Becker Muscular DystrophyPhase 1NCT02434627
Sodium Nitrate for Muscular Dystrophy
A Phase 1 study testing Sodium Nitrate in people with Becker Muscular Dystrophy, run by Cedars-Sinai Medical Center at 1 site in United States.
Trial summary
The investigators' previous work in males with Becker Muscular Dystrophy shows that functional sympatholysis is restored by acute inorganic nitrate supplementation. This was translated from work using the mdx mouse model of dystrophinopathy. 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
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2015to Apr 2018
- 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 45 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Clinical diagnosis of muscular dystrophy
- Age 15-45 years of age
- Ambulatory
- No clinical evidence of heart failure
- Maximum voluntary contraction, measured by hand grip dynamometer, of 20-40 kg
Who cannot join
- Hypertension, diabetes, or heart failure by standard clinical criteria
- Elevated B-type Natruiretic Peptide level (>100 pg/ml)
- Left Ventricular Ejection Fraction < 50%
- Wheelchair bound
- Cardiac rhythm disorder, specifically: rhythm other than sinus, Supraventricular Tachycardia, atrial fibrillation, ventricular tachycardia
- Continuous ventilatory support
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- Liver disease
- Renal impairment
- Contraindications to NO donors (use of nitrates, alpha-blockers, CYP3A inhibitors, amlodipine, or Phosphodiesterase type 5 (PDE5) inhibitors) Glucocorticoid therapy and prophylactic use of Angiotensin Converting Enzyme (ACE) inhibitors and beta-blockers for cardiac protection will not be exclusion criteria.
- Maximum voluntary contraction of less than 20 kg or greater than 40 kg
- Ventilation Devices (Continuous Positive Airway Pressure (CPAP), Bilevel Positive Airway Pressure (BiPAP))
Trial locations and contacts
1 site in 1 country.
United States1 site
Cedars-Sinai Medical Center
Los Angeles, California, 90048
About this study
The investigators' previous work in males with Becker Muscular Dystrophy shows that functional sympatholysis is restored by acute inorganic nitrate supplementation. This was translated from work using the mdx mouse model of dystrophinopathy. Recent work has shown that there is a frank improvement in grip strength when mdx mice are treated with an inorganic Nitric Oxide (NO) donor. The purpose of this study is to determine if chronic treatment with an inorganic NO donor can benefit patients with muscular dystrophy beyond blood flow regulation.
- Conditions
- Becker Muscular Dystrophy
- Being tested
- Sodium Nitrate
- Keywords
- Becker, Dystrophy
- 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 NitrateDrug
Patients will be given sodium nitrate daily in the form of beetroot juice.
| Study group | Role | What they receive |
|---|---|---|
| Sodium Nitrate (Beetroot Juice) | Experimental | Sodium nitrate in the form of beetroot juice will be administered orally. Patients will be assessed with a number of functional muscle assessments. Receives: Drug: Sodium Nitrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in maximal handgrip strength
Measured: Change from baseline in handgrip strength at 3 months
Secondary outcomes (5)
Change in muscle function - Performance of Upper Limb Scale
Change in functional muscle assessment as measured by the Performance of Upper Limb Scale
Measured: Change from baseline in muscle function - the Performance of Upper Limb Scale at 3 months
Change in muscle tissue markers - histology and proteomics
Change in tissue markers such as neuronal Nitric Oxide Synthase (nNOS) content and location and nitrosative stress by histology and proteomics
Measured: Change from baseline in muscle tissue markers at 3 monthss
Change in systolic wall strain - imaging
Change in the cardiac wall strain as measured by Cardiac Magnetic Resonance Imaging
Measured: Change from baseline in cardiac systolic wall strain at 3 months
Change in muscle function - North Star Ambulatory Assessment
Change in functional muscle assessment as measured by the North Star Ambulatory Assessment
Measured: Change from baseline in muscle function - North Star Ambulatory Assessment at 3 months
Change in muscle function - 6 minute walk test
Change in functional muscle assessment as measured by the 6 minute walk test
Measured: Change from baseline in muscle function - 6 minute walk test at 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 May 2015 (estimated)
- Study startJun 2015
- Primary completionApr 2018
- Study completionApr 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 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
- NCT02434627
- Sponsor's protocol ID
- Pro35228
- Status last verified
- Oct 2016
- First submitted
- 28 Apr 2015
- Last updated
- 13 Jan 2020
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sodium Nitrate for Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02434627
- Acronym / sponsor ID
- Pro35228
- Conditions
- Becker Muscular Dystrophy
- Keywords
- Becker, Dystrophy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Cedars-Sinai Medical CenterUniversity, hospital or other
- Study start
- Jun 2015
- Primary completion
- Apr 2018 (actual)
- Study completion
- Apr 2018 (actual)
- First posted
- 5 May 2015 (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.
The investigators' previous work in males with Becker Muscular Dystrophy shows that functional sympatholysis is restored by acute inorganic nitrate supplementation. This was translated from work using the mdx mouse model of dystrophinopathy. Recent work has shown that there is a frank improvement in grip strength when mdx mice are treated with an inorganic Nitric Oxide (NO) donor. The purpose of this study is to determine if chronic treatment with an inorganic NO donor can benefit patients with muscular dystrophy beyond blood flow regulation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sodium Nitrate (Beetroot Juice) | Experimental | Drug: Sodium NitrateSodium nitrate in the form of beetroot juice will be administered orally. Patients will be assessed with a number of functional muscle assessments. |
Who may qualify
- Ages
- 15 to 45 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Clinical diagnosis of muscular dystrophy
- Age 15-45 years of age
- Ambulatory
- No clinical evidence of heart failure
- Maximum voluntary contraction, measured by hand grip dynamometer, of 20-40 kg
Key exclusion criteria (11 listed)
- Hypertension, diabetes, or heart failure by standard clinical criteria
- Elevated B-type Natruiretic Peptide level (>100 pg/ml)
- Left Ventricular Ejection Fraction < 50%
- Wheelchair bound
- Cardiac rhythm disorder, specifically: rhythm other than sinus, Supraventricular Tachycardia, atrial fibrillation, ventricular tachycardia
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 NitratePatients will be given sodium nitrate daily in the form of beetroot juice. | Drug | Sodium Nitrate (Beetroot Juice) |
Primary outcome
Change in maximal handgrip strength
Time frame: Change from baseline in handgrip strength at 3 months
5 secondary outcomes
Change in muscle function - Performance of Upper Limb Scale
Time frame: Change from baseline in muscle function - the Performance of Upper Limb Scale at 3 months
Change in muscle tissue markers - histology and proteomics
Time frame: Change from baseline in muscle tissue markers at 3 monthss
Change in systolic wall strain - imaging
Time frame: Change from baseline in cardiac systolic wall strain at 3 months
Change in muscle function - North Star Ambulatory Assessment
Time frame: Change from baseline in muscle function - North Star Ambulatory Assessment at 3 months
Change in muscle function - 6 minute walk test
Time frame: Change from baseline in muscle function - 6 minute walk test at 3 months
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 13 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 Phase 1 study testing Sodium Nitrate 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 5 inclusion and 11 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 13 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?
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 maximal handgrip strength (time frame: Change from baseline in handgrip strength at 3 months). 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 13 Jan 2020.