Clinical trials: Skeletal Abnormalities
Growth Hormone Deficiency, Idiopathic Short Stature
Skeletal Muscle Effects of GH in Boys
The purpose of the study is to measure the functional effects of recombinant GH in skeletal muscle, in addition to growth promotion, in short prepubertal boys with either growth hormone deficiency or idiopathic short stature.
- Trial name
- NCT04020913
- Testing
- Somatropin injection
- Where
- United States
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Nemours Children's Clinic
Fibrodysplasia Ossificans Progressiva, FOP
A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP)
This study is researching an experimental drug called garetosmab, referred to as "study drug". The study is focused on children and adolescent participants with FOP.
- Trial name
- OPTIMA-2 (NCT07559513)
- Testing
- garetosmab
- Volunteers
- 18 people
- Sponsor
- Regeneron Pharmaceuticals
Enchondromatosis
Uncovering Genes Behind Cartilage Tumors and Vascular Anomalies Using Genomic Sequencing
Background: Ollier disease (OD) and Maffucci syndrome (MS) are rare disorders that increase the risk of cancers in cartilage tissue. These tumors can lead to severe skeletal deformities beginning in childhood.
- Trial name
- NCT06749366
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Achondroplasia
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia.
- Trial name
- NCT03197766
- Testing
- BMN 111
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 121 people
- Sponsor
- BioMarin Pharmaceutical
Three Dimensional, Dento-Skeletal
Three Dimensional Evaluation of Dento-Skeletal Effects of Leaf Self Expander in Cleft Lip and Palate Patients
This study aims to evaluate the dento-alveolar changes after maxillary expansion in cleft lip and palate patients by Leaf Self Expander, as compared to conventional Rapid Maxillary Expander using digital cast analysis and…
- Conditions
- Three Dimensional, Dento-Skeletal, Leaf Self Expander, Cleft Lip
- Trial name
- NCT07384338
- Testing
- Rapid maxillary expansion, Leaf Self Expander
- Where
- Egypt
- Volunteers
- 20 people
- Sponsor
- Al-Azhar University
Achondroplasia
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
Study 111-206 is a Phase 2 randomized, double-blind, placebo-controlled clinical trial of BMN 111 in infants and young children with a diagnosis of achondroplasia.
- Trial name
- NCT03583697
- Testing
- BMN 111
- Where
- 16 sites in United States, Australia and 2 more
- Volunteers
- 75 people
- Sponsor
- BioMarin Pharmaceutical
Turner Syndrome
Long-Term Growth and Skeletal Effects of Early Growth Hormone Treatment in Turner Syndrome
This is an extension study that will gather long-term data on the effect of early growth hormone (GH) treatment on adult height and other aspects of health and development in girls with Turner syndrome.
- Trial name
- NCT00266656
- Testing
- Humatrope
- Where
- 9 sites in United States
- Volunteers
- 69 people
- Sponsor
- Eli Lilly and Company
Apnea, Airway Obstruction, Nasal
Upper Airway's Pressure Drop Analyses After Mandibular Advancement and Maxillary Expansion
Breathing is a crucial function for everyone. Breathing impairment in children could lead to behavioral and cognitive problems at least.
- Conditions
- Apnea, Airway Obstruction, Nasal, Mandible Small, Maxillary Hypoplasia
- Trial name
- NCT04190953
- Testing
- Twin block then Hyrax, Hyrax then Twin block
- Where
- Canada
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- University of Alberta
Gaucher Disease
Advancing Integrated Therapies for Gaucher Disease
The purpose of this study is to better understand the natural history, clinical outcomes, and biological features of Gaucher disease in patients receiving standard medical care
- Trial name
- NCT07758816
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Yale University
Skeletal Dysplasia
Extreme Morphology and Metabolic Health
Researchers at the University of Bath are investigating the metabolism of individuals with extreme body size, including those with skeletal dysplasia (commonly known as dwarfism), to manage health risks such as heart disease.
- Trial name
- NCT05991609
- Where
- United Kingdom
- Volunteers
- 102 people, healthy volunteers welcome
- Sponsor
- University of Bath
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
Class III Malocclusion, Skeletal Malocclusion
Comparison of Skeletal Anchorage Versus Conventional Tooth-Borne Face Mask Therapy Following the Alt-RAMEC Protocol
This study investigated two different methods of maxillary protraction in growing patients with skeletal Class III malocclusion characterized by maxillary deficiency.
- Conditions
- Class III Malocclusion, Skeletal Malocclusion, Maxillary Deficiency
- Trial name
- MiniFM (NCT07279662)
- Testing
- Skeletal Anchorage Supported Maxillary Protraction, Tooth-Borne Maxillary Protraction Appliance
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people
- Sponsor
- Murat Kaan Erdem
Danon Disease
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (rAAV9) vector containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B)…
- Trial name
- NCT06092034
- Testing
- RP-A501
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 18 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Cleft Lip and Palate
Skeletal and Dental Maturational Stages in a Sample of Egyptian Subjects With Cleft Lip and Palate Deformity
The aim of this research is to evaluate the skeletal and dental maturation of cleft lip and palate patients in a sample of Egyptian population using the CVM as indicator for the skeletal maturation and the Demirjian method as…
- Trial name
- NCT05031039
- Volunteers
- 250 people
- Sponsor
- Cairo University
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)
Achondroplasia
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia
- Trial name
- NCT03424018
- Testing
- BMN 111
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 119 people
- Sponsor
- BioMarin Pharmaceutical
Skeletal Malocclusion
Cephalometric Evaluation of a Clear Mandibular Advancement Appliance Based on the Twin-block Design
This study evaluates the cephalometric effects of a clear mandibular advancement appliance for the treatment of skeletal class II growing patients suffering from mandibular deficiency.
- Trial name
- NCT03824574
- Testing
- Clear Mandibular Advancement Appliance
- Volunteers
- 20 people
- Sponsor
- Ain Shams University
Pediatric ALL, Congenital Heart Disease
Exercise MRI to Evaluate Cardiorespiratory Fitness in Children With Heart Disease
There are many barriers to heart-healthy lifestyles in pediatric patients with acquired and congenital heart disease. Investigators want to further understand how participants heart and skeletal muscles work together during…
- Conditions
- Pediatric ALL, Congenital Heart Disease, Heart Transplantation
- Trial name
- NCT06325280
- Testing
- Exercise cardiac MRI Assessment, Cardiopulmonary Exercise Test
- Where
- Canada
- Volunteers
- 20 people
- Sponsor
- University of Alberta
Gaucher's Disease
Bone Response to Enzyme Replacement in Gaucher's Disease
The purpose of this study is to examine how the skeleton responds to repeated doses of enzyme replacement therapy in patients with type I Gaucher's disease who have had their spleens removed.
- Trial name
- NCT00001416
- Testing
- CEREDASE™
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Skeletal Dysplasia
Identification and Characterization of Bone-related Genetic Variants in Families
Identify novel genetic variants causing skeletal dysplasia in Qatari populations and to additionally develop an understanding of how those variants influence skeletal biology at a molecular and cellular level.
- Trial name
- NCT02762318
- Testing
- Samples With DNA
- Where
- Qatar
- Volunteers
- 5 people, healthy volunteers welcome
- Sponsor
- Weill Medical College of Cornell University