Clinical trials: Skeletal Abnormalities, open to volunteers
Class II Malocclusion, Mandibular Deficiency
Dentoskeletal Effects of 3D-Printed Fixed Twin Block Versus MARA Appliances in Management of Skeletal Class II
Objective: to evaluate the dentoskeletal effects of CAD/CAM 3D-Printed fixed twin block versus MARA appliances in the management of skeletal class II due to Mandibular Deficiency in Preadolescence Class II malocclusion is…
- Trial name
- NCT07340242
- Testing
- MARA appliance, fixed twin block
- Where
- Egypt
- Volunteers
- 60 people
- Sponsor
- Mansoura University
Mucopolysaccharidosis IV Type A, Morquio A Syndrome
Non-invasive Functional Assessment and Pathogenesis of Morquio A
Morquio A disease is a devastating systemic skeletal disease in which detailed progression and pathogenesis remain unknown. The proposed project aims to establish a non-invasive objective assessment that can be applicable to all…
- Conditions
- Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
- Trial name
- NIFAMA (NCT05284006)
- Testing
- Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Nemours Children's Clinic
Cerebrovascular Stroke, Foot Drop
ENMS With Balance Feedback for Post-stroke Ankle-foot Rehabilitation
In this work, a novel hybrid ankle-foot robot is designed for gait rehabilitation after stroke, i.e., exoneuromusculoskeleton with balance sensing feedback (ENMS-BSF) by integrating the advantages of soft pneumatic muscle…
- Conditions
- Cerebrovascular Stroke, Foot Drop, Foot Inversion Deformity
- Trial name
- NCT04934787
- Testing
- ENMS-BSF assisted gait training
- Where
- China
- Volunteers
- 60 people
- Sponsor
- The Hong Kong Polytechnic University
Hypochondroplasia
An Interventional Study of Infigratinib in Children With Hypochondroplasia
ACCEL2/3 is a Phase 2/3 study. The purpose of the Phase 2 portion of the study (ACCEL2/3) is to evaluate the efficacy and safety of infigratinib in children with hypochondroplasia (HCH) receiving infigratinib at one of two doses…
- Trial name
- HCH (NCT06873035)
- Testing
- infigratinib 0.128 mg/kg/day, infigratinib 0.25 mg/kg/day
- Where
- 24 sites in United States, Australia and 8 more
- Volunteers
- 24 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Statin Adverse Reaction
Exercise Intensity and Muscle Recovery in Statin Users
This study aims to investigate how different types and intensities of exercise affect muscle recovery in individuals taking statin medications compared to healthy controls.
- Trial name
- STN-EX (NCT07563452)
- Testing
- Dose-Escalation Exercise Protocol
- Where
- Austria
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Raffaele Mazzolari
SEDC, Hypochondrogenesis
A Natural History Study in Children With a Type II Collagen Disorder With Short Stature
There are relatively few data available on type II collagen disorders, and evidence is lacking on the disease course in relation to symptoms and development of complications, the level of actual disease burden over time as well…
- Conditions
- SEDC, Hypochondrogenesis, Semd, Strudwick Type, Kniest Dysplasia
- Trial name
- ROCKET (NCT05408715)
- Testing
- Natural History Study
- Where
- 2 sites in France, Spain
- Volunteers
- 60 people
- Sponsor
- Innoskel
Fibrodysplasia Ossificans Progressiva, FOP
A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP)
This study is researching an experimental drug called garetosmab, referred to as "study drug". The study is focused on children and adolescent participants with FOP.
- Trial name
- OPTIMA-2 (NCT07559513)
- Testing
- garetosmab
- Volunteers
- 18 people
- Sponsor
- Regeneron Pharmaceuticals
Duchene Muscular Dystrophy, Becker Muscular Dystrophy
Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies
The purpose of this research study is to determine the potential of a multiparametric ultrasound approach to non-invasively monitor disease progression and to serve as an objective outcome measure for future clinical trials in…
- Trial name
- INNOVAN (NCT07378553)
- Where
- France
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Nantes University Hospital
Achondroplasia
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in participants with ACH who…
- Trial name
- NCT05145010
- Testing
- Infigratinib
- Where
- 31 sites in United States, Argentina and 8 more
- Volunteers
- 325 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Genetic Skeletal Diseases, Skeletal Dysplasia
Decoding the Genetic Landscape of Skeletal Diseases
This 5-year project aims to (1) search for genetic causes for yet unsolved congenital skeletal disorders (GSDs); (2) study consequences of the newly identified pathogenic variants in cells and in transgenic mice, (3) summarize…
- Conditions
- Genetic Skeletal Diseases, Skeletal Dysplasia, Molecular Causes, Skeletal Disorder
- Trial name
- SKDLAND (NCT05876416)
- Where
- Sweden
- Volunteers
- 450 people, healthy volunteers welcome
- Sponsor
- Karolinska Institutet
Hereditary Multiple Osteochondromas
Functional and Morphological Characterization of Multiple Osteochondromas Disorder
The main purpose of the study is the characterization of functional and morphological alterations due to multiple osteochondromas in a pediatric and adult population through the identification of relevant anthropometric and…
- Trial name
- MAMBO (NCT06703736)
- Where
- Italy
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Istituto Ortopedico Rizzoli
Skeletal Class II Malocclusion, Skeletal Class III Malocclusion
Clinical and Radiographic Evaluation of Low Sagittal Medial Ramus Osteotomy for Treatment of Skeletal Class II and Class III Malocclusion
This prospective clinical trial aims to evaluate the clinical and radiographic outcomes of the low sagittal medial ramus osteotomy-a technical modification of the conventional sagittal split osteotomy (SSO)-for treating patients…
- Conditions
- Skeletal Class II Malocclusion, Skeletal Class III Malocclusion, Dentofacial Deformities
- Trial name
- NCT07746700
- Testing
- Low Sagittal Medial Ramus Osteotomy
- Volunteers
- 11 people
- Sponsor
- Al-Azhar University
Achondroplasia
Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants < 3 years old with ACH.
- Trial name
- NCT07169279
- Testing
- Infigratinib is provided as a single dose of minitablets for oral administration, Infigratinib is provided as sprinkle capsules for daily oral administration
- Where
- 12 sites in United States, Australia and 5 more
- Volunteers
- 77 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Achondroplasia
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
This is a multicenter, multinational, randomized, active-controlled, operationally seamless Phase 2/3 study of BMN 333 in treatment-naïve pediatric participants with achondroplasia (ACH).
- Trial name
- ASPEN (NCT07441876)
- Testing
- BMN 333, Vosoritide Injection [Voxzogo]
- Where
- 28 sites in United States, Australia and 7 more
- Volunteers
- 160 people
- Sponsor
- BioMarin Pharmaceutical
Polyneuropathy, Charcot Marie Tooth Disease (CMT)
Effects of Resistance Exercises in Hereditary Sensory-Motor Neuropathy (Charcot-Marie-Tooth Disease)
The goal of this clinical trial is to compare the effects of an 8-week resistance exercise training program on skeletal muscle quality, functional capacity, and quality of life in young individuals aged 15 to 29 years, with and…
- Trial name
- PNP (NCT07152197)
- Testing
- Progressive Resistance Exercise Training
- Where
- 2 sites in Chile
- Volunteers
- 22 people, healthy volunteers welcome
- Sponsor
- Universidad de La Frontera
Musculoskeletal Abnormalities, Obesity
GLP-1R Actions on Muscle and the Skeleton
The GRAMS study objectives are to assess the musculoskeletal changes that occur after weight loss using GLP-1 based therapy. A lifestyle intervention with diet and exercise is included to assess any mitigating effects are…
- Conditions
- Musculoskeletal Abnormalities, Obesity, Sarcopenic Obesity, Osteoporosis
- Trial name
- GRAMS (NCT07154719)
- Testing
- Tirzepatide, Lifestyle toolkit
- Where
- 2 sites in United States
- Volunteers
- 50 people
- Sponsor
- Pennington Biomedical Research Center
Gaucher Disease
Advancing Integrated Therapies for Gaucher Disease
The purpose of this study is to better understand the natural history, clinical outcomes, and biological features of Gaucher disease in patients receiving standard medical care
- Trial name
- NCT07758816
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Yale University
Skeletal Deformities, Orthognathic Surgical Procedures
Impact of 3D-Printed Anatomical Model on Patient Understanding in Orthognathic Surgery
The goal of this clinical trial is to learn whether using three dimensional (3D)-printed anatomical models improves the informed consent process for participants undergoing orthognathic surgery.
- Conditions
- Skeletal Deformities, Orthognathic Surgical Procedures, Anxiety, Patient Education
- Trial name
- NCT07068412
- Testing
- 3D-printed Orthognathic Surgical Simulation, 2D Visual Representations of Skeletal Movements in Orthognathic Surgery
- Where
- 2 sites in Saudi Arabia
- Volunteers
- 60 people
- Sponsor
- King Abdulaziz University
Duchenne Muscular Dystrophy (DMD)
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
The primary objective of this study is to evaluate the safety and tolerability of SPOT-03 administered by intravenous (IV) infusion to DMD patients.
- Trial name
- NCT07188012
- Testing
- SPOT-03
- Where
- China
- Volunteers
- 9 people
- Sponsor
- Shanghai Siponuoyin Biotechnology Co Ltd
Dentofacial Abnormalities, Dentofacial Deformities
Orthognathic Speech Pathology: Phonetic Contrasts of Patients With Dental Discrepancies Pre- and Post-Treatment Analyses
The investigators are studying how speech is effected by jaw and tooth position in jaw surgery patients. Eighty percent of our jaw surgery patients have speech pathologies, compared to five percent of the general population, but…
- Conditions
- Dentofacial Abnormalities, Dentofacial Deformities, Dentofacial Anomalies, Including Malocclusion, Malocclusion
- Trial name
- OSP (NCT04117360)
- Testing
- Orthognathic surgery and Orthodontics
- Where
- United States
- Volunteers
- 180 people
- Sponsor
- University of North Carolina, Chapel Hill