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Muscular Dystrophies clinical trials

View clinical trials related to Muscular Dystrophies.

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NCT ID: NCT06295718 Enrolling by invitation - Clinical trials for Duchenne Muscular Dystrophy

Tele-assessment of Functional Performance and Quality of Life in Patients With Duchenne Muscular Dystrophy: Validity and Reliability Study

Start date: July 1, 2023
Phase:
Study type: Observational

Thanks to tele-assessment methods, it may be possible to evaluate DMD patients without traveling to clinical centers. In recent years, the applicability of remote assessment methods in DMD patients, as in many populations, is being investigated. However, studies have generally focused on a single evaluation parameter such as physical function, a special evaluation method or a special evaluation tool. The aim of this study is to investigate whether remote assessment of functional performance and quality of life in DMD patients is valid and reliable. If a valid and reliable tele-evaluation method that includes functional performance and quality of life parameters is found to be valid and reliable, the travel burden on patients and caregivers can be eased, patients' stress and anxiety related to travel can be reduced, caregivers can save time and energy and provide patients with the best possible treatment.

NCT ID: NCT06244082 Enrolling by invitation - Clinical trials for Duchenne Muscular Dystrophy

Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping

EXPLORE44OLE
Start date: January 22, 2024
Phase: Phase 2
Study type: Interventional

AOC 1044-CS2 (EXPLORE44-OLE) is an Open-label Study to Evaluate the Pharmacodynamics and Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants with Mutations Amenable to Exon 44 Skipping.

NCT ID: NCT06066580 Enrolling by invitation - Clinical trials for Becker Muscular Dystrophy

Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy

MESA
Start date: November 2, 2023
Phase: Phase 2
Study type: Interventional

EDG-5506-203 MESA is an open-label extension study to assess the long-term effect of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy

NCT ID: NCT05967351 Enrolling by invitation - Clinical trials for Duchenne Muscular Dystrophy

A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study

EXPEDITION
Start date: September 27, 2023
Phase: Phase 3
Study type: Interventional

The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study. No study drug will be administered as part of this study. Pre-infusion baseline will be defined as the timepoint just prior to infusion of delandistrogene moxeparvovec from a previous clinical study. Each participant will be followed for a minimum of 5 years post-infusion of delandistrogene moxeparvovec from a previous clinical study. The duration of participation in this study is dependent on the length of follow-up the participant completed post-infusion of delandistrogene moxeparvovec from a previous clinical study.

NCT ID: NCT05956132 Enrolling by invitation - Clinical trials for Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A

Clinical and Biochemical Features for the Identification of Dominant Calpainopathies

DOM-CAL
Start date: September 1, 2023
Phase:
Study type: Observational

Mutations in the CAPN3 gene cause muscular dystrophies with dysfunction in calpain-3. Calpainopathies are usually inherited in an autosomal recessive manner but in some families they can occur in a dominant inheritance. The significance of heterozygous variants is difficult to interpret in the absence of family history. In this study, the investigators will review the clinical and laboratory information in a cohort of patients identified in the participating centers, with the aim of improving the diagnostic strategy of dominant calpainopathies.

NCT ID: NCT04626674 Enrolling by invitation - Clinical trials for Muscular Dystrophy, Duchenne

A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)

ENDEAVOR
Start date: November 23, 2020
Phase: Phase 1
Study type: Interventional

This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with DMD. The maximum participant duration for this study is 156 weeks.

NCT ID: NCT04009408 Enrolling by invitation - Clinical trials for Muscular Dystrophies

Expiratory Muscle Strength Training (EMST) in Neuromuscular Disorders

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

The purpose of this study is to investigate the impact of expiratory muscle strength training (EMST) on the swallowing, breathing, oral intake, quality of life and cough function of people with oculopharyngeal muscular dystrophy (OPMD).

NCT ID: NCT03836300 Enrolling by invitation - Clinical trials for Duchenne Muscular Dystrophy

Parent and Infant Inter(X)Action Intervention (PIXI)

Start date: November 30, 2018
Phase: N/A
Study type: Interventional

The objective is to develop and test, through an iterative process, an intervention to address and support the development of infants with a confirmed diagnosis of a neurogenetic disorder with associated developmental delays or intellectual and developmental disabilities. The proposed project will capitalize and expand upon existing empirically based interventions designed to improve outcomes for infants with suspected developmental delays. Participants will be infants with a confirmed diagnosis of a neurogenetic disorder (e.g., fragile X, Angelman, Prader-Willi, Dup15q, Phelan-McDermid, Rhett, Smith Magenis, Williams, Turner, Kleinfelter, Down syndromes, Duchenne muscular dystrophy) within the first year of life and their parents/caregivers. The intervention, called the Parent and Infant Inter(X)action Intervention (PIXI) is a comprehensive program inclusive of parent education about early infant development and the neurogenetic disorder for which they were diagnosed, direct parent coaching around parent-child interaction, and family/parent well-being support. The protocol includes repeated comprehensive assessments of family and child functioning, along with an examination of feasibility and acceptability of the program.

NCT ID: NCT03655223 Enrolling by invitation - Diabetes Mellitus Clinical Trials

Early Check: Expanded Screening in Newborns

Start date: October 15, 2018
Phase:
Study type: Observational

Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and families who screen positive, and 3) evaluate the Early Check program. The Early Check screening will lead to earlier identification of newborns with rare health conditions in addition to providing important data on the implementation of this model program. Early diagnosis may result in health and development benefits for the newborns. Infants who have newborn screening in North Carolina will be eligible to participate, equating to over 120,000 eligible infants a year. Over 95% of participants are expected to screen negative. Newborns who screen positive and their parents are invited to additional research activities and services. Parents can enroll eligible newborns on the Early Check electronic Research Portal. Screening tests are conducted on residual blood from existing newborn screening dried blood spots. Confirmatory testing is provided free-of-charge for infants who screen positive, and carrier testing is provided to mothers of infants with fragile X. Affected newborns have a physical and developmental evaluation. Their parents have genetic counseling and are invited to participate in surveys and interviews. Ongoing evaluation of the program includes additional parent interviews.

NCT ID: NCT03373968 Enrolling by invitation - Clinical trials for Duchenne Muscular Dystrophy

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study

Start date: October 24, 2017
Phase: Phase 2/Phase 3
Study type: Interventional

This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.