Clinical trials: Musculoskeletal Abnormalities
Gorlin Syndrome
Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin Syndrome
The aim of this clinical study is to find out how well Patidegib Gel 2% works in preventing new basal cell carcinomas (BCCs) developing on the face of adults with Gorlin syndrome, and how safe Patidegib Gel 2% is to use.
- Trial name
- NCT06050122
- Testing
- Patidegib Topical Gel, Patidegib Topical Gel with no active patidegib
- Where
- 40 sites in United States, France and 5 more
- Volunteers
- 113 people
- Sponsor
- Sol-Gel Technologies, Ltd.
Muscular Dystrophy, Duchenne
Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
This study is being performed as a post-approval safety study (PASS), per the Pharmacovigilance Risk Assessment Committee (PRAC) of the European Medicines Agency (EMA), to gather data on Translarna (ataluren) safety…
- Trial name
- NCT02369731
- Where
- 71 sites in Austria, Brazil and 14 more
- Volunteers
- 316 people
- Sponsor
- PTC Therapeutics
Paralysis, Hyperkalemic Periodic, Hypokalemic Periodic Paralysis
Phase III Randomized, Double-Blind, Placebo-Controlled Study of Dichlorphenamide for Periodic Paralyses and Associated Sodium Channel Disorders
OBJECTIVES: I. Assess the efficacy of dichlorphenamide in the treatment of episodic weakness attacks in patients with hyperkalemic periodic paralysis, paramyotonia congenita with periodic paralysis, and hypokalemic periodic…
- Conditions
- Paralysis, Hyperkalemic Periodic, Hypokalemic Periodic Paralysis, Paramyotonia Congenita
- Trial name
- NCT00004802
- Testing
- dichlorphenamide
- Volunteers
- 64 people
- Sponsor
- National Center for Research Resources (NCRR)
Multiple Osteochondroma, Enchondromatosis
Survey on Multiple Osteochondromas and Enchondromatoses During the Transition From Paediatric Care to Adult Care
This prospective observational study aims to understand the transition experiences and unmet needs of patients living with multiple osteochondromas or enchondromatoses during the paediatric to adult care, in order to produce a…
- Trial name
- PJMOE (NCT07839637)
- Where
- Italy
- Volunteers
- 91 people, healthy volunteers welcome
- Sponsor
- Istituto Ortopedico Rizzoli
Fibromyalgia, Behcet Syndrome
Frequency of Fibromyalgia in Behcet Disease
In various studies, the incidence of fibromyalgia in Behçet's patients is widely distributed. It was aimed to investigate the frequency and severity of fibromyalgia in Behcet disease in province Bursa.
- Trial name
- NCT03514056
- Testing
- group 1:Patients diagnosed with Behcet disease, group 2: Patient diagnosed with fibromyalgia without Behcet disease
- Where
- Turkey (Türkiye)
- Volunteers
- 146 people
- Sponsor
- Bursa Yuksek Ihtisas Training and Research Hospital
X-linked Hypophosphatemia
Study of the Anti-FGF23 Antibody, Burosumab, in Adults With XLH
This is phase 3b open-label, international, multicenter study to continue to monitor the long-term safety and efficacy of burosumab in adult patients with XLH that participated in previous clinical trials with burosumab…
- Trial name
- NCT03920072
- Testing
- Burosumab
- Where
- 10 sites in France, Ireland and 2 more
- Volunteers
- 35 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
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
Scoliosis; Idiopathic, Infantile, Scoliosis; Juvenile
Physical Activity, Motor Competence, Pulmonary Function, and Quality of Life in Children With Severe Spinal Disease
Observation study about physical activity, motor competence, pulmonary function, and health related quality of life in children, surgically treated for early onset scoliosis
- Conditions
- Scoliosis; Idiopathic, Infantile, Scoliosis; Juvenile, Scoliosis Neuromuscular, Scoliosis in Neurofibromatosis
- Trial name
- NCT03494829
- Where
- Norway
- Volunteers
- 50 people
- Sponsor
- Oslo University Hospital
Congenital Hip Dysplasia
Pericapsular Nerve Group (PENG) Block for Congenital Hip Dislocation Surgery
Developmental dysplasia of the hip (DDH) is one of the major disorder of the pediatric population with an incidence of 3 to 5 per 1000 children.
- Trial name
- NCT04212039
- Testing
- Saline Solution, Bupivacaine
- Where
- Turkey (Türkiye)
- Volunteers
- 48 people
- Sponsor
- Ataturk University
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
Gout, Low Medication Adherence
SToRytelling to Improve DiseasE Outcomes in GOut: The STRIDE-GO2 Study
The objective is to test the efficacy of a patient-centered, culturally relevant narrative intervention, or "storytelling," based on the solid conceptual foundation of the narrative communication theory and the constructs of the…
- Conditions
- Gout, Low Medication Adherence, Health Related Quality of Life
- Trial name
- STRIDE-GO2 (NCT02741700)
- Testing
- Gout Storytelling Video Intervention
- Where
- 3 sites in United States
- Volunteers
- 306 people
- Sponsor
- VA Office of Research and Development
Hemophilia, Arthropathy
Observational Study of Long-Term Joint Health Outcome in Hemophilia Patient
Patients with severe hemophilia A can experience repeated bleeding into the same joint (ie, a target joint; most frequently in the ankle, knee, and elbow), which can contribute to hemophilic arthropathy and, over time, ultimately…
- Trial name
- NCT04618237
- Where
- Taiwan
- Volunteers
- 35 people
- Sponsor
- Taipei Medical University Hospital
Hallux Limitus, Ankle Joint
Teenage Functional Hallux Limitus and Ankle Dorsiflexion Study
This study aims to identify a possible correlation between reduced mobility of the first toe under load and reduced mobility of the ankle ROM in healthy adolescent basketball players.
- Trial name
- NCT05399472
- Testing
- Dorsiflexion lunge test, Jack Test
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- University of Bologna
Neurofibromatoses
The Neurofibromatosis-associated Tumor Biobank
Neurofibromatosis-associated Tumor is very rare bone tumor. The investigator set up the biobank to ensure every patient has the chance to participate in future research
- Trial name
- NCT03359304
- Volunteers
- 300 people
- Sponsor
- Ren tingting
Recurrent Crying Spells
Recurrent Crying Spells in Cerebral Palsy
This is a study of recurrent crying spells in Spastic Cerebral Palsy patients and their treatment.
- Trial name
- RCSCPSQ (NCT01955655)
- Testing
- Baclofen
- Where
- India
- Volunteers
- 70 people
- Sponsor
- Sathbhavana Brain Clinic
Osteogenesis Imperfecta
Single Ascending Dose Study of SAR439459 in Adults With Osteogenesis Imperfecta (OI)
SAR439459 is a human anti-Transforming growth factor β (TGFβ) monoclonal antibody. This phase 1 clinical study investigates the safety, tolerability, and activity of a single dose of SAR439459 in adult participants with OI.
- Trial name
- NCT05231668
- Testing
- SAR439459
- Where
- 13 sites in United States, Australia and 2 more
- Volunteers
- 16 people
- Sponsor
- Sanofi
Duchenne Dystrophy Muscular
Biomechanical and Morphological Changes in Dystrophic Muscle
The loss of ability to walk in many children with DMD (Duchenne muscular Dystrophy) is a pejorative event. Biomechanical and morphological unknowledge about the loss of the walk ability in children with DMD is an obstacle in…
- Trial name
- MARCHE-DMD (NCT02472990)
- Where
- 10 sites in France
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University Hospital, Brest
Myotonic Dystrophy, Steinert
Factors Associated With Hypoventilation in the Myotonic Dystrophy, Progressive Profile Over 5 Years
The aim of this study is to determine the factors associated with alveolar hypoventilation in terms of cognitive impairment, daytime sleepiness, respiratory function, nocturnal respiratory events This evaluation will clarify the…
- Trial name
- Steinert (NCT03764150)
- Where
- 6 sites in France
- Volunteers
- 113 people
- Sponsor
- University Hospital, Lille
Hallux Limitus, Hallux Rigidus
Surgical Treatment for Hallux Rigidus
The purpose of this study is to examine the long-term outcomes for the surgical treatment of end stage degeneration in the big toe joint.
- Trial name
- NCT01048450
- Testing
- Joint Resection- Keller Procedure, Joint Fusion, Hemi-metallic Joint Implant
- Where
- 6 sites in United States
- Volunteers
- 158 people
- Sponsor
- American College of Foot and Ankle Surgeons
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