Clinical trials: Skeletal Abnormalities, active
Musculoskeletal Deformity, Musculoskeletal Abnormalities
Advanced Surgical Simulation Processes in the Correction of Skeletal Defects and Deformities
Virtual Surgical Planning (VSP), Computer-Aided Surgical Simulation (CASS) for bone corrections, and the customization of implants and devices through 3D printing, known as Patient-Specific Instruments (PSI) and Graft-Specific…
- Conditions
- Musculoskeletal Deformity, Musculoskeletal Abnormalities, Musculoskeletal Disorder
- Trial name
- SIMULA (NCT06380530)
- Testing
- Deformity correction
- Where
- 7 sites in Italy
- Volunteers
- 100 people
- Sponsor
- Istituto Ortopedico Rizzoli
Hypochondroplasia
Prospective Clinical Assessment Study in Children With Hypochondroplasia
This is a long-term, multicenter, non-interventional study of children ages 2.5 to <17 years with hypochondroplasia (HCH).
- Trial name
- HCH (NCT06410976)
- Where
- 25 sites in United States, Australia and 8 more
- Volunteers
- 150 people
- Sponsor
- QED Therapeutics, a BridgeBio company
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
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
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
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
Osteogenesis Imperfecta
Effects of Bisphosphonates on OI-Related Hearing Loss
Osteogenesis Imperfecta-related hearing loss usually occurs in individuals with mild (type I) OI and is much earlier in onset than age-related hearing loss, with the majority of individuals experiencing some minor hearing loss in…
- Trial name
- NCT04152551
- Testing
- Risedronate Oral Tablet
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Hospital for Special Surgery, New York
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
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
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
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
Fibrodysplasia Ossificans Progressiva
A Study of Andecaliximab in Participants With Fibrodysplasia Ossificans Progressiva (FOP)
This study is researching an experimental drug called andecaliximab. The study will include pediatric and adult patients with fibrodysplasia ossificans progressiva (FOP).
- Trial name
- ANDECAL (NCT06508021)
- Testing
- Andecaliximab
- Where
- 3 sites in United States
- Volunteers
- 92 people
- Sponsor
- Ashibio Inc
Hypochondroplasia
Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia
Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with…
- Trial name
- NCT07393373
- Testing
- Infigratinib
- Where
- 24 sites in United States, Australia and 8 more
- Volunteers
- 135 people
- Sponsor
- QED Therapeutics, a BridgeBio company
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
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, Hypochondroplasia
Prospective Longitudinal Multicentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia
The study aims to identify which Syde®-derived digital outcomes are reliable in FGFR3-related Skeletal Dysplasia. This requires to set-up a natural history study to measure limb movements in patients with ACH or HCH.
- Trial name
- SKY-MOT3 (NCT07388966)
- Where
- 4 sites in Belgium, France and 1 more
- Volunteers
- 140 people
- Sponsor
- SYSNAV
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
Cystic Fibrosis
Skeletal Health and Bone Marrow Composition in Adolescents With Cystic Fibrosis
The investigators will be evaluating bone marrow composition via magnetic resonance imaging in adolescents diagnosed with cystic fibrosis (CF) compared to healthy, matched controls.
- Trial name
- NCT06216704
- Testing
- Magnetic resonance relaxometry, Magnetic resonance spectroscopy, Blood Draw
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Idiopathic Short Stature
Multidisciplinary Evaluation and a Genome-wide Analysis in a Cohort of Idiopathic Short Stature Patients
This trial aims to evaluate the prevalence of idiopathic short stature among children whose growth is above -2,5SD (AFPA- CRESS/Inserm -CompuGroup Medical 2018 curve) or above -2SD of the parental target size (taking child gender…
- Trial name
- PAG PETI (NCT05858606)
- Testing
- Evaluation of the prevalence of truly (authentified) idiopathic short stature after multidisciplinary clinico-radiological evaluation, analysis in a multidisciplinary consultation meeting via a secure platform (ShareConfrère) for evaluation by multidisciplinary team, Whole genome analysis for authentified idiopathic short stature
- Where
- France
- Volunteers
- 200 people
- Sponsor
- University Hospital, Montpellier
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