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A Study of Tadalafil for Duchenne Muscular Dystrophy

Primary Purpose

Muscular Dystrophy, Duchenne

Status

Terminated

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Tadalafil

Placebo

About this trial

This is an interventional treatment trial for Muscular Dystrophy, Duchenne

Eligibility Criteria

7 Years - 14 Years (Child)MaleDoes not accept healthy volunteers

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

Sites / Locations

Cedars Sinai Medical Center
University of California, Davis - Health Systems
Children's Hospital
University of Florida Health Science Center
NW Florida Clinical Research Group
Nemours Children's Hospital
Ann and Robert Lurie Children's Hospital of Chicago
University of Iowa
University of Kansas Medical Center
Washington University Medical Center
Carolinas Healthcare System
Duke University Medical Center
Childrens Hospital Medical Center
Oregon Health and Science University
Pennsylvania State University College of Medicine
Childrens Hospital of Philadelphia
Childrens Hospital of Pittsburgh
Vanderbilt University Medical Center
Children's Medical Center Dallas
Univ of Texas Health Science Center at San Antonio
University of Utah School of Medicine
Children of the King's Daughters
Seattle Children's Hospital Research Foundation
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
University of Puerto Rico, Medical Sciences Campus
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Arm Label

Placebo

0.3 mg/kg Tadalafil

0.6 mg/kg Tadalafil

Arm Description

Placebo taken orally once daily.

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

0.6 mg/kg tadalafil taken orally once daily.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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.

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.

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.

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.

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.

Pharmacokinetics (PK): Apparent Clearance (CL/F) of Tadalafil

The data reported are population estimate and inter-patient variability.

Full Information

NCT ID

NCT01865084

First Posted

May 24, 2013

Last Updated

September 25, 2019

Sponsor

Eli Lilly and Company

1. Study Identification

Unique Protocol Identification Number

NCT01865084

Brief Title

A Study of Tadalafil for Duchenne Muscular Dystrophy

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

September 2019

Overall Recruitment Status

Terminated

Why Stopped

The study is being terminated for lack of efficacy

Study Start Date

September 2013 (undefined)

Primary Completion Date

December 2015 (Actual)

Study Completion Date

March 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Eli Lilly and Company

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Muscular Dystrophy, Duchenne

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

331 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Placebo

Arm Type

Experimental

Arm Description

Placebo taken orally once daily.

Arm Title

0.3 mg/kg Tadalafil

Arm Type

Experimental

Arm Description

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

Arm Title

0.6 mg/kg Tadalafil

Arm Type

Experimental

Arm Description

0.6 mg/kg tadalafil taken orally once daily.

Intervention Type

Drug

Intervention Name(s)

Tadalafil

Other Intervention Name(s)

LY450190, Cialis

Intervention Description

Administered orally

Intervention Type

Drug

Intervention Name(s)

Placebo

Other Intervention Name(s)

Administered orally

Primary Outcome Measure Information:

Title

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

Description

Time Frame

Baseline, Week 48

Secondary Outcome Measure Information:

Title

Change From Baseline in the North Star Ambulatory Assessment (NSAA) Global Score

Description

Time Frame

Baseline, Week 48

Title

Change From Baseline in Timed Function Tests in Seconds

Description

Time Frame

Baseline, Week 48

Title

Time to Persistent 10% Worsening in 6MWD

Description

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.

Time Frame

Baseline through Week 48

Title

Time to Persistent 10% Worsening in Timed Function Tests (TFT)

Description

Time Frame

Baseline through Week 48

Title

Change From Baseline in Pediatric Outcomes Data Collection Instrument (PODCI) Scores

Description

Time Frame

Baseline, Week 48

Title

Pharmacokinetics (PK): Apparent Clearance (CL/F) of Tadalafil

Description

The data reported are population estimate and inter-patient variability.

Time Frame

Weeks 4, 12, 24 and 36: -1 Hour up to 24 Hours Postdose

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

7 Years

Maximum Age & Unit of Time

14 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

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)

Organizational Affiliation

Eli Lilly and Company

Official's Role

Study Director

Facility Information:

Facility Name

Cedars Sinai Medical Center

City

Los Angeles

State/Province

California

ZIP/Postal Code

90048

Country

United States

Facility Name

University of California, Davis - Health Systems

City

Sacramento

State/Province

California

ZIP/Postal Code

95817

Country

United States

Facility Name

Children's Hospital

City

Aurora

State/Province

Colorado

ZIP/Postal Code

80045

Country

United States

Facility Name

University of Florida Health Science Center

City

Gainesville

State/Province

Florida

ZIP/Postal Code

32610

Country

United States

Facility Name

NW Florida Clinical Research Group

City

Gulf Breeze

State/Province

Florida

ZIP/Postal Code

32561

Country

United States

Facility Name

Nemours Children's Hospital

City

Orlando

State/Province

Florida

ZIP/Postal Code

32827

Country

United States

Facility Name

Ann and Robert Lurie Children's Hospital of Chicago

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60611

Country

United States

Facility Name

University of Iowa

City

Iowa City

State/Province

Iowa

ZIP/Postal Code

52242

Country

United States

Facility Name

University of Kansas Medical Center

City

Kansas City

State/Province

Kansas

ZIP/Postal Code

66160

Country

United States

Facility Name

Washington University Medical Center

City

Saint Louis

State/Province

Missouri

ZIP/Postal Code

63110

Country

United States

Facility Name

Carolinas Healthcare System

City

Charlotte

State/Province

North Carolina

ZIP/Postal Code

28203

Country

United States

Facility Name

Duke University Medical Center

City

Durham

State/Province

North Carolina

ZIP/Postal Code

27705

Country

United States

Facility Name

Childrens Hospital Medical Center

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

45229

Country

United States

Facility Name

Oregon Health and Science University

City

Portland

State/Province

Oregon

ZIP/Postal Code

97239

Country

United States

Facility Name

Pennsylvania State University College of Medicine

City

Hershey

State/Province

Pennsylvania

ZIP/Postal Code

17033

Country

United States

Facility Name

Childrens Hospital of Philadelphia

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104

Country

United States

Facility Name

Childrens Hospital of Pittsburgh

City

Pittsburgh

State/Province

Pennsylvania

ZIP/Postal Code

15224

Country

United States

Facility Name

Vanderbilt University Medical Center

City

Nashville

State/Province

Tennessee

ZIP/Postal Code

37232

Country

United States

Facility Name

Children's Medical Center Dallas

City

Dallas

State/Province

Texas

ZIP/Postal Code

75207

Country

United States

Facility Name

Univ of Texas Health Science Center at San Antonio

City

San Antonio

State/Province

Texas

ZIP/Postal Code

78229

Country

United States

Facility Name

University of Utah School of Medicine

City

Salt Lake City

State/Province

Utah

ZIP/Postal Code

84132

Country

United States

Facility Name

Children of the King's Daughters

City

Norfolk

State/Province

Virginia

ZIP/Postal Code

23510

Country

United States

Facility Name

Seattle Children's Hospital Research Foundation

City

Seattle

State/Province

Washington

ZIP/Postal Code

98105

Country

United States

Facility Name

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.

City

Caba

ZIP/Postal Code

C1204AAD

Country

Argentina

Facility Name

City

Gent

ZIP/Postal Code

9000

Country

Belgium

Facility Name

City

Leuven

ZIP/Postal Code

3000

Country

Belgium

Facility Name

City

Liège

ZIP/Postal Code

4000

Country

Belgium

Facility Name

City

Calgary

State/Province

Alberta

ZIP/Postal Code

T3B 6A8

Country

Canada

Facility Name

City

Vancouver

State/Province

British Columbia

ZIP/Postal Code

V6H 3V4

Country

Canada

Facility Name

City

Winnipeg

State/Province

Manitoba

ZIP/Postal Code

R3A 1R9

Country

Canada

Facility Name

City

Halifax

State/Province

Nova Scotia

ZIP/Postal Code

B3K 6R8

Country

Canada

Facility Name

City

London

State/Province

Ontario

ZIP/Postal Code

N6C 2V5

Country

Canada

Facility Name

City

Ottawa

State/Province

Ontario

ZIP/Postal Code

K1H 8L1

Country

Canada

Facility Name

City

Angers

ZIP/Postal Code

49933

Country

France

Facility Name

City

Nantes

ZIP/Postal Code

44093

Country

France

Facility Name

City

Dresden

ZIP/Postal Code

01307

Country

Germany

Facility Name

City

Essen

ZIP/Postal Code

45122

Country

Germany

Facility Name

City

Freiburg

ZIP/Postal Code

79106

Country

Germany

Facility Name

City

Göttingen

ZIP/Postal Code

37075

Country

Germany

Facility Name

City

Munich

ZIP/Postal Code

80337

Country

Germany

Facility Name

City

Genova

ZIP/Postal Code

16147

Country

Italy

Facility Name

City

Milano

ZIP/Postal Code

20122

Country

Italy

Facility Name

City

Padova

ZIP/Postal Code

35128

Country

Italy

Facility Name

City

Rome

ZIP/Postal Code

00165

Country

Italy

Facility Name

City

Aichi

ZIP/Postal Code

467-8602

Country

Japan

Facility Name

City

Nagano

ZIP/Postal Code

390-8621

Country

Japan

Facility Name

City

Saitama

ZIP/Postal Code

349-0196

Country

Japan

Facility Name

City

Tokyo

ZIP/Postal Code

187-8551

Country

Japan

Facility Name

City

Seoul

ZIP/Postal Code

110-744

Country

Korea, Republic of

Facility Name

City

Leiden

ZIP/Postal Code

2300 RC

Country

Netherlands

Facility Name

City

Nijmegen

ZIP/Postal Code

6500 HB

Country

Netherlands

Facility Name

University of Puerto Rico, Medical Sciences Campus

City

San Juan

ZIP/Postal Code

000935

Country

Puerto Rico

Facility Name

City

Moscow

ZIP/Postal Code

125412

Country

Russian Federation

Facility Name

City

Barcelona

ZIP/Postal Code

08025

Country

Spain

Facility Name

City

Madrid

ZIP/Postal Code

28046

Country

Spain

Facility Name

City

San Sebastian

ZIP/Postal Code

20014

Country

Spain

Facility Name

City

Valencia

ZIP/Postal Code

46026

Country

Spain

Facility Name

City

Kaohsiung

ZIP/Postal Code

807

Country

Taiwan

Facility Name

City

Taipei

ZIP/Postal Code

100

Country

Taiwan

Facility Name

City

Adana

ZIP/Postal Code

01330

Country

Turkey

Facility Name

City

Ankara

ZIP/Postal Code

06100

Country

Turkey

Facility Name

City

Oxford

State/Province

Oxfordshire

ZIP/Postal Code

OX3 9DU

Country

United Kingdom

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

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.

IPD Sharing 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.

IPD Sharing Access Criteria

A research proposal must be approved by an independent review panel and researchers must sign a data sharing agreement.

IPD Sharing URL

https://vivli.org/

Citations:

PubMed Identifier

19941337

Citation

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.

Results Reference

background

PubMed Identifier

28972192

Citation

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.

Results Reference

derived

Learn more about this trial

A Study of Tadalafil for Duchenne Muscular Dystrophy

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