Clinical trials: Bone Development, open to volunteers
Aplastic Anemia, Pregnancy
A Prediction Model in Pregnant Women With Aplastic Anemia
Aplastic anemia (AA) is a rare haematologic disorder characterized by bone marrow failure and pancytopenia. Its occurrence during pregnancy is exceedingly rare but poses significant risks, including maternal hemorrhage…
- Conditions
- Aplastic Anemia, Pregnancy, Prediction Model
- Trial name
- NCT07101770
- Testing
- A combined retrospective and prospective observational follow-up
- Where
- China
- Volunteers
- 300 people
- Sponsor
- Peking University People's Hospital
Cardiomyopathy, Hypertrophic, Cardiomyopathy, Dilated
National Network for Cardiovascular Genomics: Advancing Cardiovascular Healthcare for Hereditary Diseases in Brazil's Unified Health System Through a Multicenter Registry
The goal of this observational study is to develop a registry of Brazilian patients with hereditary cardiovascular diseases, combining clinical and genomic data.
- Conditions
- Cardiomyopathy, Hypertrophic, Cardiomyopathy, Dilated, Cardiomyopathy Restrictive, Arrhythmogenic Right Ventricular Dysplasia
- Trial name
- RENOMICA-Hcor (NCT06546137)
- Testing
- whole genome sequencing
- Where
- 27 sites in Brazil
- Volunteers
- 1,211 people
- Sponsor
- Hospital do Coracao
Lower Back Pain, Facet Joint Pain; Low Back Pain
Bone Enhanced Ultrasound (BEUS) Data Library Development Project
A common treatment for low back pain involves fluoroscopy-guided spinal facet joint injections and/or medial branch nerve blocks.
- Conditions
- Lower Back Pain, Facet Joint Pain; Low Back Pain, Osteoarthritis (OA)
- Trial name
- NCT07036653
- Testing
- spinal ultrasound
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Queen's University
Osteogenesis Imperfecta
Pediatric Safety Follow-up Study of Prior Treatment With Romosozumab for Osteogenesis Imperfecta
The primary objective of this trial is to evaluate the safety of romosozumab in participants with osteogenesis imperfecta (OI) that have completed Study 20200105, regardless of whether they received investigational product…
- Trial name
- NCT07366086
- Testing
- Vitamin D, Calcium
- Where
- 4 sites in United States, Germany and 1 more
- Volunteers
- 71 people
- Sponsor
- Amgen
Adipocytic Neoplasm, Liposarcoma
NRSTS2021, A Risk Adapted Study Evaluating Maintenance Pazopanib, Limited Margin, Dose-Escalated Radiation Therapy and Selinexor in Non-Rhabdomyosarcoma Soft Tissue Sarcoma (NRSTS)
The study participant has been diagnosed with non-rhabdomyosarcoma (NRSTS). Primary Objectives Intermediate-Risk To estimate the 3-year event-free survival for intermediate-risk patients treated with ifosfamide, doxorubicin…
- Conditions
- Adipocytic Neoplasm, Liposarcoma, Atypical Fibroxanthoma, Angiomatoid Fibrous Histiocytoma
- Trial name
- NCT06239272
- Testing
- Surgical resection, Proton beam radiation therapy, Pazopanib
- Where
- 7 sites in United States
- Volunteers
- 139 people
- Sponsor
- St. Jude Children's Research Hospital
Autism Spectrum Disorder
A Study of Mesenchymal Stem Cell in the Treatment of Autism Spectrum Disorder
This clinical trial aims to evaluate the safety and preliminary efficacy of intravenous administration of human bone marrow-derived mesenchymal stem cells (hBMMSCs) in patients with autism spectrum disorder (ASD).
- Trial name
- ASD (NCT07177677)
- Testing
- hBMMSC
- Volunteers
- 42 people
- Sponsor
- Jiuzhitang Maker (Beijing) Cell Technology Co.,Ltd.
SEDC, Hypochondrogenesis
A Natural History Study in Children With a Type II Collagen Disorder With Short Stature
There are relatively few data available on type II collagen disorders, and evidence is lacking on the disease course in relation to symptoms and development of complications, the level of actual disease burden over time as well…
- Conditions
- SEDC, Hypochondrogenesis, Semd, Strudwick Type, Kniest Dysplasia
- Trial name
- ROCKET (NCT05408715)
- Testing
- Natural History Study
- Where
- 2 sites in France, Spain
- Volunteers
- 60 people
- Sponsor
- Innoskel
Craniofacial Fibrous Dysplasia
Morbidity in Adults With Craniofacial Fibrous Dysplasia: The CRANIO-DF Study
Fibrous dysplasia is a rare bone disorder in which normal bone is replaced by fibro-osseous tissue. Craniofacial involvement may be associated with substantial morbidity and complex multidisciplinary care.
- Trial name
- NCT07870616
- Testing
- No Intervention: Observational Cohort
- Where
- France
- Volunteers
- 500 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Arthritis, Spondylitis, Ankylosing
Natural History and Development of Spondyloarthritis
Background: Spondyloarthritis (SpA) is a group of bone and joint disorders that may cause back and joint pain and stiffness. In some cases, SpA can lead to abnormal bone growth affecting the joints and spine.
- Trial name
- NCT01422694
- Where
- United States
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
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
Osteoporosis, Sarcopenia
Skeletal Maturation and Endocrine Health in Young Adults
Diseases which can be the result of poor lifestyle choices in adult life, such as osteoporosis, obesity or poor muscle mass (sarcopenia) can also be driven by heritable genetic factors.
- Conditions
- Osteoporosis, Sarcopenia, Obesity, Cardiovascular Diseases
- Trial name
- EPIPEAK (NCT06509776)
- Where
- 9 sites in Denmark
- Volunteers
- 2,000 people
- Sponsor
- Holbaek Sygehus
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
Acute Respiratory Distress Syndrome ARDS
Driving Pressure-guided Tidal Volume Ventilation in the Acute Respiratory Distress Syndrome
Acute respiratory distress syndrome (ARDS) is associated with high mortality, some of which can be attributed to ventilator-induced lung injury (VILI) when artificial ventilation is not customized to the severity of lung injury.
- Trial name
- DRIVENT (NCT06322758)
- Testing
- Tidal volume customization in the acute respiratory distress syndrome
- Volunteers
- 750 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Creation of an Italian Network of Biobanks of Rare Diseases
ScATtEred Rare Disease Biobanks: a Model of Sample/Data Collection With susTainablE and Shared Criteria
Rare diseases (RDs) have been defined by the European Union (EU) as life-threatening or chronically debilitating conditions affecting less than 1 person in 2000.
- Trial name
- SATELLITES (NCT06782230)
- Where
- Italy
- Volunteers
- 800 people, healthy volunteers welcome
- Sponsor
- IRCCS Policlinico S. Donato
Skeletal Disorders
Study of Skeletal Disorders
Background: There are 461 conditions that affect the bones (skeletal disorders). Many of these are not well understood and do not have any specific treatments. Researchers want to collect more data about these conditions.
- Trial name
- NCT05031507
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Turner Syndrome, Short Stature Homeobox Gene Mutation
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, SHOX-D, SGA, or ISS between ages of ≥2 and <18 years with open growth plates.
- Conditions
- Turner Syndrome, Short Stature Homeobox Gene Mutation, Idiopathic Short Stature, Small for Gestational Age at Delivery
- Trial name
- NCT07221851
- Testing
- Lonapegsomatropin [SKYTROFA®], Somatropin Pen Injector
- Where
- 51 sites in United States, France and 5 more
- Volunteers
- 186 people
- Sponsor
- Ascendis Pharma A/S
Rheumatic Disease
Natural History, Pathogenesis and Outcome of Melorheostosis A Rare Osteosclerotic Disease
Background: The rare disease melorheostosis causes bones to thicken. This may lead to pain, and can affect bones, joints, and muscles. Researchers want to learn more about the disease and how it progresses.
- Trial name
- NCT02504879
- Where
- United States
- Volunteers
- 350 people, healthy volunteers welcome
- Sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Growth Hormone Deficiency
A Long-Term Safety Trial of LUM-201 in Children With Idiopathic Growth Hormone Deficiency Who Have Previously Completed a LUM-201 Clinical Trial (OraGrowtH211)
This is a multi-national trial. The trial aims to study the long-term safety of LUM-201 in subjects with Idiopathic Pediatric Growth Hormone Deficiency (iPGHD).
- Trial name
- OraGrowtH211 (NCT05796440)
- Testing
- LUM-201
- Where
- 7 sites in United States, Australia
- Volunteers
- 60 people
- Sponsor
- Lumos Pharma
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
Osteogenesis Imperfecta, Rare Bone Disorders
Morphology in Oral Rare Syndromes & Artificial Intelligence for Clinical Diagnosis
MOSAIC aims to determine whether oro-dental morphological anomalies, particularly palatal morphology, associated with rare bone and cartilage diseases can be precisely characterized using 3D digital models analysed through…
- Conditions
- Osteogenesis Imperfecta, Rare Bone Disorders, Hypophosphatemia, X-Linked
- Trial name
- MOSAIC (NCT07666269)
- Testing
- intra-oral 3D optical impression
- Where
- France
- Volunteers
- 240 people, healthy volunteers welcome
- Sponsor
- University Hospital, Bordeaux