ClinicalTrials.gov
ClinicalTrials.gov Menu

Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy (REVERSE-DBMD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01168908
Recruitment Status : Terminated (As recommended by the DSMB.)
First Posted : July 23, 2010
Results First Posted : March 21, 2018
Last Update Posted : March 21, 2018
Sponsor:
Collaborator:
Johns Hopkins University
Information provided by (Responsible Party):
Kathryn Wagner, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions: Duchenne Muscular Dystrophy
Becker Muscular Dystrophy
Intervention: Drug: Sildenafil

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Revatio (Sildenafil)

This arm will receive Revatio (sildenafil) for 12 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.

Sildenafil: 20mg tablet three times daily

Placebo

This arm will receive placebo (sugar pill) for 6 months and Revatio (sildenafil) for 6 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.

Sildenafil: 20mg tablet three times daily


Participant Flow for 2 periods

Period 1:   Double Blind, Placebo Controlled
    Revatio (Sildenafil)   Placebo
STARTED   10   10 
COMPLETED   7   8 
NOT COMPLETED   3   2 

Period 2:   Open Label
    Revatio (Sildenafil)   Placebo
STARTED   7   8 
COMPLETED   6   7 
NOT COMPLETED   1   1 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Revatio (Sildenafil)

This arm will receive Revatio (sildenafil) for 12 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.

Sildenafil: 20mg tablet three times daily

Placebo

This arm will receive placebo (sugar pill) for 6 months and Revatio (sildenafil) for 6 months. During the first 6 months, the subject and investigator will be blinded to treatment. The second 6 months, will be open label treatment with Revatio.

Sildenafil: 20mg tablet three times daily

Total Total of all reporting groups

Baseline Measures
   Revatio (Sildenafil)   Placebo   Total 
Overall Participants Analyzed 
[Units: Participants]
 10   10   20 
Age 
[Units: Years]
Mean (Standard Deviation)
 25.5  (8.2)   22.6  (4.4)   24.05  (6.6) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      0   0.0%      0   0.0%      0   0.0% 
Male      10 100.0%      10 100.0%      20 100.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
     
American Indian or Alaska Native      0   0.0%      0   0.0%      0   0.0% 
Asian      1  10.0%      2  20.0%      3  15.0% 
Native Hawaiian or Other Pacific Islander      0   0.0%      0   0.0%      0   0.0% 
Black or African American      2  20.0%      0   0.0%      2  10.0% 
White      7  70.0%      8  80.0%      15  75.0% 
More than one race      0   0.0%      0   0.0%      0   0.0% 
Unknown or Not Reported      0   0.0%      0   0.0%      0   0.0% 


  Outcome Measures

1.  Primary:   Change in Cardiac Left Ventricular End-systolic Volume (LVESV) by Cardiac Magnetic Resonance (CMR) Imaging.   [ Time Frame: 6 months compared to baseline ]

2.  Secondary:   Change in Cardiac Systolic and Diastolic Function by CMR   [ Time Frame: 6 months and 12 months ]

3.  Secondary:   Change in Cardiac Mass   [ Time Frame: 6 months and 12 months ]

4.  Secondary:   Change in Forced Vital Capacity (FVC) by Pulmonary Function Testing   [ Time Frame: 6 months and 12 months ]

5.  Secondary:   Change in Skeletal Muscle Strength   [ Time Frame: 6 months and 12 months ]

6.  Secondary:   Ejection Fraction   [ Time Frame: 6 months ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Kathryn Wagner, MD, PhD
Organization: Kennedy Krieger Institute
phone: 443-923-9525
e-mail: wagnerk@kennedykrieger.org


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Kathryn Wagner, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
ClinicalTrials.gov Identifier: NCT01168908     History of Changes
Other Study ID Numbers: NA-00036602
First Submitted: July 22, 2010
First Posted: July 23, 2010
Results First Submitted: August 24, 2017
Results First Posted: March 21, 2018
Last Update Posted: March 21, 2018