 | 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. |
| Secondary | Pharmacokinetics (PK): Apparent Clearance (CL/F) of Tadalafil | Weeks 4, 12, 24 and 36: -1 Hour up to 24 Hours Postdose | The data reported are population estimate and inter-patient variability. |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Six Minute Walk Distance (6MWD) in Meters | Baseline, Week 48 | All randomized participants who received at least one dose of study drug who had a baseline and at least one post-baseline measurement. | Meters | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in the North Star Ambulatory Assessment (NSAA) Global Score | Baseline, Week 48 | All randomized participants who received at least one dose of study drug who had a baseline and at least one post-baseline measurement. | Units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Timed Function Tests in Seconds | Baseline, Week 48 | All randomized participants who received at least one dose of study drug who had a baseline and at least one post-baseline measurement. | Seconds | LEAST_SQUARES_MEAN |
| SECONDARY | Time to Persistent 10% Worsening in 6MWD | Baseline through Week 48 | All randomized participants who received at least one dose of study drug who had complete evaluable data. Complete evaluable data was defined as having baseline measurement, complete dates at evaluable visits and a post-baseline measurement at each evaluable visit. Censored participants: placebo=71, 0.3 mg/kg=63, 0.6 mg/kg=61. | Days | MEDIAN |
| SECONDARY | Time to Persistent 10% Worsening in Timed Function Tests (TFT) | Baseline through Week 48 | All randomized participants who received at least 1 dose of study drug who had complete evaluable data.Censored participants:Rise from Floor;placebo(pl)=40,0.3 mg/kg=39,0.6 mg/kg=43;Stair Climb;pl=55,0.3 mg/kg=45,0.6 mg/kg=52;10 Meter Walk/Run pl=61,0.3 mg/kg=65,0.6 mg/kg=58,Stair Descend;pl=63,0.3 mg/kg=60,0.6 mg/kg=59. | Days | MEDIAN |
| SECONDARY | Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Scores | Baseline, Week 48 | All randomized participants who received at least one dose of study drug who had a baseline and at least one post-baseline measurement. The reason the number of participants analyzed is significantly less than the total number of randomized participants is because PODCI was administered only in English. | Units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Pharmacokinetics (PK): Apparent Clearance (CL/F) of Tadalafil | Weeks 4, 12, 24 and 36: -1 Hour up to 24 Hours Postdose | All randomized participants who received at least one dose of study drug and had evaluable PK data. | Liter per hour (L/hr) | GEOMETRIC_MEAN |
Source ClinicalTrials.gov
Results by group46
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Six Minute Walk Distance (6MWD) in Meters | Placebo | -50.99 | Standard Error 9.316 | Meters | |
| Change From Baseline in Six Minute Walk Distance (6MWD) in Meters | 0.3 mg/kg Tadalafil | -64.71 | Standard Error 9.809 | Meters | |
| Change From Baseline in Six Minute Walk Distance (6MWD) in Meters | 0.6 mg/kg Tadalafil | -59.08 | Standard Error 9.397 | Meters | |
| Change From Baseline in the North Star Ambulatory Assessment (NSAA) Global Score | Placebo | -8.80 | Standard Error 1.104 | Units on a scale | |
| Change From Baseline in the North Star Ambulatory Assessment (NSAA) Global Score | 0.3 mg/kg Tadalafil | -9.31 | Standard Error 1.181 | Units on a scale | |
| Change From Baseline in the North Star Ambulatory Assessment (NSAA) Global Score | 0.6 mg/kg Tadalafil | -8.96 | Standard Error 1.115 | Units on a scale | |
| Change From Baseline in Timed Function Tests in Seconds | Placebo | Rise from the Floor(n=92,75,89) | 4.16 | Standard Error 1.120 | Seconds |
| Change From Baseline in Timed Function Tests in Seconds | 0.3 mg/kg Tadalafil | Rise from the Floor(n=92,75,89) | 3.60 | Standard Error 1.223 | Seconds |
| Change From Baseline in Timed Function Tests in Seconds | 0.6 mg/kg Tadalafil | Rise from the Floor(n=92,75,89) | 4.81 | Standard Error 1.156 | Seconds |
| Change From Baseline in Timed Function Tests in Seconds | Placebo | 10 Meter Walk/Run(n=105,90,100) | 1.11 | Standard Error 0.204 | Seconds |
1 to 10 of 46Source ClinicalTrials.gov
See all 46 results by groupParticipant flow21
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Double Blind Period (DB) | STARTED | Placebo | 116 | |
| Double Blind Period (DB) | STARTED | 0.3 mg/kg Tadalafil | 102 | |
| Double Blind Period (DB) | STARTED | 0.6 mg/kg Tadalafil | 113 | |
| Double Blind Period (DB) | Received at Least One Dose of Study Drug | Placebo | 116 | |
| Double Blind Period (DB) | Received at Least One Dose of Study Drug | 0.3 mg/kg Tadalafil | 102 | |
| Double Blind Period (DB) | Received at Least One Dose of Study Drug | 0.6 mg/kg Tadalafil | 112 | |
| Double Blind Period (DB) | COMPLETED | Placebo | 111 | |
| Double Blind Period (DB) | COMPLETED | 0.3 mg/kg Tadalafil | 98 | |
| Double Blind Period (DB) | COMPLETED | 0.6 mg/kg Tadalafil | 107 | |
| Double Blind Period (DB) | NOT COMPLETED | Placebo | 5 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 participant flowAdverse events245
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal pain | Gastrointestinal disorders | Placebo - DB | 6 | 116 | 7 |
| other | Abdominal pain | Gastrointestinal disorders | 0.3 mg/kg Tadalafil -DB | 4 | 102 | 4 |
| other | Abdominal pain | Gastrointestinal disorders | 0.6 mg/kg Tadalafil - DB | 9 | 112 | 9 |
| other | Abdominal pain | Gastrointestinal disorders | 0.3 mg/kg Tadalafil - OLE | 2 | 150 | 2 |
| other | Abdominal pain | Gastrointestinal disorders | 0.6 mg/kg Tadalafil - OLE | 3 | 165 | 3 |
| other | Abdominal pain upper | Gastrointestinal disorders | Placebo - DB | 7 | 116 | 7 |
| other | Abdominal pain upper | Gastrointestinal disorders | 0.3 mg/kg Tadalafil -DB | 5 | 102 | 6 |
| other | Abdominal pain upper | Gastrointestinal disorders | 0.6 mg/kg Tadalafil - DB | 8 | 112 | 8 |
| other | Abdominal pain upper | Gastrointestinal disorders | 0.3 mg/kg Tadalafil - OLE | 4 | 150 | 5 |
| other | Abdominal pain upper | Gastrointestinal disorders | 0.6 mg/kg Tadalafil - OLE | 2 | 165 | 2 |
1 to 10 of 245Source ClinicalTrials.gov
See all 245 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo - DB | ||
| Total, all-cause mortality | 0.3 mg/kg Tadalafil -DB | ||
| Total, all-cause mortality | 0.6 mg/kg Tadalafil - DB | ||
| Total, all-cause mortality | 0.3 mg/kg Tadalafil - OLE | ||
| Total, all-cause mortality | 0.6 mg/kg Tadalafil - OLE | ||
| Total, other adverse events | Placebo - DB | 83 | 116 |
| Total, other adverse events | 0.3 mg/kg Tadalafil -DB | 82 | 102 |
| Total, other adverse events | 0.6 mg/kg Tadalafil - DB | 92 | 112 |
| Total, other adverse events | 0.3 mg/kg Tadalafil - OLE | 68 | 150 |
| Total, other adverse events | 0.6 mg/kg Tadalafil - OLE | 84 | 165 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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