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Becker Muscular Dystrophy clinical trials

View clinical trials related to Becker Muscular Dystrophy.

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NCT ID: NCT06378203 Recruiting - Clinical trials for Muscular Dystrophies

Rehabilitation in Muscular Dystrophies From the Hospital Facility to the Home: Pilot Project [RIMUDI]

RIMUDI
Start date: December 15, 2023
Phase: N/A
Study type: Interventional

Until twenty years ago physical exercise in muscular dystrophies was considered harmful to the muscle cells, inducing an acceleration of cell necrosis. In fact, it is now certain and validated that an active lifestyle and the practice of controlled and regular physical activity are to be considered therapeutic in neuromuscular pathologies with the aim of optimizing muscular and cardio-respiratory function and preventing atrophy In particular, it seems that the optimal care is extensive and can be carried out in a safe and controlled manner even at home. It is well documented that exercise has beneficial effects on muscle with increased strength and muscular endurance.

NCT ID: NCT05409079 Recruiting - Cerebral Palsy Clinical Trials

Schulze Muscular Dystrophy Ability Clinical Study

Start date: May 26, 2022
Phase: N/A
Study type: Interventional

The primary objective of the Schulze study is to evaluate the function of the upper limbs of subjects diagnosed with neuromuscular disorders, with and without use of the Abilitech Assist device in the clinic and home environments. Functional outcomes will include documenting active range of motion and the ability to perform activities of daily living (ADLs) using the standardized Canadian Occupational Performance Measure (COPM) and the Role Evaluation of Activities of Life (REAL) assessments. Secondary objectives are to assess the safety record and report on adverse events (AEs) and parameters related to device usage, including device usage time and the time required to don/doff the device. Secondary objectives also include characterization of user upper limb performance based on etiology.

NCT ID: NCT05291091 Recruiting - Clinical trials for Becker Muscular Dystrophy

Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)

Start date: July 6, 2022
Phase: Phase 2
Study type: Interventional

A study of sevasemten (EDG-5506) in Becker muscular dystrophy (known as CANYON) and pivotal cohort (known as GRAND CANYON). The EDG-5506-201 CANYON study was expanded to include an additional 120 adult participants in a cohort called GRAND CANYON, that is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of sevasemten in adults with Becker. CANYON is fully enrolled; GRAND CANYON is currently enrolling.

NCT ID: NCT05257473 Recruiting - Clinical trials for Muscular Dystrophies

Defining Endpoints in Becker Muscular Dystrophy

GRASP-01-002
Start date: April 13, 2022
Phase:
Study type: Observational

This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)

NCT ID: NCT05166109 Recruiting - Clinical trials for Becker Muscular Dystrophy

A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD)

Start date: July 7, 2022
Phase: Phase 2
Study type: Interventional

This Phase II pilot study is a randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, PD, and exploratory clinical efficacy of vamorolone 500mg (250mg for body weight <50 kg) daily administered orally compared to placebo over a treatment period of 24 weeks in males with BMD. Funding Source - FDA OOPD

NCT ID: NCT05019625 Recruiting - Clinical trials for Duchenne Muscular Dystrophy

Biomarker Development for Muscular Dystrophies

Start date: February 20, 2015
Phase:
Study type: Observational

Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. The investigators are interested in finding less invasive methods that reduce the need for muscle biopsies. The purpose of this research is to learn about the possibility of detecting and measuring the activity and severity of muscular dystrophies by examining a urine sample and a blood sample, and some muscles in the arms and legs using tests called ultrasound and electrical impedance myography; both tests are painless and non-invasive. The information that is gathered from this study may help to evaluate, prevent, diagnose, treat, and improve the understanding of human muscle diseases.

NCT ID: NCT04972604 Recruiting - Clinical trials for Duchenne Muscular Dystrophy

CureDuchenne Link®: A Resource for Research

CDLink
Start date: July 9, 2021
Phase:
Study type: Observational [Patient Registry]

CureDuchenne link is a data hub comprised of integrated biospecimens, clinical data, and self- and/or caregiver-reported information from participants. Anyone over 4 weeks old who has been diagnosed with DMD or BMD or who is a carrier of DMD or BMD can join. Parents or legal guardians can sign up their child(ren).

NCT ID: NCT04668716 Recruiting - Clinical trials for Duchenne Muscular Dystrophy

Brain Involvement in Dystrophinopathies Part 2

Start date: October 11, 2021
Phase:
Study type: Observational

The objective of this study is to understand the relationship between DMD and BMD brain comorbidities, and the location of the gene mutation which causes the disease.

NCT ID: NCT04583917 Recruiting - Clinical trials for Duchenne Muscular Dystrophy

Brain Involvement in Dystrophinopathies Part 1

Start date: March 30, 2021
Phase:
Study type: Observational

The objective of this study is to collect data from a large cohort of individuals with DMD and BMD focusing on the neurobehavioural aspects of these conditions and their correlation to the location of the DMD gene mutation.

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.