Muscular Dystrophy Clinical Trials

University of Pennsylvania Centralized Observational Research Repository on Neurodegenerative Disease

The aim of this study is to create a repository of both cross-sectional and longitudinal data, imaging and biological specimens for neurodegenerative disease research.

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Tranexamic Acid for the Prevention of Obstetrical Hemorrhage After Cesarean Delivery: A Randomized Controlled Trial

Please see attached document.

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 …