Clinical trials: Deformity; Bone
Short Stature
Growth Hormone Treatment in Patients With Aggrecan (ACAN) Deficiency
This is an open-label, single-arm prospective pilot study to study the effects of a single dose regimen of daily growth hormone medication (Norditropin) on pre-pubertal children with Aggrecan deficiency.
- Trial name
- NCT03288103
- Testing
- Norditropin
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Growth Hormone Deficiency
Use of miRNAs in Growth Hormone Deficiency (GHD)
This study aims at improving knowledge about the diagnosis of growth hormone deficiency (GHD) and treatment with growth hormone (GH), with the goal of providing information on the presence of new biomarkers, such as miRNAs, for…
- Trial name
- GH-miRNA2 (NCT06455956)
- Where
- Italy
- Volunteers
- 400 people
- Sponsor
- University of Parma
Gorham Disease, Lymphatic Diseases
Imaging of Lymphatic Anomalies
Lymphatic Anomalies (LA) is characterized by proliferation of lymphatic tissue causing deterioration of pulmonary function. Understanding changes in lymphatic anatomy in these patients is hindered by the difficulty of imaging the…
- Conditions
- Gorham Disease, Lymphatic Diseases, Lymphangiomatosis
- Trial name
- NCT02744027
- Testing
- Dynamic Contrast Enhanced Magnetic Resonance Lymphangiography (DCMRL), Heavy Weighted T2 Imaging (T2W), Lymph Node Access
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Children's Hospital of Philadelphia
Short Stature, Growth Hormone Deficiency
The IGFBP-3 Stimulation Test: A New Tool for the Diagnosis of Growth Hormone Deficiency in Children.
This project is designed to answer the question: Is there an acute IGFBP-3 response in normal children? Our specific hypothesis states that under the influence of growth hormone secretagogues, intact IGFBP-3 molecule will undergo…
- Trial name
- NCT00235599
- Testing
- IGFBP-3 Stimulation Test
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- University of Massachusetts, Worcester
Hemifacial Microsomia
Computer Guided for Mandibular Distraction Osteogenesis
Objectives: Distraction Osteogenesis is an integral part in management of mandibular defects associated with Hemifacial Microsomia (HFM) and post-ankylotic mandibular defects.
- Trial name
- NCT03869021
- Testing
- 3D printed surgical guide constructed, free hand surgery
- Where
- Egypt
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Yasser Mohamed Nabil Ahmed Khirat El Hadidi
Osteogenesis Imperfecta, Total Absence of Permanent Teeth
Site Preservation After Tooth Extraction
This study is mainly targeted affected teeth which could not be retained, and patients are willing to undergo implant repair at the later stage.
- Conditions
- Osteogenesis Imperfecta, Total Absence of Permanent Teeth, Tooth Socket
- Trial name
- NCT05258019
- Volunteers
- 88 people, healthy volunteers welcome
- Sponsor
- Nanfang Hospital, Southern Medical University
Achondroplasia
A Study to Learn About Recifercept in Patients With Achondroplasia
The purpose of this study is to learn about the study medicine (called recifercept) in people with achondroplasia. Achondroplasia is a very rare disease and patients of achondroplasia have short arms and legs.
- Trial name
- NCT05659719
- Testing
- Recifercept
- Where
- United States
- Volunteers
- 248 people
- Sponsor
- Pfizer
Adult Rhabdomyosarcoma, Adult Synovial Sarcoma
Cixutumumab in Treating Patients With Relapsed or Refractory Solid Tumors
This phase II trial is studying the side effects and how well cixutumumab works in treating patients with relapsed or refractory solid tumors. Monoclonal antibodies, such as cixutumumab, can block tumor growth in different ways.
- Conditions
- Adult Rhabdomyosarcoma, Adult Synovial Sarcoma, Childhood Hepatoblastoma, Childhood Synovial Sarcoma
- Trial name
- NCT00831844
- Testing
- cixutumumab, laboratory biomarker analysis
- Where
- 105 sites in United States, Australia and 1 more
- Volunteers
- 116 people
- Sponsor
- National Cancer Institute (NCI)
Osteogenesis Imperfecta
High Resolution Thermal Imaging to Identify Vertebral Fractures in Children and Young People With Osteogenesis Imperfecta
Brittle bone disease also known as osteogenesis imperfecta (OI) is characterised by a defect in the bone tissue that leads to recurrent fractures and significant bone deformities in children.
- Trial name
- THERMOI (NCT04231916)
- Testing
- Thermal imaging device
- Volunteers
- 11 people
- Sponsor
- Sheffield Children's NHS Foundation Trust
Degenerative Disc Disease, Degenerative Scoliosis
Clinical Outcome Measure at Stryker Spine
This is a multicenter, prospective, retrospective, Post Market Clinical Follow-up (PMCF) study in subjects who have undergone or will undergo surgery utilizing one or more Stryker devices according to Stryker cleared Instructions…
- Trial name
- COMPASS (NCT06226272)
- Testing
- Spinal Fusion, Spinal Deformities Correction
- Where
- 7 sites in United States
- Volunteers
- 5,222 people
- Sponsor
- VB Spine, LLC
Wilms Tumor, Fibrosarcoma
Study of High-Dose Chemotherapy With Bone Marrow or Stem Cell Transplant for Rare Poor-Prognosis Cancers
The purpose of this study is to determine whether very high dosages of chemotherapy will improve the chance of surviving cancer.
- Conditions
- Wilms Tumor, Fibrosarcoma, Carcinoma, Round Cell, Nasopharyngeal Cancer
- Trial name
- NCT00141765
- Testing
- Myeloablative Chemotherapy, Stem Cell Rescue
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of Michigan Rogel Cancer Center
Severe Aplastic Anemia
Retrospective Study of Patients With Severe Aplastic Anemia Who Developed High Risk Clonal Evolution With Chromosome 7 Abnormalities After Immunosuppressive Therapy
Background: Severe aplastic anemia (SAA) is a form of bone marrow failure. It usually results from a cytotoxic T cell attack on the marrow stem cell. Two treatments can be used for SAA.
- Trial name
- NCT04436367
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Inherited Metabolic Disorders (IMD)
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
- Trial name
- NCT03406962
- Testing
- MGTA-456
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Magenta Therapeutics, Inc.
Neuromuscular Diseases, Congenital Diaphragmatic Hernia
Respiratory Muscles and Work of Breathing in Children
Respiratory muscle testing allows a quantitative assessment of inspiratory and expiratory muscles in children of any age with primary or secondary respiratory muscle impairment, in order to better understand the pathophysiology…
- Conditions
- Neuromuscular Diseases, Congenital Diaphragmatic Hernia, Lung Diseases, Cardiac Diseases
- Trial name
- WOB&MR_Ped (NCT05051254)
- Testing
- Esogastric pressure measurement
- Where
- France
- Volunteers
- 550 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Achondroplasia
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
The purpose of this clinical trial is to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of navepegritide 100 μg/kg compared to placebo (inactive drug) in adolescents aged 12 to 18 years with Achondroplasia.
- Trial name
- teACH (NCT06732895)
- Testing
- Navepegritide
- Where
- 5 sites in Canada, Denmark and 3 more
- Volunteers
- 25 people
- Sponsor
- Ascendis Pharma A/S
Fanconi Anemia
Bone Marrow Cell Gene Transfer in Individuals With Fanconi Anemia
Fanconi anemia (FA) is a disease that affects an individual's bone marrow. It is caused by a defective gene in the bone marrow cells that produce various types of blood cells.
- Trial name
- NCT00272857
- Testing
- Retrovirus Construct
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Boston Children's Hospital
Sickle Cell Disease, Sickle Cell Trait
Bone Loss, Physical Function and Frailty in Older Women With Sickle Cell Trait Sickle Cell Trait
This is a cross-sectional, clinical research study comparing postmenopausal women of African Descent (AD) with different hemoglobin genotypes: normal and sickle cell trait (SCT). This research study has two purposes.
- Trial name
- NCT07083531
- Testing
- Healthy volunteers without SCT, Healthy volunteers with SCT
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- UConn Health
Maxillofacial Bone Defects, Alveolar Bone Atrophy
Gene-activated Matrix for Bone Tissue Repair in Maxillofacial Surgery
The purpose of this study is to evaluate safety and efficacy of gene-activated matrix ("Nucleostim") for regeneration of bone tissue in maxillofacial area.
- Conditions
- Maxillofacial Bone Defects, Alveolar Bone Atrophy, Jaw Fractures, Maxillofacial Bone Deformities
- Trial name
- NCT02293031
- Testing
- Gene-activated matrix "Nucleostim"
- Where
- 2 sites in Russia
- Volunteers
- 12 people
- Sponsor
- NextGen Company Limited
Duchenne Muscular Dystrophy
Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy
This study is single arm, single center trial to study the safety and efficacy of bone marrow-derived autologous specific populations of stem cells and mesenchymal stem cells for the treatment of Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT03067831
- Testing
- Stem Cells
- Where
- Jordan
- Volunteers
- 20 people
- Sponsor
- Stem Cells Arabia
Scoliosis Idiopathic, Scoliosis; Adolescence
Comparison of the Effects of Controlled Schroth Exercise and Home Exercise Programs
The aim of this study is to compare the effects of Schroth 3D exercise method on home symmetry, trunk topography, scapula symmetry, pelvic symmetry, health related quality of life and cosmetic deformity perception in adolescents…
- Trial name
- NCT04203394
- Testing
- Schroth Exercise Group, Home Exercise Group
- Where
- Turkey (Türkiye)
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- Eastern Mediterranean University
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