Becker Muscular DystrophyPhase 1NCT02847975
Sodium Nitrate to Improve Blood Flow
A Phase 1 study testing Sodium nitrate in people with Becker Muscular Dystrophy, run by Cedars-Sinai Medical Center at 2 sites in United States.
Trial summary
Investigators recently showed that tadalafil restores functional sympatholysis in patients with Becker muscular dystrophy (BMD). If tadalafil restores functional sympatholysis in BMD via the NO-cyclic guanosine monophosphate pathway, then functional sympatholysis should also be restored by sodium nitrite- which is an… Read more about this study
- Who is it for?
- Ages 15 to 55 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Becker Muscular Dystrophy
- What is being tested?
- Sodium nitrate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 2 sitesUnited States
- Study dates
- Oct 2013to Dec 2014
- Phase
- 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 15 to 55 years
- Men only
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Becker muscular dystrophy
- age 15-55 years of age
- ambulatory
Who cannot join
- hypertension, diabetes, or heart failure by standard clinical criteria
- elevated brain natriuretic peptide level (>100 pg/ml)
- Left ventricular ejection fraction < 50%
- cardiac rhythm disorder, specifically: rhythm other than sinus, supraventricular tachycardia, atrial fibrillation, ventricular tachycardia, heart block
- continuous ventilatory support
- liver disease
Show all 10Show fewer
- renal impairment
- history of asthma or bronchospasm
- use of any medications other than common supplements
- unable to perform handgrip exercise
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Cedars-Sinai Heart Institute
Los Angeles, California, 90048
Cedars-Sinai Medical Center
Los Angeles, California, 90048
About this study
Investigators recently showed that tadalafil restores functional sympatholysis in patients with Becker muscular dystrophy (BMD). If tadalafil restores functional sympatholysis in BMD via the NO-cyclic guanosine monophosphate pathway, then functional sympatholysis should also be restored by sodium nitrite- which is an indirect nitric oxide donor.
- Conditions
- Becker Muscular Dystrophy
- Being tested
- Sodium nitrate
- Keywords
- Becker, dystrophy, sympatholysis
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sodium nitrateDietary supplement
Sodium nitrate will be ingested orally
| Study group | Role | What they receive |
|---|---|---|
| Sodium nitrate | Experimental | Sodium nitrate will be administered orally and functional sympatholysis assessed before and after treatment Receives: Dietary Supplement: Sodium nitrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
change in muscle tissue oxygenation
The pre-specified primary outcome is the pre vs. post treatment change in functional sympatholysis measured by muscle oxygenation.
Measured: change from baseline to post treatment (3-4 hours)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jul 2016 (estimated)
- Study startOct 2013
- Primary completionDec 2014
- Study completionDec 2014
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 1
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cedars-Sinai Medical Center
- Sponsor type
- University, hospital or other
- 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
- NCT02847975
- Sponsor's protocol ID
- Pro00033300
- Status last verified
- Aug 2018
- First submitted
- 30 Apr 2014
- Last updated
- 6 Aug 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Published http://www.ncbi.nlm.nih.gov/pubmed/26437761
Publications
Martin EA, Barresi R, Byrne BJ, Tsimerinov EI, Scott BL, Walker AE, Gurudevan SV, Anene F, Elashoff RM, Thomas GD, Victor RG. Tadalafil alleviates muscle ischemia in patients with Becker muscular dystrophy. Sci Transl Med. 2012 Nov 28;4(162):162ra155. doi: 10.1126/scitranslmed.3004327.
PubMed 23197572
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sodium Nitrate to Improve Blood Flow
- ClinicalTrials.gov ID
- NCT02847975
- Acronym / sponsor ID
- Pro00033300
- Conditions
- Becker Muscular Dystrophy
- Keywords
- Becker, dystrophy, sympatholysis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Cedars-Sinai Medical CenterUniversity, hospital or other
- Study start
- Oct 2013
- Primary completion
- Dec 2014 (actual)
- Study completion
- Dec 2014 (actual)
- First posted
- 28 Jul 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.
Investigators recently showed that tadalafil restores functional sympatholysis in patients with Becker muscular dystrophy (BMD). If tadalafil restores functional sympatholysis in BMD via the NO-cyclic guanosine monophosphate pathway, then functional sympatholysis should also be restored by sodium nitrite- which is an indirect nitric oxide donor.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sodium nitrate | Experimental | Dietary Supplement: Sodium nitrateSodium nitrate will be administered orally and functional sympatholysis assessed before and after treatment |
Who may qualify
- Ages
- 15 to 55 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Becker muscular dystrophy
- age 15-55 years of age
- ambulatory
Key exclusion criteria (10 listed)
- hypertension, diabetes, or heart failure by standard clinical criteria
- elevated brain natriuretic peptide level (>100 pg/ml)
- Left ventricular ejection fraction < 50%
- cardiac rhythm disorder, specifically: rhythm other than sinus, supraventricular tachycardia, atrial fibrillation, ventricular tachycardia, heart block
- continuous ventilatory support
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 |
|---|---|---|
| Sodium nitrateSodium nitrate will be ingested orally | Dietary supplement | Sodium nitrate |
Primary outcome
change in muscle tissue oxygenation
Time frame: change from baseline to post treatment (3-4 hours)
The pre-specified primary outcome is the pre vs. post treatment change in functional sympatholysis measured by muscle oxygenation.
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 6 Aug 2018.
- Background: PubMed 23197572Martin EA, Barresi R, Byrne BJ, Tsimerinov EI, Scott BL, Walker AE, Gurudevan SV, Anene F, Elashoff RM, Thomas GD, Victor RG. Tadalafil alleviates muscle ischemia in patients with Becker muscular dystrophy. Sci Transl Med. 2012 Nov 28;4(162):162ra155. doi: 10.1126/scitranslmed.3004327.
- Individual participant dataSharing planned: No. Published http://www.ncbi.nlm.nih.gov/pubmed/26437761
Related trials for Becker Muscular Dystrophy
RecruitingObservational study
Biomarker Development for Muscular Dystrophies
Myotonic Dystrophy, Duchenne Muscular Dystrophy
Active, not recruitingObservational study
Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Recruiting
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Rare Genetic Muscle Diseases, Muscular Dystrophy, Duchenne
Active, not recruitingPhase 1
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy
RecruitingObservational study
Does Chronic Pain Change Over Time in Children With Duchenne Muscular Dystrophy?
Duchenne Muscular Dystrophy (DMD), Chronic Pain
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 1 study testing Sodium nitrate 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?
The sponsor lists 3 inclusion and 10 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 6 Aug 2018. 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
change in muscle tissue oxygenation (time frame: change from baseline to post treatment (3-4 hours)). 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 NCT02847975, 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 6 Aug 2018.