Clinical trials: Bone Deformity
Childhood Langerhans Cell Histiocytosis, Leukemia
Umbilical Cord Blood and Placental Blood Transplantation in Treating Patients With Hematologic Cancer or Aplastic Anemia
RATIONALE: Umbilical cord blood or placental blood transplantation may be able to replace immune cells that were destroyed by the chemotherapy or radiation therapy that was used to kill cancer cells.
- Conditions
- Childhood Langerhans Cell Histiocytosis, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00008164
- Testing
- umbilical cord blood transplantation
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Herbert Irving Comprehensive Cancer Center
Pectus Excavatum
Pain, Exercise and Psychological Well-being in Pectus Excavatum
Recovery following major surgical procedures can be influenced by both physical (optimization of cardiopulmonary function, pain control, activity) and psychological factors.
- Trial name
- PEP (NCT02337621)
- Testing
- No intervention
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Phoenix Children's Hospital
Funnel Chest
Functional Capacity of Exercise and Lung Function in Patients Submitted Early Rehabilitation
The aim of this study was to compare the functional capacity of exercise by six-minute walk test and lung function by spirometry, before and after Nuss procedure in subjects who have undergone the early rehabiliation program with…
- Trial name
- NCT02441452
- Testing
- Early rehabilitation
- Volunteers
- 40 people
- Sponsor
- Instituto do Coracao
Fanconi Anemia
Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100
The purpose of this research study is to determine whether an experimental drug called AMD3100 used in combination with another medication called G-CSF is safe and can help to increase the amount of blood stem cells (called CD34+…
- Trial name
- NCT00479115
- Testing
- AMD3100, AmCell CliniMACs
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Inherited BMF Syndrome, Inherited Platelet Disorder
Diagnosis and Phenotype Characterisation Using Genomics in Patients With Inherited Bone Marrow Failure (IBMDx Study)
This project seeks to perform whole genome sequence (WGS) and whole transcriptome sequence (WTS) analysis on 350 patients with suspected inherited bone marrow failure syndromes and related disorder (IBMFS-RD) in order to increase…
- Conditions
- Inherited BMF Syndrome, Inherited Platelet Disorder, Hematologic Diseases
- Trial name
- IBMDx (NCT05196789)
- Testing
- whole genome and transcriptome sequencing
- Where
- Australia
- Volunteers
- 350 people
- Sponsor
- Peter MacCallum Cancer Centre, Australia
Hypophosphatasia (HPP)
Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)
This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed Study ENB-006-09 (NCT00952484).
- Trial name
- NCT01203826
- Testing
- Asfotase Alfa
- Where
- 2 sites in United States, Canada
- Volunteers
- 12 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Alpha Mannosidosis
The Natural History of Alpha-Mannosidosis
The natural history study of the rare lysosomal disease alpha-mannosidosis will answer the question; why the rare disease develops as it does?
- Trial name
- HUE-MAN (NCT00498420)
- Where
- 4 sites in Czechia, Germany and 2 more
- Volunteers
- 45 people
- Sponsor
- Zymenex A/S
Sickle Cell Anemia, Hemoglobinopathy
Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies
The major goal of this study is to determine the risks and benefits of bone marrow transplants in patients with severe thalassemia or sickle cell disease. Participation in this project will be for two years.
- Conditions
- Sickle Cell Anemia, Hemoglobinopathy, Thalassemia
- Trial name
- NCT00040469
- Testing
- Campath -1H, Dilantin, Busulfan
- Where
- 2 sites in United States
- Volunteers
- 15 people
- Sponsor
- Baylor College of Medicine
Hypopituitarism
Arimidex Multicenter Trial in Growth Hormone (GH) Deficient Boys
The purpose of this study is to see if Arimidex, an aromatase inhibitor, can delay epiphyseal fusion and increase predicted adult height in boys who are growth hormone deficient, in puberty, and who are taking growth hormone.
- Trial name
- NCT00133354
- Testing
- Arimidex (Anastrozole), Growth Hormone
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- Nemours Children's Clinic
Marfan Syndrome, Loeys-Dietz Syndrome
Genotypes and Craniofacial Phenotypes in Orthodontic Patients With Marfan and Loeys-Dietz Syndromes
The aim of this retrospective observational study is to investigate the association between genotype and craniofacial phenotype in orthodontic patients affected by Marfan and Loeys-Dietz syndromes.
- Trial name
- NCT07360704
- Testing
- Cephalometric analysis
- Where
- Italy
- Volunteers
- 39 people
- Sponsor
- Andrea Scribante
Osteogenesis Imperfecta
Urinary Biomarkers of OI Pathobiology
Osteogenesis imperfecta (OI) is a rare inherited disorder that causes bones to break easily. Individuals with osteogenesis imperfecta break bones often and may have other problems, including hearing loss, dental problems, pain…
- Trial name
- NCT02531087
- Where
- 7 sites in United States, Canada
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Baylor College of Medicine
Romberg's Disease, Craniofacial Microsomia
Role of Mesenchymal Stem Cells in Fat Grafting
Contour deformity of face causes both functional as well as aesthetic problems for the patient. Fat being an autologous tissue source is considered as an ideal soft-tissue filler because it is abundant, readily available…
- Conditions
- Romberg's Disease, Craniofacial Microsomia, Lipodystrophy, Mixed Connective Tissue Disease
- Trial name
- NCT02494752
- Testing
- Fat graft enriched with ex vivo expanded stem cells, Fat graft not enriched with ex vivo expanded stem cells
- Volunteers
- 30 people
- Sponsor
- King Edward Medical University
Growth Failure, X-linked Severe Combined Immunodeficiency (XSCID)
Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1
This study will evaluate the safety and effectiveness of insulin-like growth factor-1 (IGF-1) to treat patients with X-linked severe combined immunodeficiency (XSCID).
- Conditions
- Growth Failure, X-linked Severe Combined Immunodeficiency (XSCID), Growth Hormone Resistence
- Trial name
- NCT00490100
- Testing
- Increlex
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hypoparathyroidism, Pseudo Hypoparathyroidism
Institution of an Italian Multicenter Database of Patients Affected by Hypoparathyroidism or Pseudohypoparathyroidism
The goal of this observational study is to create, manage and analyze a multicenter national database of patients affected by hypoparathyroidism or pseudohypoparathyroidism, aimed at collecting and studying anamnestic…
- Trial name
- NCT06645899
- Where
- 41 sites in Italy
- Volunteers
- 600 people
- Sponsor
- F.I.R.M.O. - Fondazione Italiana Ricerca sulle Malattie dell'Osso - Ente del Terzo Settore
Silver Russell Syndrome, Prader-Willi Syndrome
Aromatase Inhibitor in Bone Maturation, Children With Silver Russell or Prader-Willi Syndrome
There is currently no drug with pediatric marketing authorization capable of limiting the advance in bone maturation of children with aggressive adrenarche.
- Trial name
- ANASILPRA (NCT01520467)
- Testing
- Anastrozole
- Where
- France
- Volunteers
- 27 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Marfan Syndrome
Comparison of Two Medications Aimed at Slowing Aortic Root Enlargement in Individuals With Marfan Syndrome
Marfan syndrome is a hereditary connective tissue disorder. Many individuals with this condition die because of the associated heart and blood vessel abnormalities.
- Trial name
- NCT00429364
- Testing
- Losartan Potassium, Atenolol
- Where
- 26 sites in United States, Belgium and 1 more
- Volunteers
- 608 people
- Sponsor
- Carelon Research
Thalassemia
Zinc & Bone Health in Thalassemia: The Think Zinc Study
The purpose of this study is to test whether zinc can improve bone health in young patients with thalassemia.
- Trial name
- ThinkZn (NCT00459732)
- Testing
- Zinc
- Where
- 3 sites in United States
- Volunteers
- 45 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Lysosomal Storage Disease, Peroxisomal Disorder
Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
This study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) in patients with high-risk lysosomal and peroxisomal disorders using a novel…
- Trial name
- NCT01626092
- Testing
- Campath-1H, Clofarabine, Melphalan
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Congenital Hypothyroidism
Evaluation of an Intensified Systematic Screening for Congenital Hypothyroidism in Premature Newborns
Currently in France, screening for congenital hypothyroidism (CH) in premature infants is done by a single TSH assay on filter paper. However, European recommendations advise repeating the assay within the first month of life.
- Trial name
- PREMATHYRO (NCT07425028)
- Testing
- Blood test
- Where
- France
- Volunteers
- 1,600 people
- Sponsor
- University Hospital, Lille
Spinal Muscular Atrophy Type I, Scoliosis
The Effect of Spinal Orthosis on the Development of Scoliosis and Chest Deformity in Type I Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a serious neuromuscular disease characterized by the degeneration of alpha motor neurons in the spinal cord, resulting in progressive proximal muscle atrophy and denervation.
- Conditions
- Spinal Muscular Atrophy Type I, Scoliosis, Chest Deformities, Spinal Orthosis
- Trial name
- NCT05878418
- Testing
- Thoracolumbosacral Spinal Orthosis (TLSO), Trunk Exercise (TE), Pulmonary Rehabilitation(PR) and Pulmonary Care (PC)
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Medipol University
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