Clinical trials: Skeletal Dysplasia
Oral Submucous Fibrosis
Metformin Repurposing in Oral Submucous Fibrosis: Unveiling In Vitro Signaling Pathways, Progressing to Clinical Trial
OSF is a widespread health issue in Asian countries, notably Pakistan, linked to the consumption of pan, chalia, and gutka, affecting a rising number of young individuals as an epidemic.
- Trial name
- MROSF (NCT06332612)
- Testing
- Metformin Hydrochloride, betamethasone dipropionate, Pentoxifylline
- Where
- Pakistan
- Volunteers
- 30 people
- Sponsor
- Ziauddin University
Sickle Cell Disease, Thalassemia
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor.
- Conditions
- Sickle Cell Disease, Thalassemia, Anemia, Granuloma
- Trial name
- NCT00730314
- Testing
- Hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Children's Hospital Los Angeles
Asthma
TGF-(Beta) and Susceptibility to RSV
Background: Human respiratory syncytial virus (RSV) is a virus that causes respiratory tract infections, and is frequently responsible for hospital visits in infants and children.
- Trial name
- NCT01224691
- Where
- United States
- Volunteers
- 113 people
- Sponsor
- National Institute of Environmental Health Sciences (NIEHS)
Muscle Injury, Pulsed Electromagnetic Therapy
Investıgatıon of The Effıciency of Pulsed Electromagnetıc Field Therapy and Stretching Exercises
In this study, a total of 69 Wistar Albino rats were used, five of which were in the preliminary study. The preliminary study was planned to determine the ideal PEMF treatment time.
- Conditions
- Muscle Injury, Pulsed Electromagnetic Therapy, Muscle Stretching Exercise
- Trial name
- NCT05988476
- Testing
- Exercise, Pulsed Electromagnetic Field Therapy
- Where
- Turkey (Türkiye)
- Volunteers
- 64 people
- Sponsor
- Suleyman Demirel University
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
Osteogenesis Imperfecta
Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta
The primary objective of the study is to evaluate the effect of setrusumab on reduction in fracture rate, including morphometric vertebral fractures.
- Trial name
- NCT06636071
- Testing
- setrusumab
- Where
- 3 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Tumor, Facial Fracture
A Post Market Clinical Follow-up Study on Biomet Microfixation HTR PEKK (Midface), Facial & Mandibular Plates.
This study will address medical devices manufactured by Biomet Microfixation (d.b.a. Zimmer Biomet) designed for fixation and stabilization of the facial and mandibular skeleton.
- Conditions
- Tumor, Facial Fracture, Mandibular Fractures, Fibrous Dysplasia
- Trial name
- NCT04931056
- Testing
- Titanium Facial Plates, Titanium Mandibular Plates, Add-on Condyle
- Where
- Argentina
- Volunteers
- 91 people
- Sponsor
- Zimmer Biomet
Healthy, Head and Neck Cancer
Rehabilitation Assessment and Progress Tracking System Based on Video Images for Dysphagic Patients
The current cervical range of motion (CROM) and tongue movement measurement methods are not reliable between observers, which is not practical in real-world usage.
- Trial name
- NCT05141539
- Testing
- Rehabilitation assessment software, VFSS and FOIS
- Where
- Thailand
- Volunteers
- 30 people
- Sponsor
- Mahidol University
Achondroplasia, Hypochondroplasia
C-Type Natriuretic Peptide and Achondroplasia
Achondroplasia and hypochondroplasia are the most common forms of dwarfism. Recent studies have shown that a small hormone called C-type natriuretic peptide (CNP) is an important regulator of linear growth.
- Trial name
- NCT01541306
- Where
- United States
- Volunteers
- 91 people
- Sponsor
- Nemours Children's Clinic
Rheumatoid Arthritis, Osteoarthritis
A Multi-centre Study to Assess the Long-term Performance of the DePuy ASR™ System in Primary Hip Resurfacing Surgery
The purpose of this study is to monitor the performance and determine the metal ion release of the DePuy ASR™ System in the treatment of patients with hip joint disease requiring hip resurfacing surgery.
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Post-traumatic Arthritis, Collagen Disorders
- Trial name
- NCT00208377
- Testing
- DePuy ASR Hip System
- Where
- 3 sites in Australia, Germany and 1 more
- Volunteers
- 228 people
- Sponsor
- DePuy International
Osteogenesis Imperfecta
Pamidronate to Treat Osteogenesis Imperfecta in Children
This study will evaluate the effect of pamidronate a drug that decreases bone resorption (breakdown) on osteogenesis imperfecta. This is a genetic disorder of collagen, the major protein in bone.
- Trial name
- NCT00005901
- Testing
- Pamidronate (Aredia)
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
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
Achondroplasia
Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
This is a long-term, multi-center, observational study in children 2.5 to <17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life…
- Trial name
- NCT04035811
- Where
- 32 sites in United States, Argentina and 9 more
- Volunteers
- 271 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Achondroplasia
A Study in Children With Achondroplasia
The goal of this observational study is to collect the anthropometric parameters, clinical characteristics, related medical complications, health-related quality of life and treatments of children with ACH, and complete a natural…
- Trial name
- NCT07301463
- Testing
- No Interventions, complete a natural history observation of ACH for at least 6 months and up to 2 years
- Where
- 8 sites in China
- Volunteers
- 260 people
- Sponsor
- Abbisko Therapeutics Co, Ltd
Osteopetrosis
Stem Cell Transplantation for Children Affected With Osteopetrosis
Malignant infantile osteopetrosis (MIOP) is a rare fatal genetic disorder that is characterized by the bone's inability to regulate remodeling. The only curative therapy is hematopoietic stem cell transplantation.
- Trial name
- NCT00145587
- Testing
- Stem Cell Transplantation, Miltenyi Biotec CliniMACS, Systemic chemotherapy and antibodies
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- St. Jude Children's Research Hospital
Fibrodysplasia Ossificans Progressiva (FOP)
The Fibrodysplasia Ossificans Progressiva (FOP) Registry
The Fibrodysplasia Ossificans Progressiva (FOP) Registry is a global, non-interventional, voluntary database that captures demographic and disease data directly from FOP patients and their caregivers via a secure, web-based…
- Trial name
- NCT02745158
- Where
- United States
- Volunteers
- 800 people
- Sponsor
- The International FOP Association
Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI
Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
BACKGROUND/OBJECTIVE: Quantitative urine screening for mucopolysaccharides (MPS) has been the primary method for detecting mucopolysaccharidoses in children.
- Trial name
- NCT01707433
- Testing
- Enzyme testing
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Children's Hospitals and Clinics of Minnesota
Connective Tissue Disorders, Osteogenesis Imperfecta
Evaluation and Treatment of Patients With Connective Tissue Disease
This study offers evaluation and treatment of patients with a suspected connective tissue disorder. The protocol is not designed to test new treatments; rather, patients receive standard care.
- Conditions
- Connective Tissue Disorders, Osteogenesis Imperfecta, Bone Diseases, Metabolic, Melorheostosis
- Trial name
- NCT00076830
- Where
- United States
- Volunteers
- 180 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Transitional Cell Carcinoma of Bladder
PF-03446962 in Relapsed or Refractory Urothelial Cancer
Salvage chemotherapy for advanced urothelial cancer (UC) yields suboptimal response rates of 15-40%, a median progression-free survival of 2-4 months and a median overall survival of 6 months.
- Trial name
- NCT01620970
- Testing
- PF03446962
- Where
- Italy
- Volunteers
- 14 people
- Sponsor
- Fondazione IRCCS Istituto Nazionale dei Tumori, Milano
Osteogenesis Imperfecta
Nutritional Interventional in Pediatric Patients With Osteogenesis Imperfecta
BACKGROUND: In several bone disorders, adequate calcium intake is a coadjuvant intervention to regular treatment. Osteogenesis imperfecta (OI) is a collagen disorder with a range of symptoms, ranging from fractures to minimum…
- Trial name
- NCT03841188
- Testing
- Nutritional orientation
- Where
- Brazil
- Volunteers
- 52 people
- Sponsor
- Hospital de Clinicas de Porto Alegre