Duchenne Muscular DystrophyPhase 2/3NCT05195775
Tadalafil as Adjuvant Therapy for DMD
A Phase 2/3 study testing Tadalafil in people with Duchenne Muscular Dystrophy, run by University of Florida at 1 site in United States.
Trial summary
This project will assess the vascular responsiveness in leg muscles of boys with Duchenne muscular dystrophy (DMD) to one single dose of tadalafil, a common vasodilator drug, using non-invasive techniques (MRI or Doppler ultrasound). Read more about this study
- Who is it for?
- Ages 6 to 13 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Tadalafil
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 14 Dec 2021to 8 Sep 2025
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 13 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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
- Ambulatory
Who cannot join
- Older than 13.0 years of age
- Contraindication to an MR examination (e.g. aneurysm clip, severe claustrophobia, magnetic implants)
- Presence of unstable medical problems
- Presence of a secondary condition that impacts muscle function or muscle metabolism (e.g. myasthenia gravis, endocrine disorder, mitochondrial disease)
- Contraindications to Tadalafil (use of nitrates, alpha-adrenergic blockers, other phosphodiesterase 5A inhibitors)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Tadalafil as an Adjuvant to Therapy for Duchenne Muscular Dystrophy
This project will assess the vascular responsiveness in leg muscles of boys with Duchenne muscular dystrophy (DMD) to one single dose of tadalafil, a common vasodilator drug, using non-invasive techniques (MRI or Doppler ultrasound). These findings will provide proof of concept for a subsequent intervention study to demonstrate efficacy of longer-term tadalafil to counter sympathetic vasoconstriction and slow disease progression in DMD.
Read the detailed description
This project will target up to 25 ambulatory boys with DMD aged 7-30 years. Because the vascular impact of tadalafil is immediate, we will confirm that the drug target is valid target in lower extremities by assessing the change in post-exercise microvascular perfusion using Blood Oxygen Level-Dependent (BOLD) MRI or change in post-exercise hyperemia using Doppler ultrasonography. Patients will be randomized to undergo the above-mentioned assessments with or without the study drug on two separate visits.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Tadalafil
- Keywords
- therapy, treatment strategy, vasodilator, exercise
- Medical subject headings
- Muscular Dystrophy, Duchenne, Motor Activity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TadalafilDrug
Also called Cialis
Patient will be randomized to one dose of 0.6 mg/kg tadalafil on one of two study visits. On both visits, assessments (MRI, exercise testing, clinical function) will be performed 3 hours after time of dosing.
| Study group | Role | What they receive |
|---|---|---|
| Tadalafil plus BOLD-MRI | Experimental | This arm will use BOLD-MRI as the technique to monitor drug impact on skeletal muscle. Receives: Drug: Tadalafil |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change in Post-contractile BOLD Signal Relative to Resting Condition
The post contractile blood oxygen level-dependent (BOLD) response is a non-invasive index of microvascular function in muscle using MRI. BOLD images are acquired from the tibialis anterior before and after dorsiflexion exercise. A transient increase in BOLD signal is expected after performing a brief muscle contraction (reflecting increased small vessel oxygen saturation and blood volume). In healthy boys, MRI-BOLD signal was on average 4.4% above resting levels. The greater the value, the more intact the physiological response to muscle contraction. Previous study has reported MRI-BOLD to be on average 2.3% above rest in boys with DMD. We measured the post-contractile BOLD response twice in each participant; once after taking tadalafil (a vasodilator proposed to increase blood flow and microvascular perfusion) and once without taking the tadalafil. The order of assessment was randomized.
Measured: MRI will be done 3 hours after dosing/no-dosing on two separate study visits
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jan 2022
- Study start14 Dec 2021
- Primary completion16 Apr 2024
- Study completion8 Sep 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by University of FloridaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Pre/post single dose tadalafil
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Tanja Taivassalo, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05195775
- Sponsor's protocol ID
- IRB202102391
- Other IDs
- OCR41391
- Status last verified
- Feb 2025
- First submitted
- 4 Jan 2022
- Last updated
- 27 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tadalafil as an Adjuvant to Therapy for Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT05195775
- Acronym / sponsor ID
- IRB202102391
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- therapy, treatment strategy, vasodilator, exercise
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Study start
- 14 Dec 2021 (actual)
- Primary completion
- 16 Apr 2024 (actual)
- Study completion
- 8 Sep 2025 (actual)
- First posted
- 19 Jan 2022 (actual)
- Results
- Posted 27 Oct 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This project will assess the vascular responsiveness in leg muscles of boys with Duchenne muscular dystrophy (DMD) to one single dose of tadalafil, a common vasodilator drug, using non-invasive techniques (MRI or Doppler ultrasound). These findings will provide proof of concept for a subsequent intervention study to demonstrate efficacy of longer-term tadalafil to counter sympathetic vasoconstriction and slow disease progression in DMD.
Detailed description
This project will target up to 25 ambulatory boys with DMD aged 7-30 years. Because the vascular impact of tadalafil is immediate, we will confirm that the drug target is valid target in lower extremities by assessing the change in post-exercise microvascular perfusion using Blood Oxygen Level-Dependent (BOLD) MRI or change in post-exercise hyperemia using Doppler ultrasonography. Patients will be randomized to undergo the above-mentioned assessments with or without the study drug on two separate visits.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Pre/post single dose tadalafil
| Arm | Type | Receives |
|---|---|---|
| Tadalafil plus BOLD-MRI | Experimental | Drug: TadalafilThis arm will use BOLD-MRI as the technique to monitor drug impact on skeletal muscle. |
Who may qualify
- Ages
- 6 to 13 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 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
- Ambulatory
Key exclusion criteria (5 listed)
- Older than 13.0 years of age
- Contraindication to an MR examination (e.g. aneurysm clip, severe claustrophobia, magnetic implants)
- Presence of unstable medical problems
- Presence of a secondary condition that impacts muscle function or muscle metabolism (e.g. myasthenia gravis, endocrine disorder, mitochondrial disease)
- Contraindications to Tadalafil (use of nitrates, alpha-adrenergic blockers, other phosphodiesterase 5A inhibitors)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TadalafilAlso: CialisPatient will be randomized to one dose of 0.6 mg/kg tadalafil on one of two study visits. On both visits, assessments (MRI, exercise testing, clinical function) will be performed 3 hours after time of dosing. | Drug | Tadalafil plus BOLD-MRI |
Primary outcome
Percent Change in Post-contractile BOLD Signal Relative to Resting Condition
Time frame: MRI will be done 3 hours after dosing/no-dosing on two separate study visits
The post contractile blood oxygen level-dependent (BOLD) response is a non-invasive index of microvascular function in muscle using MRI. BOLD images are acquired from the tibialis anterior before and after dorsiflexion exercise. A transient increase in BOLD signal is expected after performing a brief muscle contraction (reflecting increased small vessel oxygen saturation and blood volume). In healthy boys, MRI-BOLD signal was on average 4.4% above resting levels. The greater the value, the more intact the physiological response to muscle contraction. Previous study has reported MRI-BOLD to be on average 2.3% above rest in boys with DMD. We measured the post-contractile BOLD response twice in each participant; once after taking tadalafil (a vasodilator proposed to increase blood flow and microvascular perfusion) and once without taking the tadalafil. The order of assessment was randomized.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Tanja Taivassalo principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Tadalafil in people with Duchenne Muscular Dystrophy, run by University of Florida at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 Oct 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Change in Post-contractile BOLD Signal Relative to Resting Condition (time frame: MRI will be done 3 hours after dosing/no-dosing on two separate study visits). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Oct 2026.