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Brain INvolvement in Dystrophinopathies: BIND: Deep functional phenotyping of Duchenne Muscular Dystrophy and Becker Muscular Dystrophy patients (WP5) Part 2

The primary objective of the second part of the study, which is the focus of this application, is to perform a comparative analysis of the cognitive profile and MRI outcomes of DMD/BMD patients.

A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD)

The primary objective of this study is to evaluate the effect of single daily SC administration of elamipretide for 48 weeks on the distance walked (in meters) on the 6-minute walk test.The secondary objectives of this study are:- To evaluate the…

Active Monitoring versus an Abduction Device for treatment of Infants with Centered Dysplastic Hips, a Comprehensive Cohort Study (TReatment with Active Monitoring (TRAM)-Trial)

The aim of this study is to compare treatment with an abduction device to active monitoring of infants with centered dysplastic hips (Graf type IIa- or IIb or IIc) during the first year of life.

A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled, 48-Week, ParallelGroup Study of the Efficacy and Safety of Losmapimod in Treating Patients with Facioscapulohumeral Muscular Dystrophy (REACH)

This study has been transitioned to CTIS with ID 2024-512737-33-00 check the CTIS register for the current data. Primary Objective:Part A:To evaluate the efficacy of losmapimod for the treatment of FSHD by demonstrating slowing of disease…

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva (PROGRESS)

This study has been transitioned to CTIS with ID 2023-504129-38-00 check the CTIS register for the current data. Primary:To determine the efficacy of INCB000928 for the prevention of new HO in participants with FOP.Key Secondary:To further evaluate…

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy

Primary Objective:Double-blind period : Evaluate the effect of SRP-4045 and SRP-4053 (combined-active group) compared to placebo on ambulation, endurance, and muscle function, as measured by the 6MWTSecondary Objectives:- Double-blind period:…

A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1

This study has been transitioned to CTIS with ID 2023-510353-42-00 check the CTIS register for the current data. The purpose of this first-in-human study is to evaluate the safety, tolerability, efficacy, pharmacokinetics (PK), and pharmacodynamics…