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DMD clinical trials

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NCT ID: NCT06290713 Not yet recruiting - Exercise Clinical Trials

Vasodilator and Exercise Study for DMD (VASO-REx)

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

Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal muscle and improve cardiovascular health) and tadalafil, an FDA-approved vasodilator (to optimize blood flow and muscle perfusion which is impaired and often overlooked in DMD). Target: improved muscle function, vascular health, and DMD 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: NCT05635266 Recruiting - Stroke Clinical Trials

Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives

Start date: October 26, 2021
Phase:
Study type: Observational

To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and preventative methods for specific and complex conditions.

NCT ID: NCT05102916 Recruiting - SMA Clinical Trials

Swiss Registry for Neuromuscular Disorders

Swiss-Reg-NMD
Start date: June 20, 2018
Phase:
Study type: Observational [Patient Registry]

The Swiss Patient Registry for DMD/BMD and SMA was launched in 2008 in order to give Swiss patients access to new therapies. It was founded with the financial support of several patient organizations and research foundations. Since 2008, children, adolescents and adults with DMD, BMD and SMA are registered with the help of all major muscle centers in Switzerland. After nearly ten years of activity, the Swiss Patient Registry for DMD/BMD and SMA implemented several adaptations in 2018 to meet current and future expectations of patient's organizations, health authorities and research organizations.

NCT ID: NCT04337112 Approved for marketing - Clinical trials for Muscular Dystrophy, Duchenne

The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation

Start date: n/a
Phase:
Study type: Expanded Access

This is an open label expanded access program for boys, 3 to 12 years old, for the treatment of Duchenne muscular dystrophy (DMD) with confirmed mutation(s) in the dystrophin gene that is amenable to skipping of exon 53.

NCT ID: NCT03649919 Withdrawn - DMD Clinical Trials

Multi-center Clinical Study on the Diagnosis and Treatment Management of Rare Neurological Disease in Children

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

The incidence of rare diseases is extremely low, the disease is numerous, the symptoms are serious, and the detection technology is complicated. Countries have different definitions of rare diseases. The definition of rare diseases in China is defined as: diseases with a prevalence of less than 1 in 500 000 or newborns with an incidence of less than 1/10 000 are rare diseases. Due to the low incidence of rare diseases and the accumulation of multiple organs and systems in most diseases, clinicians lack comprehensive and systematic understanding. Patients often face great difficulties in seeking medical treatment and diagnosis. Currently, there is a lack of systematic and rare diseases in China. Management, diagnosis and treatment of rare diseases, making the diagnosis of rare diseases, prevention interventions seriously lagging behind, obviously behind the management of developed countries and regions; rare diseases are mostly related to genetic variation, with the clinical application of genetic diagnosis technology, more and more Many genetically related rare diseases have been diagnosed at an early stage; at present, precision medicine is rapidly developing, and more and more rare disease clinical trials have entered the country, bringing prospects for the treatment of rare diseases. For this reason, the management of rare diseases is particularly important. At present, some rare diseases of the nervous system can be treated early; for example, immune-related rare diseases have common normative immunotherapy and functional disability prevention, and the characteristics of single disease management of each disease; hereditary degenerative rare diseases such as progressive 2-3 multi-center clinical trials of spinal muscular atrophy and progressive muscular dystrophy have been entered into our hospital (in our hospital), X-linked pre-diagnosis of adrenal malnutrition genetic diseases, and appropriate treatment time is selected. Stem cell transplantation is in research and planning; the long-term management and comprehensive treatment of nodular sclerosis and Dravet syndrome are important for the prevention and treatment of diseases; therefore, the early diagnosis, pathogenesis and standardized treatment of rare diseases of the nervous system are urgent. And necessity.

NCT ID: NCT01890616 Completed - Constipation Clinical Trials

Constipation and Gut Transit in DMD Patients

Start date: May 1, 2013
Phase:
Study type: Observational

In this research study the investigators want to screen Duchenne Muscular Dystrophy (DMD) patients for signs and symptoms of constipation in patients over 18. The investigators hypothesized: - DMD patients have a high prevalence of constipation than in the general population. - Prevalence of constipation will increase with age and with worsening functional status.