Clinical trials: Musculoskeletal Abnormalities
Inflammatory Myopathy, Duchenne Muscular Dystrophy
Quantitative Muscle Ultrasound as a Marker of Progression in Children With Muscular Diseases
The aim of our study is to Assess skeletal muscle structural status in children with inflammatory myositis and Duchenne muscular dystrophy using musculoskeletal ultrasound and to perform a longitudinal follow up of these changes…
- Trial name
- NCT03786913
- Testing
- Quantitative muscle ultrasound measurements
- Where
- Egypt
- Volunteers
- 48 people
- Sponsor
- Benha University
Cleft Lip and Cleft Palate, Cleft Lip Bilateral
Improving the Outcomes of Nasoalveolar Molding in Complete Bilateral Cleft Lip and Palate Neonates Using a Modification in the Design of the Conventional Molding Appliance.
The goal of this clinical trial was to improve the outcome of using the nasoalveoalr molding appliances in patients with complete bilateral cleft lip and palate.
- Conditions
- Cleft Lip and Cleft Palate, Cleft Lip Bilateral, Complete Bilateral Cleft Lip and Palate
- Trial name
- NCT07814625
- Testing
- vomer molding appliance, conventional molding appliance
- Where
- Egypt
- Volunteers
- 20 people
- Sponsor
- Tanta University
Duchenne Muscular Dystrophy
Prognostic Factors Affecting Duchenne Muscular Dystrophy
Determination of prognostic factors affecting ambulation of duchenne muscular dystrophy
- Trial name
- NCT03372655
- Volunteers
- 82 people
- Sponsor
- Assiut University
Marfan Syndrome
Children and Adolescents With Marfan Syndrome: 10,000 Healthy Steps and Beyond
Marfan patients are at risk of sudden death due to weakening of the wall of the large blood vessel leading from the heart (aorta).
- Trial name
- NCT03567460
- Testing
- Exercise
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Stanford University
Haemophilia
National Survey of People With Haemophilia in Portugal
The purpose of this study is to implement a national survey to collect information regarding Portuguese people with haemophilia (PWH) on socio-demographic, clinical and psychosocial factors.
- Trial name
- NCT02870114
- Where
- Portugal
- Volunteers
- 146 people
- Sponsor
- University of Minho
Idiopathic Hypoparathyroidism, Autosomal Dominant Hypocalcaemia
Characterization of Patients With Non-surgical Hypoparathyroidism and Pseudohypoparathyroidism
Hypoparathyroidism (hypoPT) and pseudohypoparathyroidism (Ps-hypoPT) are rare diseases, characterized by low levels of parathyroid hormone [PTH] and plasma calcium or high plasma PTH and low plasma calcium, respectively.
- Conditions
- Idiopathic Hypoparathyroidism, Autosomal Dominant Hypocalcaemia, Pseudohypoparathyroidism
- Trial name
- NCT02551120
- Where
- Denmark
- Volunteers
- 100 people
- Sponsor
- University of Aarhus
Limb-Girdle Muscular Dystrophy, Type 2E
Limb Girdle Muscular Dystrophy Type 2E Recruitment Study
This study is to recruit and establish baseline measurements for potential subjects that may be eligible for a gene therapy trial.
- Trial name
- NCT03492346
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Nationwide Children's Hospital
Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy < 18y in Norway: Genotype/Phenotype, Growth, Puberty, Bone Health and Quality of Life.
The study will give a consent based epidemiological overview of Norwegian patients with Duchenne muscular dystrophy younger than 18 years of age. Genotype of the population will be described.
- Trial name
- NCT01963897
- Where
- Norway
- Volunteers
- 73 people
- Sponsor
- Oslo University Hospital
Duchenne Muscular Dystrophy (DMD), Neuromuscular Disorders
Relation Between Muscle Architecture and Functional Ability in Children With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD) is a progressive X-linked neuromuscular disorder characterized by muscle degeneration, pseudohypertrophy, and declining functional mobility.
- Conditions
- Duchenne Muscular Dystrophy (DMD), Neuromuscular Disorders, Muscle Architecture
- Trial name
- NCT07437378
- Where
- Egypt
- Volunteers
- 26 people
- Sponsor
- Deraya University
Pectus Excavatum, Cryo Analgesia
Phrenic Infiltration as Cryoanalgesia Adjuvant for Early Postoperative Pain Following the Nuss Procedure
The goal of this observational ambispective study is to compare the effectiveness of two pain management strategies in pediatric patients undergoing the Nuss procedure for pectus excavatum.
- Conditions
- Pectus Excavatum, Cryo Analgesia, Phrenic Nerve-related Diaphragmatic Impairment
- Trial name
- PHABULA (NCT07290582)
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- Papa Giovanni XXIII Hospital
Duchenne Muscular Dystrophy
Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
In some patients with Duchenne muscular dystrophy (DMD), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the dystrophin protein.
- Trial name
- NCT00264888
- Testing
- PTC124
- Where
- 3 sites in United States
- Volunteers
- 38 people
- Sponsor
- PTC Therapeutics
Velo-cardio-facial Syndrome, Psychosis
Metyrosine (Demser®) for the Treatment of Psychotic Disorders in Patients With Velocardiofacial Syndrome
This is an exploratory clinical investigation. The objectives of this study are to evaluate the safety, steady-state pharmacokinetics, and efficacy of metyrosine (Demser®) for the treatment of psychosis in patients with…
- Trial name
- NCT01127503
- Testing
- Metyrosine
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Bausch Health Americas, Inc.
Plagiocephaly, Nonsynostotic
Observe the Effects of the Serkel Cranial Remolding Orthosis on Infants With Deformational Plagiocephaly
The purpose of this study is to determine the safety and effectiveness of a specific brand of cranial remolding orthosis (a custom made helmet), which is new to the United States market
- Trial name
- NCT06831513
- Testing
- Serkel 3D CRO
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
Becker Muscular Dystrophy
Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
EDG-5506-203 MESA is an open-label extension study to assess the long-term effect of sevasemten (EDG-5506) on safety, biomarkers, and functional measures in adults and adolescents with Becker muscular dystrophy
- Trial name
- MESA (NCT06066580)
- Testing
- Sevasemten
- Where
- 48 sites in United States, Australia and 10 more
- Volunteers
- 260 people
- Sponsor
- Edgewise Therapeutics, Inc.
Clubfoot
Evertor Muscle Activity as a Predictor of Achilles Tenotomy in the Management of Idiopathic Varus Equinus Clubfoot
Clubfoot is an orthopedic congenital malformation of the lower limb of the newborn evaluated by the Dimeglio score. There is a deficit of muscular balance between the agonists and antagonists to the deformity.
- Trial name
- PBVE-Muscle (NCT05767762)
- Testing
- scale Idiopathic Clubfoot
- Where
- France
- Volunteers
- 50 people
- Sponsor
- Fondation Lenval
Craniosynostosis, Bleeding
Tranexamic Acid for Craniofacial Surgery
This is a randomized, blinded, prospective study that will investigate the potential benefit of tranexamic acid to reduce the intraoperative bleeding and blood transfusions in pediatric patients undergoing craniofacial surgeries.
- Trial name
- NCT00722436
- Testing
- Tranexamic acid, saline
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Pittsburgh
Plagiocephaly, Cranial Defect
Associations of Cranial Outcomes and Parental Expectations and Satisfaction
This study aims to investigate the association between objective cranial morphological changes and parental expectations and satisfaction in infants undergoing treatment with a cranial remolding orthosis (CRO).
- Conditions
- Plagiocephaly, Cranial Defect, Orthosis, Expectancy
- Trial name
- NCT07356401
- Testing
- Cranial orthosis
- Where
- Turkey (Türkiye)
- Volunteers
- 10 people
- Sponsor
- Gulhane School of Medicine
Plagiocephaly, Nonsynostotic
Cranial Orthotic Device Versus Repositioning Techniques for the Management of Plagiocephaly: the CRANIO Randomized Trial
This study evaluates the effectiveness of repositioning techniques and neck stretching exercises in comparison to cranial orthotic devices (COD) in correcting plagiocephaly in infants.
- Trial name
- CRANIO (NCT02370901)
- Testing
- Helmet, home therapies
- Where
- United States
- Volunteers
- 226 people, healthy volunteers welcome
- Sponsor
- Pat Ricalde,DDS, MD, FACS
Postoperative Pain
Prediction of Acute Postoperative Pain and Analgesic Consumption
Pain is an expected part of surgical recovery but effective pain management remains challenging. The high variability in postoperative pain experience and analgesic treatment response between patients is part of the challenge.
- Trial name
- MIRPEX-3 (NCT02230865)
- Where
- Denmark
- Volunteers
- 40 people
- Sponsor
- Kasper Grosen
Duchenne Muscular Dystrophy (DMD)
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
This Phase 2 clinical trial will evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of tegacorat oral solution in male participants 4 to less than 7 years of age with Duchenne muscular dystrophy…
- Trial name
- NCT07911995
- Testing
- Tegacorat
- Volunteers
- 36 people
- Sponsor
- Grünenthal GmbH
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