Clinical trials: Muscle Disorders
Congenital Muscular Dystrophy Due to Lamin A/C Mutation
HBCMD01- Expanded Access for the Treatment of Congenital Muscular Dystrophy.
This individual patient expanded access IND is requested for a patient diagnosed with LMNA-related congenital muscular dystrophy (L-CMD).
- Trial name
- NCT05154851
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Duchenne Muscular Dystrophy
A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
The primary objective of this study is to evaluate the safety of a 0.9 milligrams per kilogram (mg/kg) and 0.45 mg/kg daily dose of deflazacort with a comparable natural history control group after 52 weeks of treatment in males…
- Trial name
- PTCEMF (NCT03642145)
- Testing
- Deflazacort
- Sponsor
- PTC Therapeutics
Spasticity, Cerebral Palsy
Shockwaves Therapy and Botulinum Toxin for the Treatment of Spasticity in Patients With Cerebral Palsy. A Cross Over RCT
Spasticity is a motor disorder that can affect the lower limbs in up to 75% patients with cerebral palsy, having a significantly effect on their mobility and quality of life.
- Trial name
- NCT02400619
- Testing
- shock waves, Botulinum Toxin Type A
- Where
- Spain
- Volunteers
- 70 people
- Sponsor
- Xavi Vidal Novellas
Juvenile Idiopathic Arthritis, Systemic Lupus Erythematosus
Development of a Therapeutic Endpoint in Pediatric Rheumatologic Conditions
The overarching goal of this study is the development of a physiologic endpoint of pain and treatment effect in three distinct rheumatology populations.
- Conditions
- Juvenile Idiopathic Arthritis, Systemic Lupus Erythematosus, Fibromyalgia
- Trial name
- NCT04833465
- Testing
- No Intervention
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- Children's National Research Institute
Soft Tissue Sarcoma, Bone Sarcoma
Trial of Sunitinib and/or Nivolumab Plus Chemotherapy in Advanced Soft Tissue and Bone Sarcomas
Phase I-II, single-arm, non-randomized, open-label, multicenter, international clinical trial, with two stages. Stage one has two cohorts (soft tissue sarcoma and bone sarcoma) and stage two has eight cohorts (DDCS, EMC, VS, SFT…
- Trial name
- ImmunoSarc (NCT03277924)
- Testing
- Sunitinib 37.5 MG, Sunitinib 25 MG [Sutent], Nivolumab 100 MG/10 ML [Opdivo], Epirubicin
- Where
- 13 sites in Italy, Spain and 1 more
- Volunteers
- 197 people
- Sponsor
- Grupo Espanol de Investigacion en Sarcomas
Ryanodine Receptor 1-Related Myopathy, Ryanodine Receptor 1 Related Disorders
A Natural History Study of RYR1-Related Disorders
Background: Congenital myopathies (CM) are genetic disorders that can cause decreased muscle tone and muscle weakness. Most CMs in the United States are related to the ryanodine receptor 1 (RYR1) gene.
- Trial name
- NCT06287762
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Muscular Dystrophy, Duchenne, Muscular Dystrophies
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren…
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT02090959
- Testing
- Ataluren
- Where
- 58 sites in United States, Australia and 17 more
- Volunteers
- 219 people
- Sponsor
- PTC Therapeutics
Flat Foot; Rigid, Flat Foot; Spastic
Joint Preserving Surgery for Correction of Spasmodic Flat- Foot in Skeletally Mature Foot
the investigators want to obtain a functional and radiological outcome of peroneus brevis, tertius and extensor digitorum longus muscles lengthening together with calcaneal osteotomies in the treatment of the spasmodic flat- foot.
- Trial name
- NCT03880318
- Testing
- Joint Preserving Surgery in spasmodic flat foot
- Volunteers
- 22 people
- Sponsor
- Assiut University
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.
Chronic Kidney Diseases
Skeletal and Respiratory Muscle Strength, Pulmonary Function, Exercise Capacity and Physical Activity in Pediatric Chronic Kidney Disease
Studies examining musculoskeletal strength, respiratory function and muscle strength, physical activity level and exercise capacity in children with CKD and evaluating the relationships between these parameters have not been…
- Trial name
- NCT05354726
- Testing
- Assessments parameters
- Where
- Turkey (Türkiye)
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Hacettepe University
Fontan Circulation
Muscle Function in Patients With Single-chamber Heart
Groundbreaking cardiac surgery has significantly improved survival rates for patients with single-chamber hearts. However, despite these life-saving interventions, the long-term prognosis remains concerning, with an increased…
- Trial name
- Muscle in TCPC (NCT07420660)
- Where
- Sweden
- Volunteers
- 34 people
- Sponsor
- Karolinska Institutet
Lipin-1 Deficiency
International Multicenter Study on Lipin-1 Deficiency
LIPIN1 is an international registry that allows the collection of data on the follow-up, management and treatment of patients with Lipin-1 deficiency.
- Trial name
- LIPIN1 (NCT05564520)
- Where
- France
- Volunteers
- 12 people
- Sponsor
- Imagine Institute
Gait, Spastic, Reliability
Reliability of Newly-acquired Gait in Toddlers With a Typical Development and a Unilateral Cerebral Palsy
gait reliability study of toddlers with typically developing and with unilateral cerebral palsy during the first 6 months of independent walking
- Trial name
- ReliabBB-AQM (NCT04559776)
- Testing
- gait analysis
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- National Clinical Center for Neuropsychomotor Recovery for Children Dr. Nicolae Robanescu
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Vitamin D Deficiency, Musculoskeletal Diseases
Vitamin D in School Children
The primary purpose of this study it to investigate total circulating 25-hydroxyvitamin D (25(OH)D) in school children (aged 4-11years) in Northern Ireland; thereby establishing the prevalence of vitamin D status (deficiency…
- Conditions
- Vitamin D Deficiency, Musculoskeletal Diseases, Cognitive Delay, Mild
- Trial name
- DVinCHI (NCT05018988)
- Testing
- Oral vitamin D spray
- Where
- United Kingdom
- Volunteers
- 118 people, healthy volunteers welcome
- Sponsor
- University of Ulster
Apnea, Sleep Apnea Syndromes
Oropharyngeal Exercises and Post-Stroke Obstructive Sleep Apnea
This study evaluates the feasibility and effectiveness of an oropharyngeal exercise (O-PE) regimen in treating post-stroke obstructive sleep apnea, as an alternative therapy to continuous positive airway pressure (CPAP).
- Conditions
- Apnea, Sleep Apnea Syndromes, Sleep Apnea, Obstructive, Stroke
- Trial name
- NCT04212260
- Testing
- Oropharyngeal exercises
- Where
- Canada
- Volunteers
- 33 people
- Sponsor
- Sunnybrook Health Sciences Centre
Spinal Muscular Atrophy (SMA)
A Study of the Safety and Preliminary Efficacy of NKG001 in Pediatric Patients With Type 1 or Type 2 Spinal Muscular Atrophy
This study is a single-center, open-label, single-arm, non-randomized, single-dose, dose-escalation investigator-initiated trial (IIT) designed to evaluate the safety, tolerability, and preliminary efficacy of NKG001 Injection…
- Trial name
- NCT07617779
- Testing
- NKG001
- Where
- China
- Volunteers
- 21 people
- Sponsor
- Nikegen Pharmaceutical (Hangzhou) Company Limited
Advanced Malignant Solid Neoplasm, Malignant Solid Neoplasm
Ensartinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With ALK or ROS1 Genomic Alterations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH treatment trial studies how well ensartinib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with ALK or ROS1 genomic alterations that have come back…
- Conditions
- Advanced Malignant Solid Neoplasm, Malignant Solid Neoplasm, Recurrent Ependymoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
- Trial name
- NCT03213652
- Testing
- Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Bone Scan
- Where
- 126 sites in United States, Australia and 2 more
- Volunteers
- 13 people
- Sponsor
- National Cancer Institute (NCI)
Cerebral Palsy (CP)
Effects of Barefoot vs. Shod Whole Body Vibration Training in Children With Cerebral Palsy
Cerebral palsy (CP) refers to a group of lifelong conditions that affect the development of movement and coordination that lead to activity limitations due to damage to the developing fetal or neonatal brain tissue.
- Trial name
- NCT06596525
- Testing
- Conventional physical therapy, Barefoot Whole Body Vibration Training (WBVTb), Shod Whole Body Vibration Training (WBVTs)
- Where
- Turkey (Türkiye)
- Volunteers
- 36 people
- Sponsor
- Trakya University
Duchenne Muscular Dystrophy
Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD), an X-linked recessive genetic disease always progressed slowly，tends to leading proximal skeletal muscle atrophy and weakness of limbs, as well as impaired respiratory muscle and cardiac muscle.
- Trial name
- NCT01610440
- Testing
- human umbilical cord mesenchymal stem cells
- Where
- China
- Volunteers
- 15 people
- Sponsor
- Shenzhen Beike Bio-Technology Co., Ltd.
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