Duchenne Muscular Dystrophy (DMD) Clinical Trials
14 recruiting trials for Duchenne Muscular Dystrophy (DMD). Eligibility criteria explained in plain English.
Recruiting Trials
Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal...
The Active NBS Liege study is a monocentric, academic, fully remote, observational study designed to validate digital measures of motor development in children with spinal...
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and...
This is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The researchers want to: Test how...
Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
The purpose of this study is to evaluate the safety, tolerability, and efficacy of LE051 intravenous therapy in DMD patients treated with exon 51 skipping therapy.
Research on the Relationship Between Scoliosis, Pain, Quality of Life, and Trunk Muscle Compensation Patterns Among...
* Objective: The objective of this observational study is to evaluate and quantify trunk muscle compensatory movement patterns in patients with Duchenne Muscular Dystrophy (DMD)...
Urinary Titin Biomarker in DMD
A universal challenge in clinical investigation of novel therapeutics is the need for quantitative, objective biomarkers that directly address the mechanisms of disease and...
DMD Voice: Qualitative Interviews With Patients and Caregivers
The purpose of this study is to understand DMD functional losses or abilities and their association with independence and quality of life from the perspective of individuals with...
Safety and Dystrophin Expression of SPOT-mRNA03 in Duchenne Muscular Dystrophy (DMD) Patients
The primary objective of this study is to evaluate the safety and and tolerability of SPOT-mRNA03 administered by intravenous (IV) infusion to DMD patients. In addition, this...
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and...
This is a study of the investigational medicine ENTR-601-44 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The researchers want to: Test...
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
The purpose of this research is (1) to identify disease specific walking-related digital biomarkers of disease severity, and (2) monitor longitudinal changes in natural...
A Trial to Investigate the Safety and Pharmacokinetics of GRT6019 in Healthy Male Participants
The purpose of this trial is to assess the safety, tolerability, and PK of 3 doses of GRT6019 in healthy male participants. This Phase I trial will be a multiple dose trial in...
Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders
Both GIP and GLP-2 reduce bone resorption (measured as CTX) in healthy adult individuals. In this study, we will investigate whether GIP and GLP-2 reduce CTX in children with...
Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy
The purpose of the study is to assess the safety, tolerability, and efficacy of BBM-D101 to treat patients with Duchenne Muscular Dystrophy.
Fear of Falling in Muscular Dystrophy
Primary objectives WP1: Evaluate the prevalence of FOF in the study population and how this varies over time. Evaluate whether there are relationships between the variables...
Follow-up of NIV At Home in Patients with Duchenne Muscular Dystrophy
The aim of this study is to verify whether full video home polysomnography (sleep study) with nocturnal transcutaneous CO2 monitoring is feasible in the follow-up of non-invasive...