Clinical trials: Bone Deformity, open to volunteers
Neuroblastoma, Sarcoma
A Study of Cabozantinib as a Maintenance Agent to Prevent Progression or Recurrence in High-Risk Pediatric Solid Tumors
This study will expand the types of pediatric cancers being evaluated for response to cabozantinib. The current COG study is restricted to Ewing sarcoma, osteosarcoma, rhabdomyosarcoma, Wilms tumor, and a handful of uncommon…
- Trial name
- NCT05135975
- Testing
- Cabozantinib
- Where
- 6 sites in United States
- Volunteers
- 86 people
- Sponsor
- Nationwide Children's Hospital
Lymphatic Malformation, Generalized Lymphatic Anomaly (GLA)
Lymphatic Anomalies Registry for the Assessment of Outcome Data
Lymphatic anomalies are a rare subset of vascular anomalies that are poorly understood. the understanding of the natural history, long-term outcomes, risk factors for morbidity and mortality, and the relative benefit of medical…
- Conditions
- Lymphatic Malformation, Generalized Lymphatic Anomaly (GLA), Central Conducting Lymphatic Anomaly, CLOVES Syndrome
- Trial name
- NCT02399527
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- Boston Children's Hospital
Vascular Anomaly, Generalized Lymphatic Anomaly
Identification of Biomarkers for Patients with Vascular Anomalies
The study will use blood (serum and plasma) and tissue obtained from participants undergoing prescribed surgical resection of vascular anomalies of interest proposed in this study.
- Conditions
- Vascular Anomaly, Generalized Lymphatic Anomaly, Kaposiform Hemangioendothelioma, Kaposiform Lymphangiomatosis
- Trial name
- NCT03001180
- Where
- 2 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly deoxyribonucleic acid (DNA)) from the patient to treat his…
- Trial name
- NCT07708350
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Daniel Bauer
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
Pseudohypoparathyroidism Type 1a, Obesity
Setmelanotide to Treat Obesity in a Patient With Pseudohypoparathyroidism Type 1a (PHP1a)
The investigators plan to test the efficacy and safey of 6-months of open-label setmelanotide to treat obesity in a single patient with pseudohypoparathyroidism type 1a due to a GNAS mutation.
- Trial name
- NCT07496463
- Testing
- Setmelanotide
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Massachusetts General Hospital
Pectus Carinatum
Prognosis Factors for the Treatment of Pectus Carinatum With a Dynamic Compression System
Pectus carinatum is a deformation of the thoracic wall causing an aesthetic prejudice. Since 2011, our team uses a dynamic compression system to treat this deformation.
- Trial name
- Pectusdyn (NCT04452721)
- Testing
- dynamic compression system
- Where
- France
- Volunteers
- 520 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Neurological Regression, Myoclonus
Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
Study description: This is a natural history study that will evaluate any patient with enzyme or DNA confirmed GM1 or GM2 gangliosidosis, sialidosis or galactosialidosis.
- Conditions
- Neurological Regression, Myoclonus, Cherry Red Spot, Brain Atrophy
- Trial name
- NCT00029965
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
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
Hereditary Hemorrhagic Telangiectasia, Pulmonary Arterial Hypertension
DIAG723 in Adults With Hereditary Hemorrhagic Telangiectasia
This is a Phase 1/2, randomized, double-blind, placebo-controlled, first-in-human study evaluating the safety, tolerability, pharmacokinetics, and preliminary efficacy of subcutaneously administered DIAG723 in adult patients with…
- Trial name
- DIAMOND (NCT07623525)
- Testing
- DIAG723
- Where
- 11 sites in Australia, Canada and 1 more
- Volunteers
- 93 people
- Sponsor
- Diagonal Therapeutics, Inc.
Romberg Disease, Craniofacial Microsomia
Efficacy of Preconditioned Adipose-Derived Stem Cells in Fat Grafting
The aim of this study is to evaluate the comparison of therapeutic potential of curcumin preconditioned adipose derived stem cells (ASCs) enrichment fat grafting, naïve ASCs enrichment fat grafting and conventional fat grafting…
- Conditions
- Romberg Disease, Craniofacial Microsomia, Burn Scar, Post-Traumatic Scar (Disorder)
- Trial name
- NCT05610878
- Testing
- Cultured ASCs enrichment fat grafting, Curcumin-preconditioned ASCs enrichment fat grafting, Conventional fat grafting
- Where
- Pakistan
- Volunteers
- 24 people
- Sponsor
- University of the Punjab
Radiculopathy, Cervical, Myelopathy Cervical
Pilot Study of ZetaFuse™ Bone Graft for the Treatment of Cervical Degenerative Disc Disease
The goal of this pilot clinical trial is to test the safety and preliminary performance of the ZetaFuse Bone Graft in patient requiring fusion of the C3-C7 vertebral bones due to pain or loss of neurological function.
- Conditions
- Radiculopathy, Cervical, Myelopathy Cervical, Neurological Abnormality, Disc Disease
- Trial name
- NCT05971329
- Testing
- Cervical Fusion (ZetaFuse™ Bone Graft)
- Where
- 2 sites in Australia
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- Zetagen Therapeutics, Inc
Neurologic Disorders, Nervous System Diseases
Neurologic Stem Cell Treatment Study
This is a human clinical study involving the isolation of autologous bone marrow derived stem cells (BMSC) and transfer to the vascular system and inferior 1/3 of the nasal passages in order to determine if such a treatment will…
- Conditions
- Neurologic Disorders, Nervous System Diseases, Neurodegenerative Diseases, Neurological Disorders
- Trial name
- NEST (NCT02795052)
- Testing
- Intravenous and Intranasal BMSC
- Where
- 3 sites in United States, United Arab Emirates
- Volunteers
- 500 people
- Sponsor
- MD Stem Cells
Chronic Myeloproliferative Disorders, Cognitive/Functional Effects
Registry of Older Patients With Cancer
RATIONALE: Gathering information about older patients with cancer may help the study of cancer in the future. PURPOSE: This research study is gathering information from older patients with cancer into a registry.
- Conditions
- Chronic Myeloproliferative Disorders, Cognitive/Functional Effects, Leukemia, Lymphoma
- Trial name
- NCT01137825
- Where
- 7 sites in United States
- Volunteers
- 3,000 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Radiculopathy, Cervical, Myelopathy Cervical
Cervical Arthroplasty Cost Effectiveness Study (CACES)
To date, no consensus exists on which anterior surgical technique is more cost-effective to treat cervical degenerative disc disease (CDDD).
- Conditions
- Radiculopathy, Cervical, Myelopathy Cervical, Myelopathy, Compressive, Radiculopathy, Cervical Region
- Trial name
- CACES (NCT04623593)
- Testing
- ACDF, ACDA
- Where
- Netherlands
- Volunteers
- 198 people
- Sponsor
- Valérie Schuermans
Silver Russell Syndrome, Beckwith-Wiedemann Syndrome
IDMet (RaDiCo Cohort) (RaDiCo-IDMet)
The goal of this observational study is to describe the natural history of imprinting disorders (IDs) according to their metabolic profile in all patients (adults and children) affected with an ID regardless of the severity of…
- Conditions
- Silver Russell Syndrome, Beckwith-Wiedemann Syndrome, Transient Neonatal Diabetes Mellitus, Angelman Syndrome
- Trial name
- IDMet (NCT05945576)
- Where
- 20 sites in France
- Volunteers
- 2,000 people
- Sponsor
- Institut National de la Santé Et de la Recherche Médicale, France
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
RPS19 Deficient Diamond-Blackfan Anemia
Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Brief summary The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children.
- Trial name
- NCT07476183
- Testing
- APR-2020
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Apriligen, Inc.
Hemifacial Microsomia, Mucormycosis
Clinical Evaluation of Patient-Specific 3D-Printed Titanium Implants for Facial Reconstruction.
This clinical study aims to evaluate the effectiveness of patient-specific 3D-printed titanium facial implants in the reconstruction and aesthetic correction of facial defects.
- Conditions
- Hemifacial Microsomia, Mucormycosis, Oral Squamous Cell Carcinoma, Microgenia
- Trial name
- 3DFACE (NCT07654647)
- Testing
- Patient-specific titanium implants for facial reconstruction
- Where
- Syria
- Volunteers
- 30 people
- Sponsor
- Latakia University
Musculoskeletal Deformity, Musculoskeletal Diseases
Customized Bone Allografts by 3D-printing
Virtual surgical planning (VSP), the simulation of bone corrections in virtual reality ("Computer Aided Surgical Simulation": CASS) and 3D printing of customized implants and devices are achieving an increasingly central role in…
- Conditions
- Musculoskeletal Deformity, Musculoskeletal Diseases, Musculoskeletal Disorder
- Trial name
- 3D-MALF 2 (NCT05700526)
- Testing
- Planning of multiplanar and/or multifocal osteotomies in children by using VSP, PSIs and customized structural bone allograft, Routine preparation of bone graft according to standard protocols, Preparation of bone graft with virtual bone-matching and GSIs
- Where
- Italy
- Volunteers
- 42 people
- Sponsor
- Istituto Ortopedico Rizzoli