Clinical trials: DMD
DMD, Duchenne Muscular Dystrophy
Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping
AOC 1044-CS2 (EXPLORE44-OLE) is an Open-label Study to Evaluate the Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants with Mutations Amenable to Exon 44 Skipping.
- Conditions
- DMD, Duchenne Muscular Dystrophy, Duchenne, Exon 44
- Trial name
- EXPLORE44OLE (NCT06244082)
- Testing
- AOC 1044
- Where
- 10 sites in United States
- Volunteers
- 39 people
- Sponsor
- Avidity Biosciences, Inc.
Duchenne Muscular Dystrophy (DMD)
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
This is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition.
- Trial name
- ELEVATE-45 (NCT07038824)
- Testing
- ENTR-601-45
- Where
- 15 sites in Belgium, Italy and 3 more
- Volunteers
- 24 people
- Sponsor
- Entrada Therapeutics, Inc.
Muscular Dystrophies, Spinal Muscular Atrophy
Schulze Muscular Dystrophy Ability Clinical Study
The primary objective of the Schulze study is to evaluate the function of the upper limbs of subjects diagnosed with neuromuscular disorders, with and without use of the Abilitech Assist device in the clinic and home…
- Conditions
- Muscular Dystrophies, Spinal Muscular Atrophy, Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy
- Trial name
- NCT05409079
- Testing
- Abilitech Assist
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- AbiliTech Medical Inc.
Duchenne Muscular Dystrophy, Gamification
Gamified Occupational Therapy for Adolescents With Duchenne Muscular Dystrophy
This research aims to improve the quality of life, occupational performance, occupational satisfaction and emotional health of young people with Duchenne muscular dystrophy compared to the classical occupational therapy program.
- Conditions
- Duchenne Muscular Dystrophy, Gamification, Occupational Therapy
- Trial name
- DMD (NCT06402942)
- Testing
- Gamified occupational therapy programme, Classic occupational therapy programme
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people
- Sponsor
- Başak Çağla Arslan
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
Duchenne Muscular Dystrophy, Duchenne Disease
Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients
This pilot study will investigate the potential efficacy of VM100, a nutritional supplement specifically formulated for patients with DMD, on quality of life and physical symptoms.
- Conditions
- Duchenne Muscular Dystrophy, Duchenne Disease, Muscular Dystrophy in Children, DMD
- Trial name
- NCT07766980
- Testing
- VM100 Nutritional Supplement
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Florida
Duchenne Muscular Dystrophy, Gait Disorders in Children
Quality and Independence of Gait Classification Scale for DMD (QIGS-DMD)
The aim of this study was to develop a reliable and valid gait classification scale for Duchenne Muscular Dystrophy (QIGS-DMD). The items of the QIGS-DMD were designed based on the literature review considering existing…
- Trial name
- NCT05564962
- Testing
- Quality and Independence of Gait Classification Scale for Duchenne Muscular Dystrophy (QIGS-DMD)
- Where
- Turkey (Türkiye)
- Volunteers
- 69 people
- Sponsor
- Hacettepe University
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy
Wearable Technology to Assess Gait Function in SMA and DMD
The purpose of this project is to devise instrumented insoles capable of accurately measuring gait at each footfall, over multiple hours in any environment.
- Trial name
- NCT04193085
- Testing
- Observational
- Where
- United States
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- Columbia University
Duchenne Muscular Dystrophy
Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy
The task consists in reach as much bubbles as they can, the bubbles appear on the screen of the computer and should be reached in 10 seconds.
- Trial name
- NCT02891434
- Testing
- Acquisition on TouchScreen, Acquisition on Kinect, Acquisition on LeapMotion
- Where
- Brazil
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University of Sao Paulo
Neuromuscular; Disorder, Hereditary, Duchenne/Becker Muscular Dystrophy
Molecular Analysis of Patients With Neuromuscular Disease
The purpose of this study is to identify new genes responsible for neuromuscular disorders and study muscle tissue of patient with known neuromuscular disease, as well as their family members.
- Conditions
- Neuromuscular; Disorder, Hereditary, Duchenne/Becker Muscular Dystrophy, Limb-girdle Muscular Dystrophy
- Trial name
- NCT00390104
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Boston Children's Hospital
Duchenne Muscular Dystrophy
An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy
This is an open-label study to evaluate the safety and tolerability of golodirsen injection in Non-ambulant DMD patients with confirmed genetic mutations amenable to treatment by exon 53 skipping (Golodirsen).
- Trial name
- NCT04708314
- Testing
- Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53]
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- Rare Disease Research, LLC
Duchenne Muscular Dystrophy, Ambulation Difficulty
Cultural Adaptation, Validity, and Reliability of the Turkish Version of North Star Ambulatory Assessment
The aim of this study is to translate the "North Star Ambulatory Assessment (NSAA)" scale into Turkish and make its cultural adaptation and to demonstrate the reliability and validity of the Turkish version in patients with…
- Conditions
- Duchenne Muscular Dystrophy, Ambulation Difficulty, Ambulation Disorder, Neurologic
- Trial name
- NCT05549999
- Testing
- North Star Ambulatory Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 86 people
- Sponsor
- Hacettepe University
Duchenne Muscular Dystrophy
Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein.
- Trial name
- NCT02329769
- Testing
- PRO044 SC 6 mg/kg, PRO044 IV 6 mg/kg, PRO044 IV 9 mg/kg
- Where
- 5 sites in Belgium, Italy and 2 more
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
Duchenne Muscular Dystrophy, Musculoskeletal Abnormalities
Pediatric Radio Frequency Coils Generic
The purpose of this study is to evaluate and optimize advances in radio frequency (RF) coil magnetic resonance imaging (MRI) technology at Cincinnati Children's Hospital Medical Center (CCHMC).
- Trial name
- NCT01633866
- Where
- United States
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Becker Muscular Dystrophy, Muscular Dystrophies
Defining Endpoints in Becker Muscular Dystrophy
This is a 24-month, observational study of 50 participants with Becker muscular dystrophy (BMD)
- Conditions
- Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
- Trial name
- GRASP-01-002 (NCT05257473)
- Where
- 11 sites in United States, New Zealand and 1 more
- Volunteers
- 59 people
- Sponsor
- Virginia Commonwealth University
Duchenne Muscular Dystrophy
A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy
This Phase IIb study is a two part, multicenter study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of ATL1102 in non-ambulant boys with Duchenne Muscular Dystrophy aged 10 to <18 years old.
- Trial name
- NCT05938023
- Testing
- ATL1102 25mg, ATL1102 50mg
- Where
- 13 sites in Australia, Bulgaria and 3 more
- Volunteers
- 48 people
- Sponsor
- Percheron Therapeutics
Duchenne Muscular Dystrophy, Pompe Disease (Late-onset)
Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease
This is a multicenter prospective non-drug screening study. The working period is 12 months. There is no research product to be followed or used in the study.
- Trial name
- VICTORIA (NCT04120168)
- Testing
- Laboratory Tests
- Where
- 51 sites in Turkey (Türkiye)
- Volunteers
- 590 people
- Sponsor
- Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition
Duchenne Muscular Dystrophy
Long Term Follow-up for RGX-202
RGX-202-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-202 in participants who have received RGX-202 (an investigative gene therapy designed to deliver a transgene for novel…
- Trial name
- NCT06491927
- Testing
- No Intervention
- Where
- 5 sites in United States
- Volunteers
- 66 people
- Sponsor
- REGENXBIO Inc.
Duchenne Muscular Dystrophy (DMD)
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
The primary objective of this study is to evaluate the safety and tolerability of SPOT-03 administered by intravenous (IV) infusion to DMD patients.
- Trial name
- NCT07188012
- Testing
- SPOT-03
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Shanghai Siponuoyin Biotechnology Co Ltd
Duchenne / Becker Muscular Dystrophy, Dystrophia Myotonica 1
Evaluation of the Role of miR-1 in the Pathogenesis and as a Biomarker in Muscular Dystrophies and Congenital Myopathies
The study aims to find out if a specific blood molecule called miR-1, can be used as a biomarker to track the health of patients with certain muscle diseases.
- Conditions
- Duchenne / Becker Muscular Dystrophy, Dystrophia Myotonica 1, Congenital Myopathies, Healthy Participants
- Trial name
- Dystro-miR1 (NCT07415837)
- Testing
- dosage of blood biomarker miR1
- Where
- France
- Volunteers
- 104 people, healthy volunteers welcome
- Sponsor
- University Hospital, Clermont-Ferrand