Clinical trials: Musculoskeletal Deformity, active
Haemophilia A (Moderate or Severe), Hemophilia, Classic
A Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis
The rationale for conducting this open-label phase 4 study is to assess whether once-weekly prophylaxis with efanesoctocog alfa (50 IU/kg) improves the disease course of existing synovial hypertrophy and prevents the risk of…
- Conditions
- Haemophilia A (Moderate or Severe), Hemophilia, Classic, Haemophilia, Hemophilia
- Trial name
- NCT06752850
- Testing
- Efanesoctocog alfa
- Where
- 10 sites in Italy, Norway and 2 more
- Volunteers
- 37 people
- Sponsor
- Swedish Orphan Biovitrum
Cranioplasty, Craniofacial Abnormalities
Stryker PEEK Customized Implant: A Prospective Post Market Follow Up to Evaluate Safety, Performance and Effectiveness
The purpose of this study is to confirm the safety, performance and effectiveness of Stryker's PEEK Customized Implants when used for the augmentation and/or restoration of bony and/or soft tissue deformities in the cranial and…
- Conditions
- Cranioplasty, Craniofacial Abnormalities, Craniofacial Injuries
- Trial name
- NCT05362370
- Testing
- Stryker's PEEK Customized Implant
- Where
- 13 sites in United States, Austria and 4 more
- Volunteers
- 99 people
- Sponsor
- Stryker Craniomaxillofacial
Pes Planus
The Effect of Orthosis and Exercise in Pes Planus
There is no definite evidence for the conservative treatment of pes planus. We want to verify the combination efficacy of orthosis and toe walking exercise for the treatment of pes planus.
- Trial name
- NCT03494374
- Testing
- UCBL orthosis
- Where
- South Korea
- Volunteers
- 200 people
- Sponsor
- Ju Seok Ryu
Myotonic Dystrophy Type 1 (DM1)
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1).
- Trial name
- ACHIEVE (NCT05481879)
- Testing
- DYNE-101
- Where
- 20 sites in United States, Australia and 6 more
- Volunteers
- 127 people
- Sponsor
- Dyne Therapeutics
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Scoliosis, Spine Deformity
Spine Surgery for Lenke 1 Adolescent Idiopathic Scoliosis
Idiopathic scoliosis of developmental age (AIS) is the most vertebral deformity in the adolescent population, with a prevalence of 1-3%.
- Conditions
- Scoliosis, Spine Deformity, Adolescent Idiopathic Scoliosis
- Trial name
- BS23 (NCT06396286)
- Testing
- single rod
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Istituto Ortopedico Rizzoli
Cervical Fusion
Clinical and Radiological Outcomes of Posterior Cervical Fusion Supplemented With Interfacet Spacers
This is a prospective study to assess fusion rates and cervical sagittal parameters following posterior cervical arthrodesis procedures supplemented with CIS.
- Trial name
- NCT04041583
- Testing
- Cervical Interfacet Spacers
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Ohio State University
Basal Cell Carcinoma, Locally Advanced Basal Cell Carcinoma
Oral Hedgehog Inhibitors in the Treatment of Basal Cell Carcinoma in the Netherlands: a Prospective Registration Study
Background: Oral hedgehog inhibitors vismodegib and sonidegib have been used for the treatment of locally advanced (laBCC), metastatic basal cell carcinoma (mBCC) and in basal cell nevus syndrome (BCNS) patients.
- Conditions
- Basal Cell Carcinoma, Locally Advanced Basal Cell Carcinoma, Metastatic Basal Cell Carcinoma, Gorlin Syndrome
- Trial name
- NCT05463757
- Testing
- Vismodegib, Sonidegib
- Where
- 8 sites in Netherlands
- Volunteers
- 80 people
- Sponsor
- Maastricht University Medical Center
Cleft Palate, Cleft Lip and Palate
Book Sharing for Toddlers With Clefts
Children with clefts exhibit difficulty with language and literacy compared to children without clefts. However, little is known about interventions to address these difficulties in the cleft population.
- Trial name
- BOOST (NCT06338319)
- Testing
- Book-Sharing for Toddlers with Clefts (BOOST)
- Where
- United States
- Volunteers
- 320 people
- Sponsor
- Seattle Children's Hospital
Muscular Dystrophies, Myopathies
Observational Study to Observe Variations of Gait Parameters in Patients With Neuromuscular Diseases
This study has the general objective of observing walking parameters during a clinical test to objectively estimate fatigue in patients with neuromuscular diseases.
- Conditions
- Muscular Dystrophies, Myopathies, Spinal Muscular Atrophy (SMA), Charcot Marie Tooth Disease
- Trial name
- NCT06666816
- Testing
- inapplicable
- Where
- 2 sites in Italy
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- IRCCS Eugenio Medea
Duchenne Muscular Dystrophy
AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD
This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT05683379
- Testing
- AAV8 DetectCDx
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- REGENXBIO Inc.
Mucopolysaccharidoses, Progressive Pseudorheumatoid Dysplasia
Pediatric Arthropathy Beyond Inflammation: Clinical Spectrum and Diagnostic Approach at Assiut University Children Hospital
To identify the most common underlying causes of non-inflammatory arthropathy in children presenting to Assiut University Children Hospital for through clinical evaluation, laboratory testing, and imaging, in order to improve…
- Conditions
- Mucopolysaccharidoses, Progressive Pseudorheumatoid Dysplasia, Farber Disease, Osteogenesis Imperfecta
- Trial name
- NCT07173010
- Volunteers
- 35 people
- Sponsor
- Assiut University
Gout, Inflammatory Rheumatism
Treat-to-target by Email During Urate-lowering Therapy in Gout
Gout is secondary to urate crystal deposition after chronic elevation of serum urate level (SUL). Long-term lowering SUL below 360 µmol/L allows dissolution of deposited crystals and disease cure.
- Conditions
- Gout, Inflammatory Rheumatism, Hyperuricemia, Posology Adjustment
- Trial name
- GOUTEmail (NCT04733079)
- Testing
- Cleanweb electronic messaging ePro, Usual follow-up
- Where
- 2 sites in France
- Volunteers
- 204 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Pectus Excavatum
Pectus Excavatum Camouflage (IT)
The clinical study evaluates the use of a custom-made medical-grade polycaprolactone-PCL Pectus scaffold implantation with Autologous Fat Grafting for pectus excavatum camouflage (IT).
- Trial name
- NCT05634070
- Testing
- PCL Pectus scaffold implantation and autologous fat grafting
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- BellaSeno GmbH
Osteogenesis Imperfecta
Audio-vestibular Evaluation of Children and Young Adults With Osteogenesis Imperfecta
The aim is to determine whether vestibular deficits are present in OI, then to establish whether a correlation exists between genetic type, severity of OI and audiovestibular phenotype.
- Trial name
- AVOI (NCT05419960)
- Testing
- Vestibular Assessment, Petrous bone Computed Tomography (CT), Magnetic Resonance Imaging (MRI)
- Where
- France
- Volunteers
- 44 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Flat Feet, Flexor Tendon Rupture
Medial Displacement Calcaneal Osteotomy and FDL- Transfer - With a Human, Allogeneic Cortical Bone Screw
The goal of this observational study is to compare the use of a screw made of human bone (Shark-Screw®, Surgebright-GmbH) with the metal/Bio-Tenodesis screw (Arthrex) in the treatment of the symptomatic flatfoot using the…
- Conditions
- Flat Feet, Flexor Tendon Rupture, Tibialis Posterior Dysfunction, Deformity, Foot
- Trial name
- NCT05643079
- Testing
- medializing calcaneal osteotomy and/or transfer of the flexor digitorum longus tendon (FDL) with metal/Bio-Tenodesis screws, medializing calcaneal osteotomy and/or transfer of the flexor digitorum longus tendon (FDL) with Shark Screws®
- Where
- Austria
- Volunteers
- 40 people
- Sponsor
- Orthopedic Hospital Vienna Speising
Postural Kyphosis, Spine
Reliability of PHAS in Postural Hyperkyphosis
This observational, cross-sectional study aims to evaluate the reliability of the Postural Habits and Awareness Scale (PHAS) in patients with postural hyperkyphosis.
- Conditions
- Postural Kyphosis, Spine, Assessment, Self
- Trial name
- NCT06907901
- Testing
- PHAS Assessment
- Where
- Turkey (Türkiye)
- Volunteers
- 128 people
- Sponsor
- Istanbul University - Cerrahpasa
Congenital Adrenal Hyperplasia, Hypogonadotropic Hypogonadism
Growing up With the Young Endocrine Support System (YESS!)
Transition from paediatric to adult endocrinology is a challenge for adolescents, families and doctors. Up to 25% of young adults with chronic endocrine disorders are lost to follow-up ('drop-out') once the young adult moves out…
- Conditions
- Congenital Adrenal Hyperplasia, Hypogonadotropic Hypogonadism, Growth Hormone Deficiency, Combined Pituitary Hormone Deficiency
- Trial name
- YESS (NCT04252001)
- Testing
- YESS! game, Snake '97, Transition-toolkit
- Where
- 7 sites in Belgium, Netherlands and 2 more
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- dr. Laura C. G. de Graaff-Herder
Cervical Deformity
Radiographic and Clinical Evaluation of Surgical Treatment for Cervical Deformity: A Multi-Center Study 2.0
Multi-center, prospective, non-randomized study to evaluate outcomes of surgically treated patients with adult cervical spinal deformity.
- Trial name
- PCD 2 (NCT04194996)
- Testing
- Surgical intervention
- Where
- 16 sites in United States
- Volunteers
- 200 people
- Sponsor
- International Spine Study Group Foundation
Maxillary Deficiency, Bone Resorption
Using Chin Bone Rings for Sinus Lifting and Dental Implants in One Surgery
This study evaluates the Bone Ring Technique, a single stage procedure that combines external sinus floor elevation and dental implant placement using an autogenous bone ring harvested from the chin.
- Trial name
- BRT-SINUS (NCT07619846)
- Testing
- Bone Ring Technique (BRT)
- Where
- Syria
- Volunteers
- 10 people
- Sponsor
- Damascus University