Clinical trials: Muscle Disorders
Duchenne Muscular Dystrophy
Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD)
Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients with Duchenne Muscular Dystrophy(DMD)
- Trial name
- NCT05338099
- Testing
- EN001
- Where
- South Korea
- Volunteers
- 7 people
- Sponsor
- ENCell
Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles
Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
The objective of this study is to evaluate the long-term safety and efficacy of Ace-ER treatment in subjects with GNEM.
- Conditions
- Hereditary Inclusion Body Myopathy, Distal Myopathy With Rimmed Vacuoles, Distal Myopathy, Nonaka Type, GNE Myopathy
- Trial name
- NCT02736188
- Testing
- Aceneuramic Acid Extended-Release Tablets
- Where
- 14 sites in United States, Bulgaria and 5 more
- Volunteers
- 143 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Musculoskeletal Pain
Low Dose of Cannabidiol (CBD) to Treat Mild to Moderate Musculoskeletal Pain
The purpose of the study is to evaluate whether Pure Green tablets is a better pain reliever for the treatment of mild to moderate musculoskeletal pain.
- Trial name
- NCT04193631
- Testing
- Cannabidiol (CBD)
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Pure Green
Duchenne Muscular Dystrophy
An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
The main purposes of this study are to see if it is safe to use a new medication called vamorolone for more than two weeks in children with Duchenne muscular dystrophy (DMD), to see if vamorolone works for the treatment for DMD…
- Trial name
- NCT02760277
- Testing
- Vamorolone 0.25 mg/day/day, Vamorolone 0.75 mg/day/day, Vamorolone 2.0 mg/day/day
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 48 people
- Sponsor
- ReveraGen BioPharma, Inc.
Thymidine Kinase 2 Deficiency
An Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With TK2 Deficiency
This is a Phase 2 prospective open-label treatment study of the safety and efficacy of doxecitine and doxribtimine in study participants with thymidine kinase 2 (TK2) deficiency who participated in the retrospective study…
- Trial name
- Continuation (NCT03845712)
- Testing
- doxecitine and doxribtimine
- Where
- 8 sites in United States, Israel and 1 more
- Volunteers
- 47 people
- Sponsor
- UCB BIOSCIENCES, Inc.
Musculoskeletal Inflammation, Exercise-Induced Muscle Damage
A Clinical Trial to Evaluate the Effects of Peptide Gummies on Markers of Inflammation, Physical Performance, and Recovery
This study will evaluate the effects of Parlay Recover peptide gummies (containing Body Protection Compound BPC-157) on markers of inflammation, physical performance, and recovery in physically active adults.
- Conditions
- Musculoskeletal Inflammation, Exercise-Induced Muscle Damage, Post-Exercise Recovery, Musculoskeletal Pain
- Trial name
- NCT07752381
- Testing
- Parlay Recover Peptide Gummies
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Parlay Wellness
Sporadic Inclusion Body Myositis
Safety and Efficacy of REGN2477+REGN1033 in Patients With Sporadic Inclusion Body Myositis
The primary objective of the study is to evaluate the efficacy of REGN2477+REGN1033 in combination on total lean mass, as measured by Dual-energy X-ray absorptiometry (DXA) in patients with sporadic inclusion body myositis…
- Trial name
- NCT03710941
- Testing
- REGN2477+REGN1033
- Sponsor
- Regeneron Pharmaceuticals
Endometriosis, Uterine Fibroids, Male Hormone-sensitive Prostate Cancer, Assisted Reproduction
A Clinical Study Evaluating the Relative Bioavailability of SHR7280 Dry Suspension and Tablets
To assess the relative bioavailability of SHR7280 dry suspension and tablets in healthy subjects. To assess the safety and tolerability of a single dose of SHR7280 dry suspension and tablets.
- Trial name
- NCT05868057
- Testing
- SHR7280 tablets, SHR7280 dry suspension
- Where
- China
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Uterine Fibroids, Polyps
HOME Study: Hysteroscopic Office Myomectomy Evaluation
The purpose of this study is to demonstrate safety and effectiveness of the MyoSure Tissue Removal System when used by community gynecologists in an office setting for the treatment of patients with symptomatic intrauterine…
- Trial name
- HOME (NCT01152112)
- Testing
- Myomectomy
- Where
- 9 sites in United States
- Volunteers
- 74 people
- Sponsor
- Hologic, Inc.
Non-muscle Invasive Bladder Cancer
CAVATAK (CVA21) in Non-muscle Invasive Bladder Cancer (VLA-012 CANON)
The study consisted of 2 sequential parts. Part A assessed the safety and tolerability of CAVATAK administered via intravesical instillation in patients with non-muscle invasive bladder cancer scheduled to undergo TUR.
- Trial name
- CANON (NCT02316171)
- Testing
- CVA21, Mitomycin C
- Where
- United Kingdom
- Volunteers
- 16 people
- Sponsor
- Viralytics
Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
The KHENERGYC Study
This a randomized placebo controlled, double-blind phase II study to explore the pharmacokinetics, safety and efficacy of sonlicromanol in children (from birth to 17 years) with genetically confirmed mitochondrial disease of…
- Conditions
- Mitochondrial Diseases, Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation, MELAS, Subacute Necrotizing Encephalomyelopathy
- Trial name
- NCT04846036
- Testing
- Sonlicromanol
- Where
- Netherlands
- Volunteers
- 24 people
- Sponsor
- Khondrion BV
Low Muscle Strength, Malnutrition
Efficacy of Oral Nutritional Supplement in Community-Dwelling Adults Following Acute Care
The study aims to determine the effects of Oral Nutritional Supplement (ONS) added to usual care on body weight compared with usual care alone in community-dwelling adults aged 55 years and older who are at risk of malnutrition…
- Trial name
- NCT07753239
- Testing
- ONS, Standard of Care (SOC)
- Where
- 7 sites in Thailand
- Volunteers
- 304 people
- Sponsor
- Abbott Nutrition
Muscle Invasive Bladder Cancer
Durvalumab+ Gemcitabine/Cisplatin (Neoadjuvant Treatment) and Durvalumab (Adjuvant Treatment) in Patients With MIBC
A Global Study to Determine the Efficacy and Safety of Durvalumab in Combination with Gemcitabine+Cisplatin for Neoadjuvant Treatment and Durvalumab Alone for Adjuvant Treatment in Patients with Muscle-Invasive Bladder Cancer
- Trial name
- NIAGARA (NCT03732677)
- Testing
- Durvalumab, Cisplatin, Gemcitabine
- Where
- 188 sites in United States, Australia and 20 more
- Volunteers
- 1,063 people
- Sponsor
- AstraZeneca
Fibromyalgia, Painful Diabetic Peripheral Neuropathy
Examination of Pregabalin Access for Treatment of Indicated Pain Disorders: the ExPAND Study
The investigators hypothesize that fibromyalgia (FM) and painful diabetic peripheral neuropathy (pDPN) patients with access restrictions on pregabalin will lead to higher healthcare resource use and cost compared to patients…
- Trial name
- NCT01280747
- Testing
- prior authorization, no prior authorization
- Where
- 17 sites in United States
- Volunteers
- 42 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Fibromyalgia
Micronized and Ultramicronized Palmitoylethanolamide in Fibromyalgia Patients
The onset of chronic Fibromyalgia symptomatology is due to central alterations, together with peripheral neuroimmune modifications.
- Trial name
- NCT04488926
- Testing
- micronized and ultra-micronized Palmitoylethanolamide (mPEA and umPEA, 300mg + 600mg) microgranules, Standard Therapy, Rescue Drug
- Where
- Italy
- Volunteers
- 21 people
- Sponsor
- Epitech Group SpA
Duchenne Muscular Dystrophy (DMD)
A Study of PGN-EDO51 or Placebo in People With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
The study consists of 3 periods: A Screening Period (up to 45 days), a double-blind, placebo-controlled Multiple Ascending Dose (MAD) Period (28 weeks), and a Long-Term Extension (LTE) Period (108 weeks).
- Trial name
- CONNECT2-EDO51 (NCT06833931)
- Testing
- IV infusion
- Sponsor
- PepGen Inc
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain
An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).
- Trial name
- NCT06632730
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Novartis
Musculoskeletal Pain, Chronic Shoulder Pain
Digital Versus Conventional Physical Therapy for Chronic Shoulder Tendinopathy
This is a single-center, prospective, randomised, controlled study, with two parallel groups, designed to assess the clinical impact of a digital exercise program against conventional rehabilitation for shoulder tendonitis.
- Conditions
- Musculoskeletal Pain, Chronic Shoulder Pain, Shoulder Tendinopathy
- Trial name
- NCT04636528
- Testing
- Digital rehabilitation, Conventional rehabilitation
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Sword Health, SA
Dermatomyositis, Polymyositis
Study of M5049 in DM and PM Participants (NEPTUNIA)
The purpose of this study is to evaluate the efficacy and safety of orally administered M5049 in idiopathic inflammatory myopathies, specifically dermatomyositis (DM) and polymyositis (PM) participants for 24 weeks.
- Trial name
- NCT05650567
- Testing
- M5049 dose
- Where
- 31 sites in United States, Czechia and 5 more
- Volunteers
- 20 people
- Sponsor
- EMD Serono Research & Development Institute, Inc.
Malnutrition, Calorie, Sarcopenia
Malnutrition Screening Study
This is a non-interventional, cross-sectional, non-randomized, observational study to screen for the risk of malnutrition and sarcopenia in outpatients aged 18 and over who attend hospital clinics for any reason in Turkey.
- Trial name
- NCT07256925
- Where
- 69 sites in Turkey (Türkiye)
- Volunteers
- 7,500 people
- Sponsor
- Abbott Nutrition
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