Clinical trials: Musculoskeletal Abnormalities
Myotonic Dystrophy 1, DM1
The Spanish National Registry for Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1) is a rare genetic neuromuscular condition that can affect multiple organs and varies widely in how it presents.
- Conditions
- Myotonic Dystrophy 1, DM1, Myotonic Dystrophy Type 1, Myotonic Dystrophy, Congenital
- Trial name
- DM1-Hub (NCT07385443)
- Testing
- Patient Registry
- Where
- 8 sites in Spain
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Fundació Institut Germans Trias i Pujol
Funnel Chest
Cardiorespiratory Assessment and Quality of Life After Surgical Correction of Congenital Wall Malformations
The main objective of this study is to characterize the cardiorespiratory functional gain in patients with pectus excavatum following corrective surgery.
- Trial name
- HeartSoar (NCT03770390)
- Testing
- Surgical correction of pectus excavatum
- Where
- France
- Volunteers
- 43 people
- Sponsor
- University Hospital, Montpellier
Growth Disorders
Treatment of Children and Adolescents With Growth Failure Associated With Primary IGF-1 Deficiency
This is an extension study to Tercica study MS301 (NCT00125164) and is intended to collect long term safety and efficacy data on the continued use of recombinant human insulin-like growth factor-1 (rh IGF-1) in children and…
- Trial name
- NCT00330668
- Testing
- rh IGF-1 (mecasermin)
- Where
- France
- Volunteers
- 114 people
- Sponsor
- Ipsen
Gastroschisis, Bowel Obstruction
The Use of Gastrografin to Help Alleviate Bowel Obstruction in Gastroschisis Patients.
This study will investigate the use of a drug called Gastrografin to aid in bowel mobility for paediatric patients who have undergone gastroschisis surgery.
- Conditions
- Gastroschisis, Bowel Obstruction, Birth Defect
- Trial name
- NCT03334578
- Testing
- Gastrografin
- Where
- Canada
- Sponsor
- London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's
Thoracic Aorta Dilatation, Marfan Syndrome
Thoracic Aortic Dilatation Syndromes
Aortic dilatation syndromes are comprised by a group of different syndromes, of which Marfan syndrome is the best described. Many of the aorta dilatation associated syndromes are heritable connective tissue disorders but some…
- Trial name
- NCT02111668
- Where
- Denmark
- Volunteers
- 120 people
- Sponsor
- University of Aarhus
Marfan Syndrome
The Oxford Marfan Trial
The primary objective of the trial is to estimate the effects of allocation to irbesartan, or doxycycline, or a combination of both irbesartan and doxycycline, compared with placebo, on measures of elastic function of the aorta…
- Trial name
- NCT01949233
- Testing
- Irbesartan 150-300mg capsules daily for 6 months, Doxycycline 100-200mg capsules daily for 6 months
- Where
- 2 sites in United Kingdom
- Volunteers
- 56 people
- Sponsor
- University of Oxford
Duchenne Muscular Dystrophy
Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy
Researchers at Children's Hospital Boston Neurology Department invite children to participate in a new research study. Researchers are looking for boys ages 2 - 30 with Duchenne Muscular Dystrophy (DMD) and healthy boys ages 2 …
- Trial name
- QED (NCT01491555)
- Where
- United States
- Volunteers
- 73 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Clubfoot
Clubfoot DNA Repository
To build a DNA repository to enable participation in ongoing and future Clubfoot genetic linkage studies.
- Trial name
- NCT00607191
- Where
- United States
- Volunteers
- 379 people
- Sponsor
- Texas Scottish Rite Hospital for Children
X-Linked Hypophosphatemia
Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
The primary objectives of the study are to: Establish the safety profile of KRN23 for the treatment of XLH in children between 1 and 4 years old Determine the PD effects of KRN23 treatment on serum phosphorus and other PD markers…
- Trial name
- NCT02750618
- Testing
- Burosumab
- Where
- 3 sites in United States
- Volunteers
- 13 people
- Sponsor
- Kyowa Kirin, Inc.
Patellar Dislocation, Anatomic Abnormality
The Influence of Anatomic Deformities on the Clinical Efficacy in the Patients With Patellar Dislocation
All the data of patients with patellar dislocation will be collected to evaluate the anatomic deformities. The data includes X-ray, CT, MRI examinations, medical records, and physical examination information.
- Conditions
- Patellar Dislocation, Anatomic Abnormality, Trochlear Dysplasia, Femoral Anteversion
- Trial name
- NCT05243134
- Testing
- Collect the clinical data
- Where
- China
- Volunteers
- 1,000 people
- Sponsor
- Peking University Third Hospital
Sarcoidosis, Tuberculosis
The Serum Angiotensin Converting Enzyme and Lysozyme Levels in Patients Non-infectious and Infectious Uveitis
This study evaluates the significance of differences in serum angiotensin converting enzyme and lysozyme levels of patients with ocular involvement of other autoimmune inflammatory and infectious diseases.
- Trial name
- uveitis (NCT02627209)
- Testing
- spectrophotometric assay, radial immunodiffusion
- Where
- Turkey (Türkiye)
- Volunteers
- 280 people
- Sponsor
- Ozlem Sahin
Developmental Dysplasia of Hip, DDH
8 vs 12 Weeks: A Pavlik Harness Trial
Developmental dysplasia of the hip is the most common bone and joint condition in babies. It is usually treated with a harness that holds the hips in a safe position so they can develop normally.
- Conditions
- Developmental Dysplasia of Hip, DDH, Developmental Dysplasia of the Hip, Developmental Dysplasia of the Hip (DDH)
- Trial name
- NCT07847619
- Testing
- Pavlik Harness
- Where
- Canada
- Volunteers
- 110 people
- Sponsor
- The Hospital for Sick Children
Exostoses, Multiple Hereditary
Height, Ulnar Length and Forearm Function in Multiple Hereditary Exostoses
the purpose of the present registry is to describe the epidemiology of forearm deformities in patients with Hereditary Multiple Exostoses and to identify, independent predictors of severity of the disease and potential…
- Trial name
- NCT05914298
- Testing
- blood and buccal swab genetic test, PUL, Range of motion
- Where
- Italy
- Volunteers
- 408 people, healthy volunteers welcome
- Sponsor
- Istituto Ortopedico Rizzoli
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
Cerebral Palsy, Spina Bifida
The Effectiveness of Participation-focused Interventions on Body Functions of Youth With Physical Disabilities
Youth with physical disabilities face greater restrictions to participation in community-based activities than their typically developing peers, which can lead to poor health outcomes.
- Conditions
- Cerebral Palsy, Spina Bifida, Muscular Dystrophies, Musculoskeletal Disorder
- Trial name
- NCT03851107
- Testing
- Engagement in 6-week community-based activity program
- Where
- Canada
- Volunteers
- 8 people
- Sponsor
- McGill University
Duchenne Muscular Dystrophy
Comparative Study of Clinical Endpoint in DMD: Handheld Myometry (HHM) Versus CINRG Quantitative Measurement System (CQMS)
The aim of the proposed research is to compare two commonly used pediatric strength testing measures: handheld myometry (HHM) and CINRG Quantitative Measurement System (CQMS), with the goal of identifying a sensitive and valid…
- Trial name
- NCT01125709
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 30 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Muscular Dystrophy, Facioscapulohumeral
Disease Burden and Living Situation of Patients With Facioscapulohumeral Muscular Dystrophy
The goal of this observational study is to learn the patient journey, disease burden, living situation, quality of life and etc. in patients with Facioscapulohumeral Muscular Dystrophy in all sex/gender and age groups.
- Trial name
- NCT06517498
- Testing
- No Intervention
- Where
- China
- Volunteers
- 300 people
- Sponsor
- Xi'an Jiaotong University
Clubfoot
Comparison of Surgical Blade No. 11 and Ophthalmic Knife in Idiopathic Clubfoot Patients
There is limited local data available on the comparison between surgical blade no.11 and ophthalmic knife among patients with neglected clubfoot going through tenotomy.
- Trial name
- NCT07561736
- Testing
- Surgical Blade, Ophthalmic Knife
- Where
- Pakistan
- Volunteers
- 102 people
- Sponsor
- Muhammad Aamir Latif
Muscular Dystrophy
Effect of Aquatic Therapy on Pulmonary Functions in Patients With Muscular Dystrophy
This study aimed to assess the efficacy of aquatic therapy on pulmonary functions in patients with muscular dystrophy.
- Trial name
- NCT04574934
- Testing
- aquatic therapy
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- South Valley University
Duchenne Muscular Dystrophy
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KER-065 administered to adult and pediatric ambulatory and nonambulatory male participants with…
- Trial name
- NCT07704099
- Testing
- KER-065
- Where
- 2 sites in New Zealand
- Volunteers
- 36 people
- Sponsor
- Keros Therapeutics, Inc.
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