Ignite Creation Date:
2024-05-06 @ 8:13 PM
Last Modification Date:
2024-10-26 @ 3:22 PM
Study NCT ID:
NCT06290713
Status:
RECRUITING
Last Update Posted:
2024-06-25
First Post:
2024-02-09
Brief Title:
Vasodilator and Exercise Study for DMD VASO-REx
Sponsor:
University of Florida
Organization:
University of Florida
Study Overview
Official Title:
Vasodilators and Exercise as Adjuvant Therapy for Duchenne Muscular Dystrophy VASO-REx Study
Status:
RECRUITING
Status Verified Date:
2024-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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
Detailed Description:
Duchenne muscular dystrophy DMD is characterized by rapid muscle deterioration mitochondrial and vascular impairments resulting in premature loss of ambulation and mortality Disease-modifying therapeutics are emerging and although they are expected to improve muscle function and daily activity in boys with DMD most are not designed to correct the vascular impairment This impairment is due to the lack of restoration of neuronal nitric oxide synthase signaling which is crucial for vasodilation during and after exercise The investigators believe limitations in study design were responsible for the lack of efficacy Therefore this study combines tadalafil with aerobic exercise to necessitate increased blood flow and activate the drug
This Exploratory Clinical Trial will assess two potential adjuvant therapies for ambulatory boys with DMD 6 years and older 1 aerobic exercise training and 2 tadalafil an FDA-approved vasodilator drug Preclinical and clinical data including recent findings from the principal investigators lab support the use of these strategies and their potential to benefit muscle perfusion fatigue and quality of life
The study will first test for drug responsiveness increase in muscle oxygenation after a single dose Drug-responsive boys with DMD will be randomized to a 6-month intervention of daily tadalafil or placebo combined with structured cycle exercise training Participants will receive exercise-related equipment for use at home and be monitored by the research team via video The study will quantify the interventions impact on vascular impairment muscle pathophysiology inflammation fat accumulation mitochondrial dysfunction exertional fatigue and cycling performance
Our findings are expected to yield
1 Criteria to identify DMD patients most likely to benefit from tadalafil as adjuvant therapy
2 Evidence of a powerful synergy between drug impact and exercise training in DMD
This Exploratory Clinical Trial will assess two potential adjuvant therapies for ambulatory boys with DMD 6 years and older 1 aerobic exercise training and 2 tadalafil an FDA-approved vasodilator drug Preclinical and clinical data including recent findings from the principal investigators lab support the use of these strategies and their potential to benefit muscle perfusion fatigue and quality of life
The study will first test for drug responsiveness increase in muscle oxygenation after a single dose Drug-responsive boys with DMD will be randomized to a 6-month intervention of daily tadalafil or placebo combined with structured cycle exercise training Participants will receive exercise-related equipment for use at home and be monitored by the research team via video The study will quantify the interventions impact on vascular impairment muscle pathophysiology inflammation fat accumulation mitochondrial dysfunction exertional fatigue and cycling performance
Our findings are expected to yield
1 Criteria to identify DMD patients most likely to benefit from tadalafil as adjuvant therapy
2 Evidence of a powerful synergy between drug impact and exercise training in DMD
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 1R21AR079755-01 | NIH | None | None |
| PRO00050023 | OTHER | UFIRST | httpsreporternihgovquickSearch1R21AR079755-01 |