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Becker Muscular Dystrophy clinical trials

View clinical trials related to Becker Muscular Dystrophy.

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NCT ID: NCT03057002 Recruiting - Clinical trials for Duchenne Muscular Dystrophy

UTSW HP [13-C] Pyruvate Injection in HCM

HPHCM
Start date: May 1, 2018
Phase:
Study type: Observational

The study objective is to identify the earliest changes in energy substrate metabolism in patients with cardiomyopathies (CMP). To achieve this objective, we plan first to test the hypothesis that patients with CMP present focal alterations in myocardial hyperpolarized [1-13C]pyruvate flux.

NCT ID: NCT02972580 Recruiting - Clinical trials for Duchenne Muscular Dystrophy

Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD

Start date: June 2016
Phase:
Study type: Observational

Longitudinal prospective observational study. This is a 24-month study with the possibility of extending the data time points. Initially baseline, then 12 and 24 months follow up studies will be completed.

NCT ID: NCT02847975 Completed - Clinical trials for Becker Muscular Dystrophy

Sodium Nitrate to Improve Blood Flow

Start date: October 2013
Phase: Phase 1
Study type: Interventional

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.

NCT ID: NCT02434627 Completed - Clinical trials for Becker Muscular Dystrophy

Sodium Nitrate for Muscular Dystrophy

Start date: June 2015
Phase: Phase 1
Study type: Interventional

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.

NCT ID: NCT02207283 Completed - Clinical trials for Becker Muscular Dystrophy

PDE5 Inhibition to Alleviate Functional Muscle Ischemia in Becker Muscular Dystrophy

Start date: March 2012
Phase: Phase 4
Study type: Interventional

This study is intended to build on recent findings from our laboratory showing that a single dose of tadalafil (also known as Cialis) restored normal blood flow regulation in men with Becker muscular dystrophy. The investigators now wish to extend these findings by showing that the same dose of tadalafil will also prevent muscle injury and fatigue. Patients will take a single dose of tadalafil prior to exercising. Then doctors will measure whether the patients muscles receive more blood flow and are better protected during exercise.

NCT ID: NCT02165358 Completed - Clinical trials for Becker Muscular Dystrophy

Muscle MRI in Becker Muscular Dystrophy and in Limb-girdle Muscular Dystrophy Type 2I

Start date: June 2014
Phase: N/A
Study type: Observational

The purpose of this study is to investigate the paradoxical muscle enlargement in the calves and tongue seen in patients affected by Becker muscular dystrophy and Limb-girdle muscular dystrophy type 2I. The enlarged calves' muscle quality will be assessed primarily on the basis of the muscle structure on MRI and based on a calculation of muscle strength per cross-sectional area.The findings will be compared with results from non-affected controls. Additionally we want to describe the tongue muscle appearance on T1-weighted MRI.

NCT ID: NCT02147639 Completed - Clinical trials for Becker Muscular Dystrophy

Effects of Sodium Nitrate on Blood Flow in Becker Muscular Dystrophy

Start date: October 2013
Phase: Phase 2/Phase 3
Study type: Interventional

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).

NCT ID: NCT02109692 Recruiting - Clinical trials for Duchenne Muscular Dystrophy

Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies

biodystromirs
Start date: May 19, 2014
Phase: N/A
Study type: Interventional

Duchenne muscular dystrophy (DMD) , caused by mutations in the DMD gene, is the most common and most severe progressive dystrophy of the child. Although the development is rapidly progressive , there is variability in the severity of the disease between DMD patients that do not correlate with the type of mutations in the DMD gene. There are no easily measurable biomarkers for monitoring the DMD or moderate form of the disease, Becker muscular dystrophy (BMD ) . MicroRNAs (miRNAs) are involved in most cellular processes , and their expression pattern is a signature of the state of a cell . They represent a potential class of diagnostic and prognostic biomarkers. Some are specific for the skeletal myogenesis , and changes in their pattern of expression are associated with muscle diseases including muscular dystrophy. The levels of muscle- specific miRNAs are indeed greatly increased in the serum of DMD and BMD compared to control patients . The main objective of this is to validate the use of serum muscle-derived microRNAs as biomarkers of DMD patients (compared with healthy subjects). Secondary objectives are i) to investigate the relationship between circulating levels of these miRNAs and the severity of the dystrophinopathy (DMD vs BMD) and also the progression of the disease (longitudinal study), ii) to assess the specificity of these markers for dystrophinopathy (comparison with other patients with muscular dystrophy), iii) to test candidate miRNAs recently identified but not yet analyzed in the serum of patients. Clinical data and samples will be recorded at each regular consultation. miRNA levels will be quantified using Real Time Quantitative RT-PCR.

NCT ID: NCT02069756 Recruiting - Clinical trials for Duchenne Muscular Dystrophy

The Duchenne Registry

Start date: October 2007
Phase:
Study type: Observational [Patient Registry]

The Duchenne Registry is an online, patient-report registry for individuals with Duchenne and Becker muscular dystrophy and carrier females. The purpose of the Registry is to connect Duchenne and Becker patients with actively recruiting clinical trials and research studies, and to educate patients and families about Duchenne and Becker care and research. At the same time, The Duchenne Registry is a valuable resource for clinicians and researchers in academia and industry, allowing access to de-identified datasets provided by patients and their families-information that is vital to advances in the care and treatment of Duchenne. The Duchenne Registry is a member of the TREAT-NMD Neuromuscular Network.

NCT ID: NCT01856868 Completed - Clinical trials for Becker Muscular Dystrophy

Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)

Start date: May 2013
Phase: Phase 1/Phase 2
Study type: Interventional

(-)-Epicatechin will be evaluated for the treatment of progressive muscle loss and impaired skeletal muscle function in Becker Muscular Dystrophy (BMD) patients.