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Muscular Dystrophies clinical trials

View clinical trials related to Muscular Dystrophies.

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NCT ID: NCT06100887 Recruiting - Clinical trials for Duchenne Muscular Dystrophy

Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy (FOX)

Start date: March 22, 2024
Phase: Phase 2
Study type: Interventional

The FOX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics, and biomarkers in children and adolescents with Duchenne muscular dystrophy previously treated with gene therapy including a randomized, double-blind, placebo-controlled Part A, followed by an open-label part B.

NCT ID: NCT06094205 Recruiting - Clinical trials for Spinal Cord Injuries

Feasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia (BG-Speech-02)

BG-Speech-02
Start date: October 2024
Phase: N/A
Study type: Interventional

The goal of this study is to improve our understanding of speech production, and to translate this into medical devices called intracortical brain-computer interfaces (iBCIs) that will enable people who have lost the ability to speak fluently to communicate via a computer just by trying to speak.

NCT ID: NCT06093100 Recruiting - Clinical trials for Duchenne Muscular Dystrophy

Wearable Technology to Evaluate Hyperglycemia and HRV in DMD - Longitudinal Aim

Start date: August 2024
Phase:
Study type: Observational

Duchenne Muscular Dystrophy (DMD) is an X-linked disorder that causes muscle wasting, cardiopulmonary failure, and premature death. Heart failure is a leading cause of death in DMD, but substantial knowledge gaps exist regarding predisposing risk factors. In the general population, hyperglycemia, insulin resistance, and decreased heart rate variability (HRV; reflecting autonomic dysfunction) are associated with cardiomyopathy (CM). It is unclear whether these factors are associated with DMD-CM. Closing this knowledge gap may lead to novel screening and therapeutic strategies to delay progression of DMD related CM. Despite risk factors for hyperglycemia, including the use of glucocorticoids, low muscle mass, obesity, and reduced ambulation, little is known regarding glucose abnormalities in DMD. Some of these same risk factors, along with the distance needed to travel for specialty care, present significant barriers to research participation and clinical care for individuals with DMD. Remote wearable technology may improve research participation in this vulnerable population. Therefore, this study will leverage remote wearable technologies to overcome these barriers and define the relationship between dysglycemia and DMD-CM. In this Aim of the study, the investigators will assess the utility of remote wearable technology to predict changes in traditional metrics of metabolism and cardiac function. In this pilot study, 10 individuals with DMD will undergo cardiac magnetic resonance imaging (CMR) and oral glucose tolerance tests (OGTTs) at baseline and two years. The investigators will remotely assess glycemia (using continuous glucose monitors), HRV (using extended Holter monitors), and activity (using accelerometers) every 6 months over the 2 years and evaluate if changes in wearable metrics predict changes in CMR and OGTT.

NCT ID: NCT06079736 Recruiting - Clinical trials for Duchenne Muscular Dystrophy

A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment

CONNECT1-EDO51
Start date: January 8, 2024
Phase: Phase 2
Study type: Interventional

The primary purpose of the MAD period is to evaluate the safety and tolerability of multiple ascending intravenous (IV) doses of PGN-EDO51 administered to participants with Duchenne muscular dystrophy (DMD). The primary purpose of the LTE period is to evaluate the long-term safety and tolerability of PGN-EDO51 in participants who have completed the MAD period. The study consists of 3 periods: A Screening Period (up to 45 days), a Treatment and Observation Period (16 weeks), and an Extension Period (108 weeks).

NCT ID: NCT06079567 Recruiting - Clinical trials for Facioscapulohumeral Muscular Dystrophy Type 2

An 18-month Prospective Natural History Study to Gain Insight Into FSHD2 Pathophysiology and Disease Progression

INSIGHT FSHD2
Start date: October 3, 2023
Phase: N/A
Study type: Interventional

Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common inherited myopathies in adults. It is associated with genetic and epigenetic deregulation of the D4Z4 locus on the sub-telomeric region of chromosome 4q35, resulting in abnormal expression of DUX4p. Type 1 FSHD (FSHD1) is the most common form of the disease and accounts for 95% of cases, while Type 2 FSHD (FSHD2) accounts for only 5% of all FSHD cases. FSHD1 and FSHD2 are closely related in terms of genetic and epigenetic foundations, pathophysiology and clinical manifestations. Although initially described as distinct entities based on their genetics, recent information suggests that both forms of myopathy may represent the opposite ends of a spectrum of molecular diseases in which alteration of the genetic and epigenetic factors that govern DUX4 suppression in skeletal muscle have a different impact in both forms of the disease. FSHD1 and FSHD2 are both associated with re-expression of DUX4 leading to muscle atrophy, but the genetics underlying this re-expression are different, depending on whether it is type 1 or type 2. For FSHD1, it is associated with a critical contraction of the D4Z4 region and the 4qA permissive allele, leading to the expression of DUX4. In contrast, FSHD2 is caused by the inheritance of two independent genetic variations. A heterozygous mutation, mainly located on the SMCHD1 (Structural Maintenance of Chromosome flexible Hinge Domain containing 1) gene, results in a loss of function of chromatin D4Z4 repressor. This mutation, combined with the 4qA allele bearing the DU4 polyadenylation site, makes this allele permissive for the expression of the DUX4 topical gene. Therefore, because the two forms of FSHD are genetically distinct and very few patients have FSHD2, our knowledge of the impact of chromatin D4Z4 repressors, such as SMCHD1, or the progression and severity of the disease remains very limited. It is important to note that a lack of reliable biomarkers specific to the severity and progression of the disease may prevent the development of therapies to treat patients with FSHD2. This study will allow us to better understand the natural progression of FSHD2 over time, to assess the responsiveness of clinical outcome measures (COMs) and to identify and validate inflammatory serum biomarkers predicting the severity and progression of the disease.

NCT ID: NCT06054971 Recruiting - Clinical trials for Duchenne Muscular Dystrophy

NSAA NON-Interventional Study Protocol

Start date: October 2, 2023
Phase:
Study type: Observational

The purpose of this non-interventional study is to evaluate the feasibility of remotely administering the North Star Ambulatory Assessment (NSAA) to participants with Duchenne muscular dystrophy (DMD). The iTakeControl (iTC) software platform will be utilized to remotely administer and score the NSAAs.

NCT ID: NCT05996003 Recruiting - Clinical trials for Duchenne Muscular Dystrophy

NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)

Start date: February 22, 2024
Phase: Phase 2
Study type: Interventional

This is a Phase 2, open-label, multi-center, 2-part study of NS-089/NCNP-02 administered by weekly IV infusion to ambulant boys aged ≥4 to <15 years with DMD due to mutations amenable to exon 44 skipping. Participants will receive a selected dose of NS-089/NCNP-02 administered once weekly. The study consists of 2 parts: Part 1 and Part 2. Six participants (Cohort 1) will participate in both Part 1 and Part 2, and 14 participants (Cohort 2) will be added for Part 2.

NCT ID: NCT05989620 Recruiting - LGMD2I Clinical Trials

Long-Term Development of Muscular Dystrophy Outcome Assessments

GRASP-01-005
Start date: October 18, 2023
Phase:
Study type: Observational

This is a 24-month, observational study of up to 1000 participants with Limb Girdle Muscular Dystrophy (LGMD), Myotonic Dystrophy Type 2 (DM2), and late onset Pompe disease (LOPD).

NCT ID: NCT05982119 Recruiting - Clinical trials for Duchenne Muscular Dystrophy

Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study

Start date: July 10, 2020
Phase: N/A
Study type: Interventional

The objective of the ActiLiège Next study is to collect longitudinal data from patients and control subjects using a wearable magneto-inertial device. By collecting natural history data in various neuromuscular disorders (Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy 1, Charcot-Marie-Tooth, Centronuclear Myopathy, Congenital Muscular Dystrophy), we aim to validate digital outcome measures to continuously assess motor function in real-life.

NCT ID: NCT05967793 Recruiting - Clinical trials for Duchenne Muscular Dystrophy

Effect of Kinesiology Taping on Head and Trunk Control in Children With Duchenne Muscular Dystrophy

Start date: July 2023
Phase: N/A
Study type: Interventional

Muscular dystrophy (MD) is a group of muscle diseases that results in increasing weakening and breakdown of skeletal muscles over time. The disorders differ in which muscles are primarily affected, the degree of weakness, how fast they worsen, and when symptoms begin. Many people will eventually become unable to walk. Some types are also associated with problems in other organs. The muscular dystrophy group contains thirty different genetic disorders that are usually classified into nine main categories or types. The signs and symptoms consistent with muscular dystrophy are: progressive muscular wasting, poor balance, scoliosis (curvature of the spine and the back), progressive inability to walk, waddling gait, Calf deformation, Limited range of movement, respiratory difficulty, cardiomyopathy and muscle spasms This study aimed to assess the efficacy of Kinesiology Taping on head and trunk control in patients with Duchenne muscular dystrophy