Clinical trials: Musculoskeletal Abnormalities, open to volunteers
Amputation, Congenital, Upper Limb; Amelia
CNS Changes Following Upper Limb Loss
The objective of this study is to better understand the structural and functional changes that the central nervous system (CNS) undergoes following congenital upper limb loss.
- Trial name
- NCT06043518
- Testing
- MRI
- Where
- Switzerland
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- University of Zurich
Hypoparathyroidism, Pseudohypoparathyroidism
German Hypoparathyroidism Registry
Chronic hypoparathyroidism is a rare condition in which the parathyroid glands do not make enough parathyroid hormone (PTH). PTH helps control the levels of calcium and phosphate in the blood.
- Trial name
- HyPaReg (NCT07857460)
- Where
- Germany
- Volunteers
- 150 people
- Sponsor
- University Hospital Augsburg
Dentofacial Deformities
Patient-Specific vs. Conventional Positioning in Sagittal Split Ramus Osteotomy
This study aims to evaluate the clinical efficacy and positioning accuracy of a custom-made proximal segment positioning appliance designed for use in sagittal split ramus osteotomy (SSRO).
- Trial name
- NCT07125963
- Testing
- Custom-made positioning appliance-assisted mandibular segment repositioning, Conventional Manual Positioning
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Sümer Münevveroğlu
Myopathy, Muscular Dystrophies
Muscle Health Measurements Using Electrical Impedance Myography
This study is being done to further develop a device, the mScan, to measure muscle health as compared to measurements of muscle health using MRI (magnetic resonance imaging).
- Conditions
- Myopathy, Muscular Dystrophies, Myositis, Myofibrillar Myopathy
- Trial name
- NCT07502989
- Testing
- Electrical Impedance Myography
- Where
- United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Beth Israel Deaconess Medical Center
Steinert's Disease, Myotonic Dystrophy 1
Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)
The study team hypothesize that non-diabetic patients with Myotonic dystrophy type I (DM1) will improve their symptoms, especially their motor deficit which is the main feature of the disease, because of the splicing defect…
- Conditions
- Steinert's Disease, Myotonic Dystrophy 1, Metformin
- Trial name
- METFORMYO (NCT05532813)
- Testing
- Treatment taken
- Where
- France
- Volunteers
- 142 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)
This is a parallel arm non-randomized dose-escalation, open-label basket exploratory phase 1 clinical trial where Mitochondrial encephalopathy, lactic acidosis, stroke-like episodes (MELAS) and Leber's hereditary optic…
- Trial name
- NCT06792500
- Testing
- Glycerol Tributyrate
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- George Washington University
Myotonic Dystrophy 1
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared…
- Trial name
- FREEDOM2-DM1 (NCT06667453)
- Testing
- PGN-EDODM1
- Where
- 12 sites in Canada, New Zealand and 2 more
- Volunteers
- 24 people
- Sponsor
- PepGen Inc
Facioscapulohumeral Muscular Dystrophy, FSHD
A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)
- Conditions
- Facioscapulohumeral Muscular Dystrophy, FSHD, FSHD - Facioscapulohumeral Muscular Dystrophy, FSHD1
- Trial name
- FORTITUDE-3 (NCT07038200)
- Testing
- AOC-1020
- Where
- 46 sites in United States, Canada and 8 more
- Volunteers
- 200 people
- Sponsor
- Avidity Biosciences, Inc.
Lymphatic Malformation, Generalized Lymphatic Anomaly (GLA)
Lymphatic Anomalies Registry for the Assessment of Outcome Data
Lymphatic anomalies are a rare subset of vascular anomalies that are poorly understood. the understanding of the natural history, long-term outcomes, risk factors for morbidity and mortality, and the relative benefit of medical…
- Conditions
- Lymphatic Malformation, Generalized Lymphatic Anomaly (GLA), Central Conducting Lymphatic Anomaly, CLOVES Syndrome
- Trial name
- NCT02399527
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Boston Children's Hospital
Trachea Diseases, Tracheal Stenosis
Feasibility of Tracheobronchial Reconstruction Using Allogenic Aortic Patch in Children
Feasibility and safety of repairing tracheal and bronchial defects in infants and children using cryopreserved donor aortic patches.
- Conditions
- Trachea Diseases, Tracheal Stenosis, Tuberculosis; Tracheitis, Airway Disease
- Trial name
- NCT07240259
- Testing
- Cryopreserved aorta
- Where
- Taiwan
- Volunteers
- 5 people
- Sponsor
- National Taiwan University Hospital
Vascular Anomaly, Generalized Lymphatic Anomaly
Identification of Biomarkers for Patients with Vascular Anomalies
The study will use blood (serum and plasma) and tissue obtained from participants undergoing prescribed surgical resection of vascular anomalies of interest proposed in this study.
- Conditions
- Vascular Anomaly, Generalized Lymphatic Anomaly, Kaposiform Hemangioendothelioma, Kaposiform Lymphangiomatosis
- Trial name
- NCT03001180
- Where
- 2 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Multiple Osteochondromas
Registry of Multiple Osteochondromas
REM is a retrospective and prospective registry, finalized for care and research purposes. It is articulated in main sections - strongly related and mutually dependent on each other - corresponding to different data domains…
- Trial name
- REM (NCT04133285)
- Where
- Italy
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- Luca Sangiorgi
Pseudohypoparathyroidism Type 1a, Obesity
Setmelanotide to Treat Obesity in a Patient With Pseudohypoparathyroidism Type 1a (PHP1a)
The investigators plan to test the efficacy and safey of 6-months of open-label setmelanotide to treat obesity in a single patient with pseudohypoparathyroidism type 1a due to a GNAS mutation.
- Trial name
- NCT07496463
- Testing
- Setmelanotide
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Massachusetts General Hospital
Myotonic Dystrophy Type 2, Myotonic Dystrophy Type 1
Brain Structure and Clinical Endpoints in Myotonic Dystrophy Type 2
Nearly two-third of patients with myotonic dystrophy type 2 (DM2) report that impaired cognition is among the most disabling symptoms and deeply affects their quality of life.
- Trial name
- BraCE-DM2 (NCT05854433)
- Testing
- Non-interventional study
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Wake Forest University Health Sciences
Cleft Palate, Unilateral Cleft Palate
Palatoplasty Techniques Using PRF and Gentamicin-Loaded PRF in Cleft Palate
This prospective, randomized controlled trial evaluates the clinical efficacy of autologous Platelet-Rich Fibrin (PRF) and Gentamicin-Loaded PRF as regenerative and antimicrobial biological adjuncts in primary palatoplasty.
- Conditions
- Cleft Palate, Unilateral Cleft Palate, Bilateral Cleft Palate, Complete Cleft Palat
- Trial name
- NCT07752849
- Testing
- Standard palatoplasty ( control), Platelet _ Rich Fibrin ( PRF), gentamicin_ loaded PRF
- Where
- Yemen
- Volunteers
- 90 people
- Sponsor
- Al-Thawra Modern General Hospital
Pectus Carinatum
Prognosis Factors for the Treatment of Pectus Carinatum With a Dynamic Compression System
Pectus carinatum is a deformation of the thoracic wall causing an aesthetic prejudice. Since 2011, our team uses a dynamic compression system to treat this deformation.
- Trial name
- Pectusdyn (NCT04452721)
- Testing
- dynamic compression system
- Where
- France
- Volunteers
- 520 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Duchenne Muscular Dystrophy
The U.K. NorthStar Clinical Network
The goal of this natural history study is to capture the natural history of Duchenne Muscular Dystrophy (DMD) in children and adults in the United Kingdom. Children and adults with DMD will be invited to join.
- Trial name
- NS (NCT06711692)
- Where
- 26 sites in United Kingdom
- Volunteers
- 300 people
- Sponsor
- University College, London
Neurological Regression, Myoclonus
Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
Study description: This is a natural history study that will evaluate any patient with enzyme or DNA confirmed GM1 or GM2 gangliosidosis, sialidosis or galactosialidosis.
- Conditions
- Neurological Regression, Myoclonus, Cherry Red Spot, Brain Atrophy
- Trial name
- NCT00029965
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Pectus Excavatum, Chest Deformities
SPSIPB Versus SAPB for Postoperative Analgesia After Minimally Invasive Repair of Pectus Excavatum
This prospective observational cohort study will compare postoperative analgesic outcomes in patients undergoing minimally invasive repair of pectus excavatum.
- Trial name
- NCT07697222
- Testing
- Postoperative Analgesia Protocol, Bispectral Index Monitoring, Serratus Posterior Superior Intercostal Plane Block
- Where
- Turkey (Türkiye)
- Volunteers
- 80 people
- Sponsor
- Marmara University
Duchenne Muscular Dystrophy (DMD)
Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
The purpose of this study is to evaluate the safety, tolerability, and efficacy of LE051 intravenous therapy in DMD patients treated with exon 51 skipping therapy.
- Trial name
- NCT06900049
- Testing
- LE051
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Shanghai Jiao Tong University School of Medicine