Clinical trials: Musculoskeletal Deformity
Muscular Dystrophy, Muscular Disease
Studying Skeletal Muscle, Heart, and Diaphragm Imaging in Boys With Duchenne Muscular Dystrophy
Background: Duchenne muscular dystrophy (DMD) is a disease in which the muscles are unable to make the protein dystrophin. Without this protein, the muscles become gradually weaker.
- Trial name
- NCT01451281
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Myotubular Myopathy
Myotubular Myopathy Genetic Testing Study
Myotubular myopathy (XLMTM) is an X-linked disorder caused by mutations in the myotubularin gene (MTM1). The clinical spectrum is variable and ranges from individuals who require a wheelchair and full time breathing support to…
- Trial name
- NCT01817946
- Testing
- Genetic Testing
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Cure CMD
Schizophrenia, Micrognathia
The Relevance Between the microRNA-30e in Plasma and the Prognosis of Schizophrenia Patients
This study investigates the relationship of circulating microRNA-30e and schizophrenia, and shows the relevance of the aberrant microRNA-30e expression in plasma with the variation disease status.
- Trial name
- NCT03007303
- Testing
- Atypical Antipsychotic, atypical antipsychotic combined with MECT
- Where
- China
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Dalian Seventh People's Hospital
Osteogenesis Imperfecta
Adapted Physical Activity Program (APA) for Effort Rehabilitation of Children and Teenagers With Osteogenesis Imperfecta
This research study aims to evaluate the effect of a 6-month adapted physical activity program (APA) on the endurance capacities (evaluated as the maximum oxygen consumption [VO2 peak]) of children and adolescents with…
- Trial name
- BONeMOVE (NCT05927389)
- Testing
- Adapted Physical Activity program
- Where
- 2 sites in France
- Volunteers
- 30 people
- Sponsor
- University Hospital, Toulouse
Duchenne Muscular Dystrophy
A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy (LYNX)
The LYNX study is a 2-part, multicenter, Phase 2 study of safety, pharmacokinetics and biomarkers in children with Duchenne muscular dystrophy including a randomized, double-blind, placebo-controlled part A, followed by an…
- Trial name
- NCT05540860
- Testing
- Sevasemten Dose 1, Sevasemten Dose 2, Sevasemten Dose 3
- Where
- 14 sites in United States
- Volunteers
- 76 people
- Sponsor
- Edgewise Therapeutics, Inc.
Flat Feet
Plyometric Exercise With Obese Children
Obesity is one of the main factors that contribute to an acquired flat foot deformity which in turn impairs the balance strategies.
- Trial name
- NCT05026294
- Testing
- plyometric exercises, Exercises for flat feet rehabilitation
- Where
- Saudi Arabia
- Volunteers
- 47 people
- Sponsor
- Taif University
Type 2 Spinal Muscular Atrophy
Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)
This was a Phase III multi-center, single dose (1.2 x 10^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never…
- Trial name
- STEER (NCT05089656)
- Testing
- OAV101
- Where
- 42 sites in United States, Brazil and 11 more
- Volunteers
- 126 people
- Sponsor
- Novartis Pharmaceuticals
X-linked Hypophosphatemia
Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)
The objectives of the study are to: Identify a dose and dosing regimen of burosumab, based on safety and pharmacodynamic (PD) effect, in pediatric XLH participants Establish the safety profile of burosumab for the treatment of…
- Trial name
- NCT02163577
- Testing
- burosumab
- Where
- 9 sites in United States, France and 2 more
- Volunteers
- 52 people
- Sponsor
- Kyowa Kirin, Inc.
Intellectual Disability, Rubinstein-Taybi Syndrome
Functional Tests to Resolve Unsolved Rare Diseases. Rares.
Rares diseases are a heterogeneous group of conditions which need important tools for diagnosis. The use of high-throughput sequencing is able to diagnose half of the patients.
- Conditions
- Intellectual Disability, Rubinstein-Taybi Syndrome, Cystic Fibrosis, Congenital Heart Defect
- Trial name
- RID (NCT05696912)
- Testing
- Ex-vivo approach concerning 25 patients, In-vitro approach concerning 25 patients
- Where
- France
- Volunteers
- 50 people
- Sponsor
- University Hospital, Bordeaux
Cleft Palate
Effect of Furlow Palatoplasty With Buccal Myomucosal Flap on the Speech Development
About 20 patient will be collected according to the eligibility criteria and grouped into Group I : 10 patients were treated with furlow with buccinators flap Group II : 10 patients were treated with 2 flap palatoplasty All…
- Trial name
- NCT06856330
- Testing
- furlow with buccal myomucosal flap, two flap palatoplasty
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- Fayoum University
Congenital Talipes Equinovarus
Customized Orthosis for Children With Clubfoot
This study will examine the use of a novel customized Ankle Foot Orthosis (AFO), created using 3D scanning, with children being treated for clubfoot.
- Trial name
- NCT03853811
- Testing
- Ankle Foot Orthosis (AFO)
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Medical College of Wisconsin
Muscular Dystrophies
A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
This is a phase III, multicenter, open-label, uncontrolled extension study in male subjects with DMD open to eligible US and Canadian subjects who previously participated in the following studies of drisapersen: DMD114876…
- Trial name
- NCT01803412
- Testing
- Drisapersen
- Where
- 13 sites in United States, Canada
- Volunteers
- 53 people
- Sponsor
- BioMarin Pharmaceutical
Growth Hormone Deficiency, Turner Syndrome
Long-term Safety and Effectiveness of Growth Hormone With GHD, TS, CRF, SGA , ISS and PWS in Children
The purpose of this study is to evaluate the long-term safety and effectiveness of growth hormone (Eutropin Inj./Eutropin plus Inj.) treatment with GHD (Growth Hormone Deficiency), TS (Turner Syndrome),CRF (Chronic Renal…
- Conditions
- Growth Hormone Deficiency, Turner Syndrome, Chronic Renal Failure, Small for Gestational Age
- Trial name
- LGS (NCT01604395)
- Where
- South Korea
- Volunteers
- 6,000 people
- Sponsor
- LG Chem
Craniofacial Abnormalities
Mandibular Slotplates
A new osteosynthesis system for orthognathic surgery was proposed.This system allows small intra-operative adjustments of the bone fragments during the osteosynthesis phase of the operation (also known as the slot principle).
- Trial name
- NCT03679377
- Testing
- Mandibular slotplate
- Where
- Belgium
- Volunteers
- 9 people
- Sponsor
- Universitair Ziekenhuis Brussel
Impaired Synaptic Plasticity, Impaired Cognition
Synaptic Plasticity and Cognitive Function in RASopathies
The project is targeting cognitive impairment, one of the main health problems of patients with RAS pathway disorders. The aim of this study is to translate findings of animal studies to humans.
- Trial name
- SynCoRAS (NCT03504501)
- Testing
- Lovastatin, Lamotrigine
- Where
- Germany
- Volunteers
- 16 people
- Sponsor
- Technical University of Munich
Duchenne Muscular Dystrophy
Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy
The proposed clinical trial is an outgrowth of the safety record and functional improvement seen in the BMD follistatin gene therapy trial.
- Trial name
- NCT02354781
- Testing
- rAAV1.CMV.huFollistin344
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Jerry R. Mendell
Clubfoot Deformity, Forefoot Adductus
Outcomes of Eight-Plate Epiphysiodesis for Residual Clubfoot Deformities
Purpose: The purpose of this prospective clinical study is to evaluate the radiographic outcomes, effectiveness, and safety of lateral calcaneocuboid guided growth using an extra-periosteal tension-band plate (eight-plate) to…
- Trial name
- clubfoot (NCT07621328)
- Testing
- Lateral Calcaneocuboid Guided Growth (Eight-Plating)
- Where
- Egypt
- Volunteers
- 15 people
- Sponsor
- Cairo University
Duchenne Muscular Dystrophy (DMD)
Sodium/Glucose Cotransporter-2 Inhibitors (SGLT2i) Therapy in Duchenne Cardiomyopathy
This is a pharmacokinetic study (PK Study) to better understand empagliflozin dosing in pediatric Duchenne muscular dystrophy patients.
- Trial name
- CHERISH (NCT07172971)
- Testing
- SGLT-2 inhibitor, SGLT2 inhibitor
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Vanderbilt University Medical Center
Duchenne Muscular Dystrophy
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
The purpose of this study is to provide a single clinical study with a uniform approach to monitoring long-term safety and efficacy in participants who received delandistrogene moxeparvovec in a previous clinical study.
- Trial name
- EXPEDITION (NCT05967351)
- Testing
- delandistrogene moxeparvovec
- Where
- 38 sites in United States, Belgium and 7 more
- Volunteers
- 400 people
- Sponsor
- Sarepta Therapeutics, Inc.
Nonsynostotic Plagiocephaly
Brain Characteristics Noted Prior to and Following Treatment
This study will enable investigators to find out if brain structure and characteristics are affected by the shape of the infant's head, and if changes in the brain occur with helmet therapy.
- Trial name
- NCT02392195
- Testing
- Non-interventional
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.