nctId: NCT06290713 id: IRB202301491 id: 1R21AR079755-01 type: NIH link: https://reporter.nih.gov/quickSearch/1R21AR079755-01 id: PRO00050023 type: OTHER domain: UFIRST briefTitle: Vasodilator and Exercise Study for DMD (VASO-REx) overallStatus: NOT_YET_RECRUITING date: 2024-03 date: 2026-11 date: 2026-11 date: 2024-03-04 date: 2024-03-21 name: University of Florida class: OTHER name: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) briefSummary: Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal muscle and improve cardiovascular health) and tadalafil, an FDA-approved vasodilator (to optimize blood flow and muscle perfusion which is impaired and often overlooked in DMD). Target: improved muscle function, vascular health, and DMD treatment. conditions: Duchenne Muscular Dystrophy conditions: Duchenne Disease conditions: Muscular Dystrophy conditions: Muscular Dystrophy in Children conditions: Vasodilation conditions: Exercise conditions: DMD studyType: INTERVENTIONAL phases: PHASE2 allocation: RANDOMIZED interventionModel: PARALLEL interventionModelDescription: Participants are assigned to receive interventions based on their completion and results of Aim 1 (responsiveness to study drug). If participants are found to be Tadalafil-responsive after one dose, they will be included in Aim 2 of the study and will be asked to participate in the double-blinded, randomized, placebo-controlled longitudinal medication \& exercise 6-month study program. primaryPurpose: TREATMENT masking: QUADRUPLE whoMasked: PARTICIPANT whoMasked: CARE_PROVIDER whoMasked: INVESTIGATOR whoMasked: OUTCOMES_ASSESSOR count: 50 type: ESTIMATED name: Tadalafil name: Placebo name: Exercise Training measure: Vascular responsiveness after muscle contraction to a single dose of tadalafil. measure: Cycling time to fatigue measure: Quadriceps muscle Fat Fraction measure: Metabolic recovery measure: cardiopulmonary exercise testing (CPET) - Peak aerobic capacity (VO2max) measure: cardiopulmonary exercise testing (CPET) - Minute Ventilation (VE) measure: cardiopulmonary exercise testing (CPET) - Ventilatory Equivalent for Oxygen (VE/VO2) measure: cardiopulmonary exercise testing (CPET) - Gas Exchange Threshold (GET) measure: cardiopulmonary exercise testing (CPET) - Workload at Anaerobic Threshold (W at AT) measure: The 100-meter timed test (100m) measure: the North Star Ambulatory Assessment (NSAA) measure: The 4-stair climb measure: The Physical activity questionnaire (PAQ-C) measure: Pulmonary function testing - vital capacity (FVC) measure: Pulmonary function testing - forced expiratory volume in 1 second (FEV1) measure: Neurology Quality of Life (NeuroQoL) pediatric lower extremity function measure: Patient Reported Outcomes Measurement Information System (PROMIS) pediatric fatigue measure: PROMIS parent proxy for physical activity measure: PROMIS pediatric physical activity measure: Physical Activity Monitoring - Daily Step Rate measure: Physical Activity Monitoring - Time in Low-Level Activity measure: Physical Activity Monitoring - Time in Moderate-Level Activity measure: Physical Activity Monitoring - Time in High-Level Activity sex: MALE minimumAge: 6 Years stdAges: CHILD stdAges: ADULT stdAges: OLDER_ADULT hasResults: False