5 results
The longitudinal assessment of the BAFi pyrimethamine and of the HDACi valproic acid on the HIV reservoir size in HIV patients on antiretroviral therapy.
Primary Objective1. To evaluate the safety and tolerability of long-term dosing of losmapimod tablets in subjects with FSHD1Secondary Objectives1. To assess target engagement of losmapimod tablets in blood and skeletal muscle over long term dosing2…
Primary Objective:Part A:To evaluate the efficacy of losmapimod for the treatment of FSHD by demonstrating slowing of disease progression assessed by reachable workspace quantification of total relative surface area (RSA) Q1-Q5 with 500 g wrist…
A Phase 2 Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia
This is a phase 2 study intended to explore the potential use of IMR 687 to treat subjects with β thalassemia. This is the first study of IMR-687 in a β-thalassemia population, and, as such, is designed to examine the safety, tolerability, and PK,…
A Phase 2b Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease
* To evaluate the fetal hemoglobin (HbF) response to IMR-687 versus placebo * To evaluate the safety of IMR-687 versus placebo