Becker Muscular DystrophyPhase 4NCT01070511
Tadalafil in Becker Muscular Dystrophy
A Phase 4 study testing Tadalafil in people with Becker Muscular Dystrophy, run by Cedars-Sinai Medical Center at 2 sites in United States.
Trial summary
Summary for Patients: This study, funded by the Muscular Dystrophy Association, is intended to build on recent findings published in the journal Nature showing beneficial effects of tadalafil (also known as Cialis) in mice with an animal version of Duchenne and Becker muscular dystrophies. Read more about this study
- Who is it for?
- Ages 18 to 55 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Becker Muscular Dystrophy
- What is being tested?
- Tadalafil
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 48 participants
- Where
- 2 sitesUnited States
- Study dates
- Jan 2010to Dec 2012
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 18 to 55 years
- Men only
- Healthy volunteers accepted
- Adult
Becker Muscular Dystrophy Patients
- Men 18-55 years of age with a pre-existing diagnosis of Becker Muscular Dystrophy by a clinical neurologist (based on clinical criteria plus previous muscle biopsy analysis and/or DNA analysis).
Healthy Controls
- Men 18-55 years of age with no known medical conditions
Criteria for exclusion of subjects (both patients and controls)
- Any evidence of cardiopulmonary disease by history or by physical examination
- History of hypertension or blood pressure averaging ≥140/90 mmHg
- Diabetes mellitus or other systemic illness
- Heart failure by clinical exam, elevated BNP, or heart failure medication
- Serum creatinine ≥ 1.5 mg/dL
- Any history of substance abuse (including alcohol)
- Any history of psychiatric illness
- Contraindications to tadalafil (use of nitrates, alpha-blockers, other PDE5A inhibitors, or potent inhibitors of CYP3A4 such as ketoconazole or ritonavir)
- Contraindications to MRI (claustrophobia, metal implants, or seizure disorder)
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Cedars-Sinai Medical Center
Los Angeles, California, 90048
Cedars Sinai Medical Center
Los Angeles, California, 9004
About this study
Official title: Functional Muscle Ischemia and PDE5A Inhibition in Becker Muscular Dystrophy
Summary for Patients: This study, funded by the Muscular Dystrophy Association, is intended to build on recent findings published in the journal Nature showing beneficial effects of tadalafil (also known as Cialis) in mice with an animal version of Duchenne and Becker muscular dystrophies. Only two doses of tadalafil improved muscle blood flow, allowing the dystrophic mice to perform more exercise with less muscle injury. This new short-term clinical trial will move the testing from animals to human patients with Becker muscular dystrophy and examine the effects of acute tadalafil dosing on muscle blood flow during a bout of exercise. Patients will take two doses of tadalafil prior to exercising. Then doctors will measure whether muscles receive increased blood flow and therefore are better protected during exercise.
Scientific Hypothesis: In patients with Becker muscular dystrophy (particularly those with dystrophin gene mutations between exons 41-46), loss of sarcolemmal nitric oxide synthase engenders functional muscle ischemia and thus muscle edema after an acute bout of exercise. The investigators further hypothesize that PDE5A inhibition, which boosts nitric oxide-cGMP signaling, constitutes an effective new countermeasure for these patients.
Read the detailed description
There are 2 phases to this research project: (1) an initial baseline study to compare patients with Becker muscular dystrophy against healthy control subjects, and (2) a subsequent brief treatment trial only in the muscular dystrophy patients (healthy controls will not participate in this second phase of the study).
The baseline study involves an intake history, physical examination, and phlebotomy for blood chemistries and DNA followed by (a) MRI scans of the forearm muscles before and after a brief bout of handgrip exercise approximately 1-1.5 hours to complete), and (b) non-invasive forearm blood flow studies (Near Infrared Spectroscopy, Doppler Ultrasound) before and after a brief bout of handgrip exercise (approximately 3-5 hours to complete). Blood flow studies will be performed with the subject's lower body enclosed in an airtight chamber. Blood flow and oxygen delivery to the forearm muscles will be measured before and during application of lower body negative pressure at rest and during handgrip exercise. Lower body negative pressure simulates the blood flow changes that normally occur when a person sits up after lying down.
The results of the baseline study will determine which patients meet preset eligibility criteria to participate in the medication phase of the study. These criteria include (1) normal kidney and liver function tests and normal BNP levels (the latter to exclude heart failure), and (2) abnormal MRI and blood flow responses to handgrip exercise. Eligible patients will be asked to repeat the above laboratory procedures on two more study days: one day after receiving 2 pills of tadalafil (a PDE5A inhibitor that is longer-lasting than Viagra) and another day after receiving 2 placebo pills. Study procedures can be broken up into two separate days for scheduling purposes and/or to avoid fatigue. Patients completing the trial will receive both study medications and the order will be random.
In summary, healthy control subjects will only undergo the baseline study whereas Becker muscular dystrophy patients completing all phases of the project will have 3 separate study days (baseline, tadalafil, and placebo) and 2 medication visits (to receive the study medication and have their blood pressure checked by a study physician). Study days will be separated by at least two weeks to allow time for test results, to rest the forearm muscles between exercise bouts, and to ensure complete elimination of tadalafil.
We plan to enroll 24 adult men with Becker muscular dystrophy, of whom 12 men will have dystrophin gene deletions between exons 41-46, and 24 healthy control subjects.
- Conditions
- Becker Muscular Dystrophy
- Being tested
- Tadalafil, Placebo
- Keywords
- Muscular Dystrophy, Tadalafil
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TadalafilDrug
Also called Cialis
Tadalafil will be administered orally on two consecutive days. The first dose-10 mg- will be administered the afternoon before the study. The second dose -20 mg-will be administered the morning of the study.
PlaceboDrug
A placebo pill will be administered orally on two consecutive days. The first pill will be administered the afternoon before the study. The second pill will be administered the morning of the study.
| Study group | Role | What they receive |
|---|---|---|
| Tadalafil | Experimental | Not described Receives: Drug: Tadalafil |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reflex decrease in muscle tissue oxygenation (i.e., adrenergic vasoconstriction) during rhythmic handgrip exercise measured by Near Infrared Spectroscopy (NIR).
Measured: measured at a minimum of 2 week intervals for a minimum of 6 weeks total (for subjects with BMD)
Secondary outcomes (1)
Change in forearm muscle water content by magnetic resonance imaging (MRI).
Measured: measured at a minimum of 2 week intervals for a minimum of 6 weeks total (for subjects with BMD)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Feb 2010 (estimated)
- Study startJan 2010
- Primary completionDec 2012
- Study completionDec 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Becker Muscular DystrophyStudies of the same condition, open and under way.
- 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 4
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cedars-Sinai Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Muscular Dystrophy Association
- Responsible party
- Principal investigator, Ron Victor, Cedars-Sinai Medical Center
- Investigators
- Ronald G Victor, MD, Principal investigator, Cedars-Sinai Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01070511
- Sponsor's protocol ID
- MDA 158944
- Status last verified
- Aug 2013
- First submitted
- 17 Feb 2010
- Last updated
- 20 Aug 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Mickle AT, Johnston KM, Ricchetti-Masterson KL, Kennedy AR, Korpach SG, Gooch KL. The natural history of Becker muscular dystrophy: A systematic literature review. J Neuromuscul Dis. 2026 Feb 1:22143602261420045. doi: 10.1177/22143602261420045. Online ahead of print.
PubMed 41671094
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Functional Muscle Ischemia and PDE5A Inhibition in Becker Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT01070511
- Acronym / sponsor ID
- MDA 158944
- Conditions
- Becker Muscular Dystrophy
- Keywords
- Muscular Dystrophy, Tadalafil
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Cedars-Sinai Medical CenterUniversity, hospital or other
- Collaborators
- Muscular Dystrophy Association
- Study start
- Jan 2010
- Primary completion
- Dec 2012 (actual)
- Study completion
- Dec 2012 (actual)
- First posted
- 18 Feb 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Summary for Patients: This study, funded by the Muscular Dystrophy Association, is intended to build on recent findings published in the journal Nature showing beneficial effects of tadalafil (also known as Cialis) in mice with an animal version of Duchenne and Becker muscular dystrophies. Only two doses of tadalafil improved muscle blood flow, allowing the dystrophic mice to perform more exercise with less muscle injury. This new short-term clinical trial will move the testing from animals to human patients with Becker muscular dystrophy and examine the effects of acute tadalafil dosing on muscle blood flow during a bout of exercise. Patients will take two doses of tadalafil prior to exercising. Then doctors will measure whether muscles receive increased blood flow and therefore are better protected during exercise.
Scientific Hypothesis: In patients with Becker muscular dystrophy (particularly those with dystrophin gene mutations between exons 41-46), loss of sarcolemmal nitric oxide synthase engenders functional muscle ischemia and thus muscle edema after an acute bout of exercise. The investigators further hypothesize that PDE5A inhibition, which boosts nitric oxide-cGMP signaling, constitutes an effective new countermeasure for these patients.
Detailed description
There are 2 phases to this research project: (1) an initial baseline study to compare patients with Becker muscular dystrophy against healthy control subjects, and (2) a subsequent brief treatment trial only in the muscular dystrophy patients (healthy controls will not participate in this second phase of the study).
The baseline study involves an intake history, physical examination, and phlebotomy for blood chemistries and DNA followed by (a) MRI scans of the forearm muscles before and after a brief bout of handgrip exercise approximately 1-1.5 hours to complete), and (b) non-invasive forearm blood flow studies (Near Infrared Spectroscopy, Doppler Ultrasound) before and after a brief bout of handgrip exercise (approximately 3-5 hours to complete). Blood flow studies will be performed with the subject's lower body enclosed in an airtight chamber. Blood flow and oxygen delivery to the forearm muscles will be measured before and during application of lower body negative pressure at rest and during handgrip exercise. Lower body negative pressure simulates the blood flow changes that normally occur when a person sits up after lying down.
The results of the baseline study will determine which patients meet preset eligibility criteria to participate in the medication phase of the study. These criteria include (1) normal kidney and liver function tests and normal BNP levels (the latter to exclude heart failure), and (2) abnormal MRI and blood flow responses to handgrip exercise. Eligible patients will be asked to repeat the above laboratory procedures on two more study days: one day after receiving 2 pills of tadalafil (a PDE5A inhibitor that is longer-lasting than Viagra) and another day after receiving 2 placebo pills. Study procedures can be broken up into two separate days for scheduling purposes and/or to avoid fatigue. Patients completing the trial will receive both study medications and the order will be random.
In summary, healthy control subjects will only undergo the baseline study whereas Becker muscular dystrophy patients completing all phases of the project will have 3 separate study days (baseline, tadalafil, and placebo) and 2 medication visits (to receive the study medication and have their blood pressure checked by a study physician). Study days will be separated by at least two weeks to allow time for test results, to rest the forearm muscles between exercise bouts, and to ensure complete elimination of tadalafil.
We plan to enroll 24 adult men with Becker muscular dystrophy, of whom 12 men will have dystrophin gene deletions between exons 41-46, and 24 healthy control subjects.
Study design
- Primary purpose
- Basic science
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Tadalafil | Experimental | Drug: Tadalafil |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult
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: CialisTadalafil will be administered orally on two consecutive days. The first dose-10 mg- will be administered the afternoon before the study. The second dose -20 mg-will be administered the morning of the study. | Drug | Tadalafil |
| PlaceboA placebo pill will be administered orally on two consecutive days. The first pill will be administered the afternoon before the study. The second pill will be administered the morning of the study. | Drug | Placebo |
Primary outcome
Reflex decrease in muscle tissue oxygenation (i.e., adrenergic vasoconstriction) during rhythmic handgrip exercise measured by Near Infrared Spectroscopy (NIR).
Time frame: measured at a minimum of 2 week intervals for a minimum of 6 weeks total (for subjects with BMD)
1 secondary outcome
Change in forearm muscle water content by magnetic resonance imaging (MRI).
Time frame: measured at a minimum of 2 week intervals for a minimum of 6 weeks total (for subjects with BMD)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2013.
- Derived from this study: PubMed 41671094Mickle AT, Johnston KM, Ricchetti-Masterson KL, Kennedy AR, Korpach SG, Gooch KL. The natural history of Becker muscular dystrophy: A systematic literature review. J Neuromuscul Dis. 2026 Feb 1:22143602261420045. doi: 10.1177/22143602261420045. Online ahead of print.
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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.
Ronald G Victor, MD principal investigator
Cedars-Sinai Medical Center
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Tadalafil in people with Becker Muscular Dystrophy, run by Cedars-Sinai Medical Center at 2 sites 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?
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 20 Aug 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Reflex decrease in muscle tissue oxygenation (i.e., adrenergic vasoconstriction) during rhythmic handgrip exercise measured by Near Infrared Spectroscopy (NIR). (time frame: measured at a minimum of 2 week intervals for a minimum of 6 weeks total (for subjects with BMD)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01070511, which also lists 1 linked publication.
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 20 Aug 2013; AzTrials last refreshed its copy on 29 Oct 2026.