Clinical trials: Congenital Myopathy
Muscular Dystrophies, Muscular Dystrophy, Duchenne
A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called deramiocel (CAP-1002) in study participants with Duchenne…
- Conditions
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- HOPE-3 (NCT05126758)
- Testing
- Deramiocel (CAP-1002)
- Where
- 20 sites in United States
- Volunteers
- 106 people
- Sponsor
- Capricor Inc.
Urea Cycle Disorder, Organic Acidemia
Systemic Biomarkers of Brain Injury From Hyperammonemia
Ammonia is a waste product of protein and amino acid catabolism and is also a potent neurotoxin. High blood ammonia levels on the brain can manifest as cytotoxic brain edema and vascular compromise leading to intellectual and…
- Conditions
- Urea Cycle Disorder, Organic Acidemia, Maple Syrup Urine Disease, Glutaric Acidemia I
- Trial name
- NCT04602325
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Children's National Research Institute
Duchenne Muscular Dystrophy (DMD)
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 healthy male participants with administration of…
- Trial name
- NCT07347548
- Testing
- GRT6019
- Where
- France
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Grünenthal GmbH
Myotonic Dystrophy 1
Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1
The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).
- Trial name
- FREEDOM-DM1 (NCT06204809)
- Testing
- PGN-EDODM1 for infusion
- Where
- 12 sites in United States, Canada and 1 more
- Volunteers
- 24 people
- Sponsor
- PepGen Inc
Congenital Myopathy
Aerobic Training in Patients With Congenital Myopathies
Congenital myopathies are clinical and genetic heterogeneous disorders characterized by skeletal muscle weakness. Most patients experience muscle weakness and fatigue throughout their life.
- Trial name
- NCT02020187
- Testing
- Exercise on a cycle-ergometer
- Where
- Denmark
- Volunteers
- 16 people
- Sponsor
- Copenhagen University Hospital, Rigshospitalet
Myotonic Dystrophy 1, Dysphagia, Oropharyngeal
Profile of Dysphagia in Myotonic Dystrophy Type 1 (DM1)
The goal of this observational study is to learn about swallowing difficulties (dysphagia) in patients living with myotonic dystrophy type 1 (DM1).
- Trial name
- SwallowDM1 (NCT05865483)
- Where
- United Kingdom
- Volunteers
- 177 people, healthy volunteers welcome
- Sponsor
- University College, London
Age-Related Macular Degeneration, Allergies
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
To collect, preserve, and/or distribute annotated biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease
- Trial name
- NCT05635266
- Testing
- Specimen sample
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Inherited Mitochondrial Disease, Including Leigh Syndrome
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in…
- Trial name
- MITO-001 (NCT02023866)
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 36 people
- Sponsor
- Amgen
Postural Kyphosis, Neck Pain
Comparison Between Effect of Posterior Cervical Weighting and Deep Cervical Flexion Exercise on Forward Head Posture
It has been reported that the head protrudes forward relative to the body from the sagittal plane associated with the anterior shifting of the line of gravity (LOG) relative to the base of support (BOS).
- Conditions
- Postural Kyphosis, Neck Pain, Deformity of Spine, Deformity
- Trial name
- FHP (NCT04796051)
- Testing
- Neck orthosis - with posterior cervical weight, Deep cervical flexors exercise
- Where
- Egypt
- Volunteers
- 61 people, healthy volunteers welcome
- Sponsor
- Alexandria University
ALS, Autism Spectrum Disorder
Pharmacokinetic (PK) and Pharmacodynamics (PD) Study of Ilera Specific Products
This is an observational study of medical marijuana manufactured and dispensed by Ilera and given as standard treatment for a variety of approved serious medical conditions as defined by individual state law.
- Conditions
- ALS, Autism Spectrum Disorder, Cancer, Spasticity, Muscle
- Trial name
- Ilera (NCT03886753)
- Testing
- Registry, PK microsampling of blood
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Children's Hospital of Philadelphia
Muscle Weakness
Effect of PROGRESSive Training and Teststerone in Older Frail Men
In this scientific clinical investigation we will test whether testosterone and progressive resistance training can improve muscle strength and reduce the risk of falls in older men.
- Trial name
- PROGRESS (NCT02873559)
- Testing
- testosterone, Progressive muscle training
- Where
- 2 sites in Denmark
- Volunteers
- 192 people, healthy volunteers welcome
- Sponsor
- University Hospital, Gentofte, Copenhagen
X-linked Myotubular Myopathy
Respiratory Muscle Function in Untreated X-Linked Myotubular Myopathy (XLMTM)
This study is a longitudinal study evaluating the severity and progression of respiratory muscle function in patients with X-Linked Myotubular Myopathy (XLMTM) aged 0-14.
- Trial name
- NCT02453152
- Testing
- History and physical, Tidal breathing, Maximal respiratory pressures
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- University of Florida
Myocardial Fibrosis, Muscular Dystrophies
Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - ACE Inhibitor Therapy Trial
This trial intends to evaluate myocardial Fibrosis progression in Duchenne and Becker Muscular Dystrophy, as well the influence of ACE inhibitors in fibrosis progression.
- Trial name
- NCT02432885
- Testing
- Enalapril
- Volunteers
- 76 people
- Sponsor
- InCor Heart Institute
Myotonic Dystrophy, Type 1 (DM1), Myotonic Dystrophy
Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1
Participants in this study will receive two treatments, placebo and ERX-963, on different days in a randomized fashion. The primary purpose of this study is to investigate the safety and tolerability of ERX-963 in participants…
- Trial name
- NCT03959189
- Testing
- ERX-963
- Where
- 4 sites in United States
- Volunteers
- 12 people
- Sponsor
- Expansion Therapeutics, Inc.
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
Brain Involvement in Dystrophinopathies Part 1
The objective of this study is to collect data from a large cohort of individuals with DMD and BMD focusing on the neurobehavioural aspects of these conditions and their correlation to the location of the DMD gene mutation.
- Trial name
- NCT04583917
- Where
- 7 sites in Denmark, France and 4 more
- Volunteers
- 377 people
- Sponsor
- Great Ormond Street Hospital for Children NHS Foundation Trust
Congenital Myopathy, Acquired Myopathy
Magnetic Resonance Imaging and Ultrasonography in Evaluation of Muscle Diseases
This study aims to determine the association between the pathological changes detected by ultrasound and those detected in MRI in muscle diseases of different etiologies.
- Conditions
- Congenital Myopathy, Acquired Myopathy, Myositis
- Trial name
- NCT05692349
- Testing
- MRI
- Volunteers
- 36 people
- Sponsor
- Assiut University
Duchenne Muscular Dystrophy
Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
To evaluate the long-term safety and tolerability of ACE-031 administration in subjects with Duchenne muscular dystrophy (DMD) who participated in Study A031-03. [Note: This study was terminated based on preliminary safety data.
- Trial name
- NCT01239758
- Testing
- ACE-031 (Extension of cohort 1 from core study, A031-03), ACE-031 (Extension of cohort 2 from core study, A031-03), ACE-031 (Extension of cohort 3 from core study, A031-03)
- Where
- 4 sites in Canada
- Volunteers
- 11 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Myotonic Dystrophy Type 1 (DM1)
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
- Trial name
- Galileo (NCT06185764)
- Testing
- VX-670
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 47 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Duchenne Muscular Dystrophy
Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
This is a Phase 2 open-label extension (OLE) study to evaluate the long-term safety, tolerability, efficacy, pharmacokinetics, and the pharmacodynamics (PD) through potential exploratory biomarker(s) of intravenous (IV) WVE-N531…
- Trial name
- NCT07209332
- Testing
- WVE-N531
- Where
- 3 sites in Jordan, United Kingdom
- Volunteers
- 175 people
- Sponsor
- Wave Life Sciences USA, Inc.
Duchenne Muscular Dystrophy
A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
The purpose of this study is to understand the safety and effects of an experimental gene therapy called fordadistrogene movaparvovec. We are seeking participants from previous Pfizer interventional studies.
- Trial name
- NCT05689164
- Testing
- fordadistrogene movaparvovec
- Where
- 12 sites in United States
- Volunteers
- 7 people
- Sponsor
- Pfizer
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