Found 107 Muscular Dystrophy trials
A listing of Muscular Dystrophy medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
A Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy. For those with : a. Stage 1 or Stage 2 according to the Familial Amyloid Polyneuropathy (FAP) or Coutinho Stage b. Documented genetic mutation in the TTR gene …
A phase 3 multicenter randomized double-blind placebo-controlled study to assess the efficacy and safety of 400 mg twice a day oral ladarixin in patients with new-onset type 1 diabetes and a low residual -cell function
The objective of this clinical trial is to assess whether ladarixin treatment is effective in preserving -cell function and delaying the progression of T1D in adolescent and adult patients. The safety of ladarixin in the specific clinical setting will be also evaluated.
ALTUS: Performance of a Multi- Target Hepatocellular Carcinoma (HCC) Test in Subjects With Increased Risk
For subjects age 18 years and older who are at an increased risk for HCC and for whom HCC surveillance is appropriate, including subjects with cirrhosis or non-cirrhotic subjects with Hepatitis B. Approximately 3,000 subjects are expected to be enrolled, comprised of approximately 2,222 ultrasound surveillance subjects and 778 subjects …
The Penn Medicine BioBank
The Penn Medicine Biobank is collecting and storing biological specimens such as blood, as well as health information from volunteers. These samples and this information allow researchers to use them for research studies including but not limited to COVID-19, cancer, diabetes, and heart disease.
Physical Activity and Fibrosing Interstitial Lung Disease (AP-PID)
Fibrotic interstitial lungs diseases (ILD) are a group of chronic and rare diseases characterized by an altered blood oxygenation in the lung, leading to dyspnea and physical limitation. Physical training is efficient to fight vicious circle of physical deconditioning observed in ILD. Eccentric cycling is an interesting modality of endurance …
Study Evaluating the Efficacy of Synergo Radiofrequency-Induced Thermochemotherapy Effect (RITE) with Mitomycin C (Synergo RITE + MMC) in CIS Non-Muscle Invasive Bladder Cancer (NMIBC)
The objectives of the study are to determine the efficacy and safety of Synergo® RITE and mitomycin C (MMC) in the treatment of carcinoma in situ (CIS) non-muscle invasive bladder cancer in adult patients who didn't respond to 2 courses of treatment with Bacillus Calmette-Guérin (BCG) installations. The Synergo® system …
ALFAPUMP SYSTEM IN THE TREATMENT OF REFRACTORY OR RECURRENT ASCITES: A MULTICENTER SINGLE ARM WITHIN SUBJECT CROSSOVER DESIGN PIVOTAL STUDY
The POSEIDON study is a multi-center, prospective, single arm, crossover pivotal study enrolling up to 60 pivotal cohort patients and up to 30 additional Roll-in patients with refractory or recurrent ascites at up to 15 sites. Pivotal cohort patients will undergo a 3-month pre-implant observation phase after enrollment, followed by …
A Phase 3 Multicenter Randomized Double- Blind Placebo- Controlled Study to Evaluate the Efficacy and Safety of AL001 in Individuals at Risk for or With Frontotemporal Dementia Due to Heterozygous Mutations in the Progranulin Gene
The research study is being conducted to test the safety and tolerability (i.e., possible side effects) of an investigational drug called AL001 and to find out what effects AL001 has on the body. This is for people who have a confirmed progranulin gene mutation that causes frontotemporal dementia (FTD). Study …
Evaluat the Efficacy and Safety of Study Drug Mitapivat in Subjects With Sickle Cell Disease
The purpose of this study is to determine the safety and efficacy of mitapivat in patients with sickle cell disease. Patients will be randomly assigned (like flipping a coin) in a 1:1:1 ratio to receive 50mg mitapivat, 100mg mitapivat or matched placebo for twice-daily oral administration. This means that you …
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