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Tadalafil for Duchenne Muscular Dystrophy

Phase 2 & 3
Recruiting
Led By Tanja Taivassalo
Research Sponsored by University of Florida
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diagnosis of DMD confirmed by 1) clinical history with features before the age of five, 2) physical examination, 3) elevated serum creatine kinase level and 4) absence of dystrophin expression, as determined by immunostaining or Western blot (<2%) and/or DNA confirmation of dystrophin mutation.
Minimum entry age of 7.0 years
Timeline
Screening 3 weeks
Treatment Varies
Follow Up doppler ultrasound will be done 3 hours after dosing/no-dosing on two separate study visits
Awards & highlights

Study Summary

This trial will study how well a common vasodilator drug works in boys with Duchenne muscular dystrophy. The findings could help inform a longer-term intervention study to see if the drug can help slow disease progression in DMD.

Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
Tadalafil may cause headaches, indigestion, back pain, muscle aches, flushing, stuffy or runny nose. These side effects usually go away after a few hours but can last longer in some cases.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with DMD based on my early symptoms, physical exams, high creatine kinase levels, and specific tests showing lack of dystrophin.
Select...
I am at least 7 years old.
Select...
I can walk on my own without help.
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I have been diagnosed with DMD based on my symptoms, tests, and genetic confirmation.
Select...
I am at least 7 years old.
Select...
I can walk on my own without help.
Select...
I have been diagnosed with DMD based on my symptoms, tests, and genetic confirmation.
Select...
I am at least 7 years old.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~mri will be done 3 hours after dosing/no-dosing on two separate study visits
This trial's timeline: 3 weeks for screening, Varies for treatment, and mri will be done 3 hours after dosing/no-dosing on two separate study visits for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change in post-contractile BOLD response after tadalafil dosing
Change in post-exercise hyperemia after tadalafil dosing
Secondary outcome measures
Change in submaximal exercise capacity after tadalafil dosing

Side effects data

From 2017 Phase 4 trial • 635 Patients • NCT02224846
9%
Viral upper respiratory tract infection
3%
Periodontitis
3%
Prostatitis
2%
Alanine aminotransferase increased
2%
Upper respiratory tract infection
2%
Abdominal pain upper
2%
Chronic gastritis
2%
Blood uric acid increased
2%
Insomnia
1%
Pharyngitis
1%
Gout
1%
Sleep disorder
1%
Rhinitis allergic
1%
Rhinitis
1%
Eczema
1%
Influenza
1%
Back pain
1%
Diabetes mellitus
1%
Enteritis
1%
Gastritis
1%
Urinary tract infection
1%
Dizziness
1%
Epistaxis
1%
Renal cyst
1%
Gastric polyps
1%
Gastrooesophageal reflux disease
1%
Nephrolithiasis
1%
Hypertension
1%
Pyrexia
1%
Large intestine polyp
1%
Abdominal distension
1%
Abdominal pain
1%
Dental caries
1%
Diarrhoea
1%
Gamma-glutamyltransferase increased
1%
Headache
1%
Cough
100%
80%
60%
40%
20%
0%
Study treatment Arm
2.5 mg/5 mg Tadalafil
5 mg Tadalafil

Trial Design

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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Tadalafil
2014
Completed Phase 4
~3280

Find a Location

Who is running the clinical trial?

University of FloridaLead Sponsor
1,338 Previous Clinical Trials
715,291 Total Patients Enrolled
Tanja TaivassaloPrincipal InvestigatorUniversity of Florida

Media Library

Tadalafil Clinical Trial Eligibility Overview. Trial Name: NCT05195775 — Phase 2 & 3
Duchenne Muscular Dystrophy Research Study Groups: Tadalafil plus BOLD-MRI (Schedule A), Tadalafil plus Doppler ultrasound (Schedule B)
Duchenne Muscular Dystrophy Clinical Trial 2023: Tadalafil Highlights & Side Effects. Trial Name: NCT05195775 — Phase 2 & 3
Tadalafil 2023 Treatment Timeline for Medical Study. Trial Name: NCT05195775 — Phase 2 & 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many individuals are included in this clinical trial?

"That is correct, the online clinicaltrial.gov registry reflects that this study is still recruiting patients. The posting date was December 14th, 2021 and the most recent update was on November 8th, 2022. They are looking for a total of 25 participants at 1 location."

Answered by AI

Are researchers actively searching for test subjects?

"The trial, which was created on December 14th 2021 according to clinicaltrials.gov, is still recruiting patients as of November 8th 2022."

Answered by AI

Does this experimental treatment only test on people over the age of 50?

"Children that meet the age criteria of 7 to 13 years old are eligible for this study. In total, there are 61 trials underway involving patients under 18 and 50 for those over 65."

Answered by AI

Who meets the qualifications to participate in this clinical trial?

"In this particular study, researchers are only recruiting 25 patients that have muscular dystrophy (duchenne) and fall between the ages of 7-13. Most importantly, these individuals must meet the following clinical requirements: A diagnosis of DMD confirmed by 1) clinical history with features before the age of five, 2) physical examination, 3) elevated serum creatine kinase level and 4) absence of dystrophin expression, as determined by immunostaining or Western blot (<2%) and/or DNA confirmation of dystrophin mutation., a minimum entry age into the trial of 7.0 years old, and be ambul"

Answered by AI

Who else is applying?

How old are they?
< 18
What site did they apply to?
University of Florida
What portion of applicants met pre-screening criteria?
Met criteria
What state do they live in?
Puerto Rico

How responsive is this trial?

Typically responds via
Email
~4 spots leftby Oct 2024