Becker Muscular DystrophyPhase 2/3NCT02147639
Effects of Sodium Nitrate on Blood Flow in Becker Muscular Dystrophy
A Phase 2/3 study testing Sodium Nitrate, Sodium Nitrate - double dose and Increased exercise intensity in people with Becker Muscular Dystrophy, run by Cedars-Sinai Medical Center at 1 site in United States.
Trial summary
This study is intended to build on a growing body of literature showing a blood flow abnormality in patients with Becker muscular dystrophy. The investigators' laboratory recently showed that this blood flow abnormality could be corrected by a single oral dose of the drug Tadalafil (also known as Cialis). Read more about this study
- Who is it for?
- Ages 15 to 45 years, men only
- Conditions the study is for
- Becker Muscular Dystrophy
- What is being tested?
- Sodium Nitrate, Sodium Nitrate - double dose and Increased exercise intensity
- Type of treatment
- Pill or oral medicine, Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 19 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2013to May 2014
- Phase
- Phase 2/3
- 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 45 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Men 15-55 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).
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Cedars-Sinai Heart Institute
Los Angeles, California, 90048
About this study
This study is intended to build on a growing body of literature showing a blood flow abnormality in patients with Becker muscular dystrophy. The investigators' laboratory recently showed that this blood flow abnormality could be corrected by a single oral dose of the drug Tadalafil (also known as Cialis). The investigators now wish to replicate these exciting results using a common nitric oxide donor (sodium nitrate).
Read the detailed description
There are 2 phases to this research project: (1) an initial baseline study to confirm the blood flow abnormality in Becker muscular dystrophy, and (2) a subsequent brief treatment trial. There are also 3 optional study protocols: (A) dose-escalation trial, (B) placebo trial, and (C) increased exercise intensity trial.
The baseline study involves an intake history, physical examination, and phlebotomy for blood chemistries and DNA followed by 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 blood flow responses to handgrip exercise.
Eligible patients will be asked to repeat the above laboratory procedures on a subsequent study day after receiving a single dose of sodium nitrate. Eligible patients will also be offered the option to repeat the above laboratory procedures on three subsequent study visits, where upon: (A) the dose of sodium nitrate is increased, (B) a placebo is ingested, and/or (C) the level of exercise in increased.
The investigators plan to enroll 20 adult men with Becker muscular dystrophy.
- Conditions
- Becker Muscular Dystrophy
- Being tested
- Sodium Nitrate, Sodium Nitrate - double dose, Placebo, Increased exercise intensity
- 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 - double doseDietary supplement
PlaceboDietary supplement
Increased exercise intensityProcedure or surgery
| Study group | Role | What they receive |
|---|---|---|
| Sodium Nitrate | Experimental | Patients will ingest a single oral dose of sodium nitrate (~8.4 mmol) Receives: Dietary Supplement: Sodium Nitrate |
| Baseline | No intervention | This is a baseline study visit, which will serve to assess inclusion and exclusion criteria, as well as provide untreated measurments of skeletal muscle blood flow and perfusion. |
| Dose-escalation trial | Experimental | This is an optional study visit, where subjects will ingest twice the dose of sodium nitrate (~16.8 mmol). Receives: Dietary Supplement: Sodium Nitrate - double dose |
| Placebo-control trial | Placebo comparator | This is an optional study visit, where patients will ingest a placebo. Receives: Dietary Supplement: Placebo |
| Increased exercise intensity | Experimental | This is an optional study visit, where patients will be asked to repeat all of the blood flow assessments, but the exercise intensity will be increased. Receives: Procedure: Increased exercise intensity |
What is being measured
The primary outcome is the main question the study is designed to answer.
Skeletal muscle blood flow
Skeletal muscle blood flow regulation will be assessed at each visit by near infrared spectroscopy and Doppler ultrasound.
Measured: 24 hours after initial visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2014 (estimated)
- Study startOct 2013
- Primary completionMay 2014
- Study completionMay 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.
- 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
- Phase 2/3
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 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
- NCT02147639
- Sponsor's protocol ID
- NANO3
- Status last verified
- May 2014
- First submitted
- 25 Mar 2014
- Last updated
- 14 Jan 2020
- Results posted
- No
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 14 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effects of Sodium Nitrate on Blood Flow in Becker Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02147639
- Acronym / sponsor ID
- NANO3
- Conditions
- Becker Muscular Dystrophy
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Cedars-Sinai Medical CenterUniversity, hospital or other
- Study start
- Oct 2013 (actual)
- Primary completion
- May 2014 (actual)
- Study completion
- May 2014 (actual)
- First posted
- 28 May 2014 (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 study is intended to build on a growing body of literature showing a blood flow abnormality in patients with Becker muscular dystrophy. The investigators' laboratory recently showed that this blood flow abnormality could be corrected by a single oral dose of the drug Tadalafil (also known as Cialis). The investigators now wish to replicate these exciting results using a common nitric oxide donor (sodium nitrate).
Detailed description
There are 2 phases to this research project: (1) an initial baseline study to confirm the blood flow abnormality in Becker muscular dystrophy, and (2) a subsequent brief treatment trial. There are also 3 optional study protocols: (A) dose-escalation trial, (B) placebo trial, and (C) increased exercise intensity trial.
The baseline study involves an intake history, physical examination, and phlebotomy for blood chemistries and DNA followed by 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 blood flow responses to handgrip exercise.
Eligible patients will be asked to repeat the above laboratory procedures on a subsequent study day after receiving a single dose of sodium nitrate. Eligible patients will also be offered the option to repeat the above laboratory procedures on three subsequent study visits, where upon: (A) the dose of sodium nitrate is increased, (B) a placebo is ingested, and/or (C) the level of exercise in increased.
The investigators plan to enroll 20 adult men with Becker muscular dystrophy.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Sodium Nitrate | Experimental | Dietary Supplement: Sodium NitratePatients will ingest a single oral dose of sodium nitrate (~8.4 mmol) |
| Baseline | No intervention | –This is a baseline study visit, which will serve to assess inclusion and exclusion criteria, as well as provide untreated measurments of skeletal muscle blood flow and perfusion. |
| Dose-escalation trial | Experimental | Dietary Supplement: Sodium Nitrate - double doseThis is an optional study visit, where subjects will ingest twice the dose of sodium nitrate (~16.8 mmol). |
| Placebo-control trial | Placebo comparator | Dietary Supplement: PlaceboThis is an optional study visit, where patients will ingest a placebo. |
| Increased exercise intensity | Experimental | Procedure: Increased exercise intensityThis is an optional study visit, where patients will be asked to repeat all of the blood flow assessments, but the exercise intensity will be increased. |
Who may qualify
- Ages
- 15 to 45 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Men 15-55 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).
Key exclusion criteria (7 listed)
- 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
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 Nitrate | Dietary supplement | Sodium Nitrate |
| Sodium Nitrate - double dose | Dietary supplement | Dose-escalation trial |
| Placebo | Dietary supplement | Placebo-control trial |
| Increased exercise intensity | Procedure or surgery | Increased exercise intensity |
Primary outcome
Skeletal muscle blood flow
Time frame: 24 hours after initial visit
Skeletal muscle blood flow regulation will be assessed at each visit by near infrared spectroscopy and Doppler ultrasound.
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 14 Jan 2020.
- 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.
Related trials for Becker Muscular Dystrophy
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy
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 Phase 2/3 study testing Sodium Nitrate, Sodium Nitrate - double dose and Increased exercise intensity in people with Becker 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 1 inclusion and 7 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 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. 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 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?
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?
Skeletal muscle blood flow (time frame: 24 hours after initial visit). 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 NCT02147639, 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 14 Jan 2020.