Muscular DystrophyEarly Phase 1NCT02653833
The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy
A Early Phase 1 study testing Tadalafil 20 MG and beetroot juice extract in people with Muscular Dystrophy, run by Cedars-Sinai Medical Center at 1 site in United States.
Trial summary
This pilot study tests the hypothesis that the medication nitric oxide extract from beetroot juice improves blood flow to the skeletal muscle during exercise. The investigators will use cutting edge technology with contrast enhanced ultrasound to visualize the microvascular blood supply to the forearm. Read more about this study
- Who is it for?
- Ages 18 to 45 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Muscular Dystrophy
- What is being tested?
- Tadalafil 20 MG and beetroot juice extract
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Nov 2017to 1 Sep 2018
- Phase
- Early Phase 1
- 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 45 years
- Men only
- Healthy volunteers accepted
- Adult
Who may be able to join
- Healthy man, aged 18 to 45 years, currently taking no medication. OR
- Clinical diagnosis of Becker Muscular Dystrophy (BMD), aged 18 to 45 years, and currently taking no medication.
Who cannot join
- Hypertension, diabetes, heart failure, liver disease
- ECG evidence of prolonged QT interval
Trial locations and contacts
1 site in 1 country.
United States1 site
Cedars-Sinai Medical Center
Los Angeles, California, 90048
About this study
Official title: Skeletal Muscle Blood Flow in Becker Muscular Dystrophy
This pilot study tests the hypothesis that the medication nitric oxide extract from beetroot juice improves blood flow to the skeletal muscle during exercise. The investigators will use cutting edge technology with contrast enhanced ultrasound to visualize the microvascular blood supply to the forearm. Animal studies have shown reversal of muscle damage with improved delivery of blood to the exercising muscle. This research aims to understand the mechanism of action of this medication in a way it has never been studied before. The results may help benefit individuals with muscular Dystrophy in the future.
Read the detailed description
The subjects are asked to perform a graded hand-grip exercise while blood flow to the skeletal muscle is visualized by contrast enhanced ultrasound.
This is done at baseline and after taking the study agent.
- Conditions
- Muscular Dystrophy
- Being tested
- Tadalafil 20 MG, beetroot juice extract
- Keywords
- Becker, Muscular Dystrophy, Skeletal Muscle, blood flow, Ultrasound
- Medical subject headings
- Muscular Dystrophies, Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tadalafil 20 MGDrug
Blood flow to the forearm is visualized via an ultrasound technique at rest and with graded exercise. The experiment is then repeated shortly after the subject takes the study agent, beetroot juice extract.
beetroot juice extractOther
Blood flow to the forearm is visualized via an ultrasound technique at rest and with graded exercise. The experiment is then repeated shortly after the subject takes the study agent, beetroot juice extract.
| Study group | Role | What they receive |
|---|---|---|
| Healthy Controls (HC) | Experimental | Healthy men currently taking no medications will be asked to perform graded hand-grip exercise while a contrast enhanced ultrasound (CEU) probe is placed over their forearm to measure blood flow. This is repeated at baseline and shortly after taking beetroot juice extract. Receives: Drug: Tadalafil 20 MG; Other: beetroot juice extract |
| Becker Muscular Dystrophy (BMD) | Experimental | currently taking no medications will be asked to perform graded hand-grip exercise while a contrast enhanced ultrasound (CEU) probe is placed over their forearm to measure blood flow. Using a lower body negative pressure (LBNP) chamber, the subjects are tested for impaired exercise induced vasodilation to the skeletal muscle or "functional sympatholysis". This is repeated at baseline and shortly after taking beetroot juice extract. Receives: Drug: Tadalafil 20 MG; Other: beetroot juice extract |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Skeletal Muscle Blood Flow
Exercise induced skeletal muscle perfusion is measured by contrast-enhanced ultrasound (CEU) during graded voluntary contraction on a hand grip dynamometer.
Measured: At baseline and within one hours after taking beetroot juice or Tadalafil
Secondary outcomes (2)
Change in Measured Functional Sympatholysis
Functional sympatholysis (attenuation of exercise induced skeletal muscle vasoconstriction) is assessed using a lower body negative pressure chamber.
Measured: At baseline, and within an hour after taking beetroot juice or Tadalafil
Change in Measured Flow Mediated Dilation (FMD)
Endothelial function is assessed by measuring flow-mediated dilation (FMD) by Doppler ultrasound.
Measured: At baseline, and within an hour after taking beetroot juice or Tadalafil
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jan 2016 (estimated)
- Study start1 Nov 2017
- Primary completion1 Sep 2018
- Study completion1 Sep 2018
Why it stopped: PI passed away
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by Cedars-Sinai Medical CenterThe 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
- Early Phase 1
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cedars-Sinai Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Ronald G Victor, MD, Principal investigator, Cedars-Sinai Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02653833
- Sponsor's protocol ID
- Pro00041688
- Status last verified
- Jun 2019
- First submitted
- 14 Dec 2015
- Last updated
- 14 Jan 2020
- Results posted
- No
- 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 14 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Skeletal Muscle Blood Flow in Becker Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02653833
- Acronym / sponsor ID
- Pro00041688
- Conditions
- Muscular Dystrophy
- Keywords
- Becker, Muscular Dystrophy, Skeletal Muscle, blood flow, Ultrasound
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: PI passed away
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Cedars-Sinai Medical CenterUniversity, hospital or other
- Study start
- 1 Nov 2017 (actual)
- Primary completion
- 1 Sep 2018 (actual)
- Study completion
- 1 Sep 2018 (actual)
- First posted
- 12 Jan 2016 (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.
This pilot study tests the hypothesis that the medication nitric oxide extract from beetroot juice improves blood flow to the skeletal muscle during exercise. The investigators will use cutting edge technology with contrast enhanced ultrasound to visualize the microvascular blood supply to the forearm. Animal studies have shown reversal of muscle damage with improved delivery of blood to the exercising muscle. This research aims to understand the mechanism of action of this medication in a way it has never been studied before. The results may help benefit individuals with muscular Dystrophy in the future.
Detailed description
The subjects are asked to perform a graded hand-grip exercise while blood flow to the skeletal muscle is visualized by contrast enhanced ultrasound.
This is done at baseline and after taking the study agent.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Healthy Controls (HC) | Experimental | Drug: Tadalafil 20 MG, Other: beetroot juice extractHealthy men currently taking no medications will be asked to perform graded hand-grip exercise while a contrast enhanced ultrasound (CEU) probe is placed over their forearm to measure blood flow. This is repeated at baseline and shortly after taking beetroot juice extract. |
| Becker Muscular Dystrophy (BMD) | Experimental | Drug: Tadalafil 20 MG, Other: beetroot juice extractcurrently taking no medications will be asked to perform graded hand-grip exercise while a contrast enhanced ultrasound (CEU) probe is placed over their forearm to measure blood flow. Using a lower body negative pressure (LBNP) chamber, the subjects are tested for impaired exercise induced vasodilation to the skeletal muscle or "functional sympatholysis". This is repeated at baseline and shortly after taking beetroot juice extract. |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (2 listed)
- Healthy man, aged 18 to 45 years, currently taking no medication. OR
- Clinical diagnosis of Becker Muscular Dystrophy (BMD), aged 18 to 45 years, and currently taking no medication.
Key exclusion criteria (2 listed)
- Hypertension, diabetes, heart failure, liver disease
- ECG evidence of prolonged QT interval
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 |
|---|---|---|
| Tadalafil 20 MGBlood flow to the forearm is visualized via an ultrasound technique at rest and with graded exercise. The experiment is then repeated shortly after the subject takes the study agent, beetroot juice extract. | Drug | Becker Muscular Dystrophy (BMD), Healthy Controls (HC) |
| beetroot juice extractBlood flow to the forearm is visualized via an ultrasound technique at rest and with graded exercise. The experiment is then repeated shortly after the subject takes the study agent, beetroot juice extract. | Other | Becker Muscular Dystrophy (BMD), Healthy Controls (HC) |
Primary outcome
Change in Skeletal Muscle Blood Flow
Time frame: At baseline and within one hours after taking beetroot juice or Tadalafil
Exercise induced skeletal muscle perfusion is measured by contrast-enhanced ultrasound (CEU) during graded voluntary contraction on a hand grip dynamometer.
2 secondary outcomes
Change in Measured Functional Sympatholysis
Time frame: At baseline, and within an hour after taking beetroot juice or Tadalafil
Change in Measured Flow Mediated Dilation (FMD)
Time frame: At baseline, and within an hour after taking beetroot juice or Tadalafil
Results and safety
The study was stopped early (reason given: PI passed away). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jan 2020.
- 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.
Ronald G Victor, MD principal investigator
Cedars-Sinai Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Tadalafil 20 MG and beetroot juice extract in people with Muscular Dystrophy, run by Cedars-Sinai Medical Center 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 2 inclusion and 2 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: stopped early, as of the sponsor's last update on 14 Jan 2020. 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 phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 was stopped early (reason given: PI passed away). Results are not available in the source record.
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?
Change in Skeletal Muscle Blood Flow (time frame: At baseline and within one hours after taking beetroot juice or Tadalafil). 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 14 Jan 2020.