Clinical trials: Muscular Disorders, Atrophic
Myotonic Dystrophy Type 1 (DM1)
A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I
The purpose of the study is to evaluate the long-term safety and tolerability, efficacy and pharmacokinetics of VX-670 in participants with Myotonic Dystrophy Type I (DM1).
- Trial name
- NCT06926621
- Testing
- VX-670
- Where
- 16 sites in United States, Australia and 6 more
- Volunteers
- 44 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping
- Conditions
- Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Muscular Disorders, Atrophic, Muscular Disease
- Trial name
- SAFARI44 (NCT07587242)
- Testing
- AOC 1044
- Where
- 18 sites in Australia, Belgium and 6 more
- Volunteers
- 70 people
- Sponsor
- Avidity Biosciences, Inc.
Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS)
Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)
The goal of this clinical trial is to evaluate the safety and preliminary efficacy of an implantable device that records and stimulates different areas of the brain to allow adults affected by disabling paralysis (see Eligibility…
- Conditions
- Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS), Muscular Disorders, Atrophic, Brain Stem Stroke
- Trial name
- INTENT (NCT07521930)
- Testing
- INTENT Neural Interface System
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Johns Hopkins University
Muscular Disorders, Atrophic, Muscular Diseases
Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping
The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping.
- Conditions
- Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases
- Trial name
- NCT07250737
- Testing
- delpacibart zotadirsen
- Where
- 22 sites in United States
- Sponsor
- Avidity Biosciences, Inc.
DM1, Muscular Dystrophies
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
AOC 1001-CS2 (MARINA-OLE) (NCT05479981) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) (NCT05027269) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001…
- Conditions
- DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1
- Trial name
- MARINA-OLE (NCT05479981)
- Testing
- AOC 1001
- Where
- 8 sites in United States
- Volunteers
- 37 people
- Sponsor
- Avidity Biosciences, Inc.
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.
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
Muscle Atrophy, Disuse Atrophy
Effect of Knee Position on Muscle Loss During Leg Immobilization
This study looks at what happens to leg muscles when the knee is kept straight or bent during five days of wearing a brace. We want to find out if keeping the knee bent (so the thigh muscle is stretched) helps prevent muscle loss…
- Conditions
- Muscle Atrophy, Disuse Atrophy, Immobilization
- Trial name
- NCT07690150
- Testing
- Knee immobilization brace (0°) for 5 days, Knee immobilization brace (60°) for 5 days
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Wageningen University
Atrophy of Muscle Due to Disuse, Rehabilitation
Sex-specific Determinants of Early-phase Recovery From Skeletal Muscle Disuse
This project is a 2-phase, randomized clinical trial that includes 7 days of unilateral leg disuse (Phase 1), immediately followed by 1 week of bilateral leg rehabilitation (Phase 2).
- Trial name
- MAC (NCT04151901)
- Testing
- Resistance Exercise Rehabilitation, Walking-based rehabilitation
- Where
- 2 sites in United States
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- The University of Texas Health Science Center at San Antonio
Disuse Atrophy, Immobilization
Cross-education of Skill and Strength in Healthy People Subjected to an Upper-limb Disuse Model
This randomized controlled trial aims to compare manual dexterity and strength between a group undergoing a contralateral manual dexterity training program and a no-intervention control group in healthy, physically inactive…
- Trial name
- IMMO-CROSS (NCT07615062)
- Testing
- Contralateral Manual Dexterity Training
- Where
- Chile
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Universidad Catolica de Temuco
Myotonic Dystrophy 1
An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
The purpose of this study is to learn about the long-term safety and tolerability of PGN-EDODM1 in participants with myotonic dystrophy type 1 (DM1) who have completed a prior study with PGN-EDODM1.
- Trial name
- NCT07220603
- Testing
- PGN-EDODM1
- Where
- 3 sites in Canada
- Volunteers
- 48 people
- Sponsor
- PepGen Inc
Muscular Dystrophy, Duchenne, Muscular Dystrophies
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT03179631
- Testing
- Ataluren
- Where
- 64 sites in United States, Australia and 16 more
- Volunteers
- 360 people
- Sponsor
- PTC Therapeutics
Cognitive Disorders, Muscular Disorders, Atrophic
Dietary Strategy to Tackle Cognitive and Locomotor Abilities in Early Elderly Subjects
Polyphenols, precisely resveratrol, with red wine as the most substantial source, was associated with improvements in cognitive function.
- Trial name
- WinAging (NCT06871384)
- Testing
- Nonalcoholic red wine group (Intervention group), Drinking water group (Control group)
- Where
- 3 sites in Spain
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- University Rovira i Virgili
Mitochondrial Energetics, Dietary Nitrate
Effect of Dietary Nitrate on Immobilization-induced Changes in Skeletal Muscle in Young Healthy Men
Diminished use of skeletal muscle, such as occurs with many chronic diseases (e.g., heart failure or cancer cachexia), denervation, bedrest, immobilization (e.g., limb casting or bracing), etc., is a common clinical condition…
- Conditions
- Mitochondrial Energetics, Dietary Nitrate, Disuse Atrophy (Muscle) of Lower Leg
- Trial name
- NCT07161973
- Testing
- Beetroot Juice - Active
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Indiana University
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
Muscular Atrophy, Insulin Resistance
Human Skeletal Muscle Response to 5 Days of Bedrest in Young Adults
The goal of this intervention trial is to characterize skeletal muscle atrophy in healthy, young adults during short term bedrest.
- Conditions
- Muscular Atrophy, Insulin Resistance, Muscle Protein Synthesis
- Trial name
- FABRIC (NCT07178353)
- Testing
- Bedrest
- Where
- Canada
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Queen's University
Sarcopenia, Skeletal Muscle Atrophy
Prehabilitative Exercise Prior to Immobilization
This is a 10-week human study involving 24 younger (20-35 y) and 24 older (65-85 y) healthy individuals. All participants will undergo unilateral immobilization of a knee for 7-10 days, followed by 4 weeks of heavy resistance…
- Conditions
- Sarcopenia, Skeletal Muscle Atrophy, Disuse Atrophy
- Trial name
- PREHAB (NCT06205784)
- Testing
- Prehabilitative exercise
- Where
- Denmark
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Bispebjerg Hospital
Muscle Disuse, Muscle Disuse Atrophy
Muscle Response to Different Amounts of Dietary Protein During Leg Immobilization
Individuals who sustain musculoskeletal injuries (MSKI) can experience a rapid loss of muscle mass due to declines in muscle loading and activation that occur post-injury (i.e., disuse atrophy).
- Trial name
- NCT07069426
- Testing
- 20 grams protein, 40 grams protein
- Where
- United States
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- United States Army Research Institute of Environmental Medicine
Muscular Dystrophies, Muscular Dystrophy, Duchenne
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne Muscular Dystrophy (DMD).
- Conditions
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- HOPE-2 (NCT03406780)
- Testing
- CAP-1002
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Capricor Inc.
Neurodegenerative Disorders
Development of IPS from Donated Somatic Cells of Patients with Neurological Diseases
Human fibroblasts and possibly other human somatic cells may be reprogrammed into induced pluripotent stem (iPS) cells by the forced expression of transcription factors (1-5).
- Trial name
- NCT00874783
- Where
- Israel
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Hadassah Medical Organization