Tadalafil for Duchenne Muscular Dystrophy

Recruiting in Palo Alto (17 mi)

Overseen byTanja Taivassalo

Age: < 18

Sex: Male

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2 & 3

Waitlist Available

Sponsor: University of Florida

No Placebo Group

Prior Safety Data

Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?This Trial will test if tadalafil can improve blood flow in the leg muscles of boys with DMD and see if it can help slow down the disease progression. Tadalafil is a medication that is approved for the treatment of certain conditions in men.

Eligibility Criteria

Boys aged 7 to 13 with Duchenne Muscular Dystrophy (DMD) confirmed by clinical history, physical exam, elevated creatine kinase levels, and lack of dystrophin protein. They must be able to walk and have no other conditions affecting muscle function or metabolism. Those with contraindications for MRI scans or taking certain medications like nitrates cannot participate.

Inclusion Criteria

I have been diagnosed with DMD based on my early symptoms, physical exams, high creatine kinase levels, and specific tests showing lack of dystrophin.

I have been diagnosed with DMD based on my symptoms, tests, and genetic confirmation.

I can walk on my own without help.

+6 more

Exclusion Criteria

I am older than 13 years.

Contraindication to an MR examination (e.g. aneurysm clip, severe claustrophobia, magnetic implants)

I have medical conditions that are not well-controlled.

+2 more

Participant Groups

The trial is testing the effects of a single dose of Tadalafil, a drug that widens blood vessels, on leg muscle blood flow in boys with DMD using MRI or Doppler ultrasound after exercise. The goal is to see if this drug can improve blood flow and potentially slow disease progression.

2Treatment groups

Experimental Treatment

Group I: Tadalafil plus Doppler ultrasound (Schedule B)Experimental Treatment1 Intervention

The intervention is the same as in Schedule A; this arm will use Doppler ultrasound as the technique to monitor drug impact on skeletal muscle.

Group II: Tadalafil plus BOLD-MRI (Schedule A)Experimental Treatment1 Intervention

The intervention is the same as in Schedule B; this arm will use BOLD-MRI as the technique to monitor drug impact on skeletal muscle.