Duchenne Muscular Dystrophy Clinical Trial
Official title:
Functional Muscle Ischemia and PDE5A Inhibition in Duchenne Muscular Dystrophy
This study, supported by Parent Project Muscular Dystrophy, will determine if tadalafil or sildenafil can improve muscle blood flow during exercise in boys with Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is a rare, progressive and fatal muscle disease affecting
boys and accounts for 80% of muscular dystrophy cases. Tadalafil and sildenafil are
medications approved by the FDA for the treatment of erectile dysfunction and pulmonary
hypertension. This class of medication improves muscle blood flow in a mouse model of
muscular dystrophy, but their benefit to boys with DMD is unknown. The purpose of this study
is to 1) determine if tadalafil or sildenafil can improve muscle blood flow during exercise
in boys with DMD; and 2) to inform the design of a subsequent, randomized, multi-center trial
with clinical endpoints.
The investigators will enroll boys with DMD between the ages of 7 and 15 years who are
ambulatory and without clinical heart failure. Participants will undergo 6 visits over the
course of 5 weeks. The initial visit will include a medical history, physical exam,
echocardiogram, and blood draw to determine eligibility for the study. Boys will be given a
Holter monitor (a heart monitor) to wear for 48 hours to observe any irregular heartbeats or
abnormalities.
Eligible boys will be randomized to one of the two study drugs: tadalafil or sildenafil. The
boys will take a low dose (0.25mg/kg) of the study drug for the first 2 days and an
intermediate dose (0.5mg/kg) for the subsequent 5 days. Then, boys will take a higher dose
(1.0mg/kg) of the study drug for 1 week. Tadalafil will be taken once daily and sildenafil
will be taken four times daily.
Study visits will occur 2 times at baseline, 2 times during the medication, and 1 time after
washout of the medication. For these visits, boys will undergo an arm blood flow and hand
grip exercise protocol. In this procedure, blood flow and oxygen delivery to the forearm
muscles will be measured (noninvasively) before and during application of lower body negative
pressure at rest and during handgrip exercise. Lower body negative pressure stimulates the
blood flow changes that normally occur when a person sits up after lying down. During these
visits, boys will complete a quality of life questionnaire, echocardiogram, and 6-minute walk
tests. At home, boys will wear an accelerometer to measure physical activity and a Holter
monitor to check for irregular heartbeats.
For boys who wish to continue with the study, there will be an option to cross-over and
complete study visits with the drug they did not originally receive.
;
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT05575648 -
Dual Task in Duchenne Muscular Dystrophy
|
N/A | |
Terminated |
NCT03907072 -
Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy
|
Phase 2/Phase 3 | |
Not yet recruiting |
NCT06450639 -
An Open-label Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy
|
Phase 2 | |
Completed |
NCT04335942 -
Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor
|
N/A | |
Active, not recruiting |
NCT04906460 -
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT02500381 -
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
|
Phase 3 | |
Enrolling by invitation |
NCT05967351 -
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
|
Phase 3 | |
Recruiting |
NCT03067831 -
Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy
|
Phase 1/Phase 2 | |
Recruiting |
NCT01834040 -
Study Safety and Efficacy of BMMNC for the Patient With Duchenne Muscular Dystrophy
|
Phase 1/Phase 2 | |
Completed |
NCT02246478 -
A Study of TAS-205 for Duchenne Muscular Dystrophy
|
Phase 1 | |
Active, not recruiting |
NCT01772043 -
Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping
|
N/A | |
Terminated |
NCT01168908 -
Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
|
Phase 2 | |
Completed |
NCT00758225 -
Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
|
Phase 2 | |
Completed |
NCT03680365 -
Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
|
||
Recruiting |
NCT03513367 -
The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM.
|
||
Recruiting |
NCT05712447 -
Duchenne Muscular Dystrophy Video Assessment Registry
|
||
Recruiting |
NCT01484678 -
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
|
||
Completed |
NCT03319030 -
Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)
|
||
Terminated |
NCT01753804 -
A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
|
N/A | |
Completed |
NCT02530905 -
Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
|
Phase 1 |