Found 105 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.
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 …
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 …
In order to prepare for clinical trials for CMT, there is ongoing work to develop more sensitive outcome measures, and there is a need for complementary biomarkers, particularly target engagement measures that can be used in clinical trials for candidate therapies. Much of this work has been focused on CMT1A, …
An Expanded Access Program for Efgartigimod Treatment in Patients With Generalized Myasthenia Gravis
This study provides patients with generalized myasthenia gravis (gMG), who are ineligible to participate in a clinical trial, access to efgartigimod treatment before regulatory approval. This expanded access protocol applies to patients with gMG who are not enrolled in an ongoing clinical trial. Enrolled patients will receive cycles of efgartigimod …
The purpose of this research study is to compare two versions of the same medication buprenorphine, (commonly known as Suboxone) used to treat opioid use disorder, in patients in the Emergency Department (ED). One form of the medication is a slow-release form of buprenorphine (CAM2038) that lasts for about 7 …
Patients with post-traumatic stress disorder (PTSD) will be invited to participate in this research study. Participants will be assigned to take one of the following: brexpiprazole, sertraline (also known as “Zoloft”; a marketed antidepressant that has been approved by the United States FDA for the treatment of PTSD), placebo (a …
To evaluate the sensitivity, specificity, positive predictive value (PPV) and negative predictive value (NPV) of VGT-309 to detect cancer utilizing near-infrared (NIR) fluorescence and histopathology results. 1. Sensitivity: defined as the probability that the tissue fluoresces when it is cancer, as confirmed by histology (TP/(TP+FN)) 2. Specificity: defined as the …
The primary objective of the study is to assess the effect of preventing preservation-related graft injury with the use of the metra® device in enrolled subjects. The single-arm continued access phase will be used to collect additional effectiveness and safety data to support the objectives and outcomes of the original …
1) To evaluate the effect of paltusotine versus placebo on IGF-1(Insulin-like growth factor) response 2) To evaluate the effect of paltusotine versus placebo on IGF-1 level 3) To evaluate the effect of paltusotine versus placebo on GH (growth hormone) response 4) To evaluate the effect of paltusotine versus placebo on …
PURPOSE: Phase 2 study to evaluate effect of daxdilimab vs placebo to reduce DLE activity in adults 18-75 yo with DLE. 60 wks 99 pts multiple sites 3 trx grps: placebo; daxdilimab 150 mg; daxdilimab 300 mg. Randomize 1:1:1 sc injections q 4 wks Screening--4 wks; Treatment--48 wks; Follow-Up--8 wks. …
