Clinical trials: Myopathy
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.
Cerebral Palsy
Effect of Botulinum Toxin on Muscles of Children With Cerebral Palsy
Cerebral palsy (CP) is a group of non-progressive motor dysfunction but often changing, secondary to injury or brain abnormalities that occur in early stages of development.
- Trial name
- TOXIMUS_CP (NCT02853240)
- Testing
- Muscle biopsy
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Hospices Civils de Lyon
Anti-3-hydroxy-3-methylglutaryl-CoA Reductase (HMGCR) Immune-Mediated Necrotizing Myopathy
The MIGHT Trial - An Exploratory Clinical Trial of IVIG in Anti-HMGCR Immune Mediated Necrotizing Myopathy
This is a randomized, placebo-controlled, double blinded phase 2 exploratory clinical trial of intravenously administered pooled human immunoglobulin (IVIG) in anti-3-hydroxy-3-methylglutaryl-CoA reductase (HMGCR) immune mediated…
- Trial name
- NCT06599697
- Testing
- Intravenously administered pooled human immunoglobulin (IVIG)
- Where
- 5 sites in United States
- Volunteers
- 12 people
- Sponsor
- University of Alabama at Birmingham
Increase Muscle Strength in Patients With Muscular Dystrophy
Efficacy Safety of Granulocyte Colony-stimulating Factor Treatment Children and Adolescents With Muscular Dystrophy
Importance: Currently the gold standard treatment for ambulant patients is corticosteroids. Granulocyte colony-stimulating factor (G-CSF) has been reported to exert the proliferation of satellite cells, the regulation of myoblast…
- Trial name
- NCT02814110
- Testing
- Granulocyte colony-stimulating factor (Filgrastim)
- Where
- Poland
- Volunteers
- 27 people
- Sponsor
- Medical University of Bialystok
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.
TGCT
Study of Emactuzumab for Tenosynovial Giant Cell Tumor (TGCT)
This is a multicenter, Phase 3, randomised, double-blind, placebo-controlled study, which aims to evaluate the efficacy and safety of the investigational drug emactuzumab for the treatment of patients with localized or diffuse…
- Trial name
- TANGENT (NCT05417789)
- Testing
- Emactuzumab
- Where
- 33 sites in United States, Belgium and 10 more
- Volunteers
- 128 people
- Sponsor
- SynOx Therapeutics Limited
Autosomal Dominant Hypocalcemia (ADH)
Efficacy and Safety of Encaleret Compared to Standard of Care in Participants With ADH1
The primary purpose of the study is to understand the effectiveness, safety, and tolerability of encaleret when compared to standard of care (SoC) treatment in participants with Autosomal Dominant Hypocalcemia Type 1 (ADH1).
- Trial name
- CALIBRATE (NCT05680818)
- Testing
- Encaleret, Standard of Care
- Where
- 25 sites in United States, Australia and 8 more
- Volunteers
- 67 people
- Sponsor
- Calcilytix Therapeutics, Inc., a BridgeBio company
Myogenic Temporomandibular Joint Disorders, Internal Derangement of the Tempromandibular Joint
Adjustment Technique for Occlusal Stabilizing Splint
This study aims to clarify the technique of occlusal stabilizing splint adjustment then to assess the overall clinical outcomes and patient satisfaction.
- Conditions
- Myogenic Temporomandibular Joint Disorders, Internal Derangement of the Tempromandibular Joint, Temporomandibular Disorders (TMDs), Temporomandibular Joint Disc Displacement
- Trial name
- NCT07397013
- Testing
- Stabilizing Splint(3-point of Contact), Stabilizing Splint (Centric and Eccentric)
- Where
- Egypt
- Volunteers
- 800 people
- Sponsor
- Mansoura University
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
Reactive Airways Dysfunction Syndrome (Disorder), Muscle Weakness
Efficacy of Inspiratory Muscle Training on Elite Swimmers
This study is a randomized controlled trial on the efficacy of inspiratory muscle training on swimming performance, airway dysfunction and perceived breathlessness in elite swimmers.
- Conditions
- Reactive Airways Dysfunction Syndrome (Disorder), Muscle Weakness, Dyspnea
- Trial name
- PEAK (NCT03062735)
- Testing
- Inspiratory Muscle Training
- Where
- Portugal
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Universidade do Porto
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
Chronic Exertional Compartment Syndrome
Utilization of High Frequency Ultrasound to Diagnose Chronic Exertional Compartment Syndrome
The purpose of this study is to investigate high frequency ultrasound as a future modality for the diagnosis of chronic exertional compartment syndrome (CECS).
- Trial name
- NCT03736174
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Colorado, Denver
Neuromuscular Disease
Diagnostic Evaluation of Patients With Neuromuscular Disease
The objectives of this protocol are to: 1) screen patients with various neuromuscular disorders and facilitate their entry into appropriate research protocols; 2) help resolve puzzling diagnostic neuromuscular problems and train…
- Trial name
- NCT00015470
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Cerebral Palsy, Muscle Spasticity
Dysport® Pediatric Lower Limb Spasticity Follow-on Study
The purpose of this research study was to determine the long term safety and efficacy of repeated treatments with Dysport® used in the treatment of lower limb spasticity in children with dynamic equinus foot deformity due to…
- Conditions
- Cerebral Palsy, Muscle Spasticity, Children
- Trial name
- NCT01251380
- Testing
- Botulinum toxin type A
- Where
- 27 sites in United States, Chile and 4 more
- Volunteers
- 216 people
- Sponsor
- Ipsen
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
Glycogen Storage Disease, GSD Type 0A
Rare Glycogen Storage Diseases Natural History Study
The purpose of this study is to collect and study key medical data about several ultra-rare GSDs (Glycogen Storage Diseases) including, but not limited to, GSD types 0a, 0b, VII, X, XII, XIII, XV, PRKAG2 syndrome and Danon…
- Conditions
- Glycogen Storage Disease, GSD Type 0A, GSD Type 0B, GSD VII
- Trial name
- NCT06795152
- Testing
- No intervention
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Duke University
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