Clinical trials: Deformity; Bone
Osteogenesis Imperfecta, Osteogenesis Imperfecta Type III
Mesenchymal Cell Therapy in Osteogenesis Imperfecta (OI)
This is a Phase 1/2 study to determine the safety and efficacy of allogeneic (third party), bone-marrow derived mesenchymal stromal cells (MSCs) for the treatment of Osteogenesis Imperfecta (OI) Type 3.
- Trial name
- NCT05559801
- Testing
- Bone marrow-derived mesenchymal stromal cells (MSCs)
- Volunteers
- 12 people
- Sponsor
- Emory University
PKU, Hyperphenylalaninemia
Study of a National Cohort of Adult Patients With Phenylketonuria
Phenylketonuria (PKU) is a metabolic disease of genetic origin. This is a rare disease (incidence 1 / 16000 births) which is the subject of a systematic neonatal screening in France, because it is treatable by a diet low in…
- Trial name
- ECOPHEN (NCT01619722)
- Where
- 16 sites in France
- Volunteers
- 220 people
- Sponsor
- University Hospital, Tours
Congenital Funnel Chest
Metal Contamination After Minimally Invasive Repair of Pectus Excavatum (MIRPE)
The purpose of this study is to determine whether there is a metal wear debris after minimally invasive repair of pectus excavatum and if there's a clinical relevance.
- Trial name
- NCT04751266
- Testing
- minimally invasive repair of pectus excavatum
- Volunteers
- 28 people
- Sponsor
- Dr. Caroline Fortmann
Rare Diseases, Marfan Syndrome
Analysis of Muscular Properties in Patients With MFS and EDS
The goal of this observational study is to learn and assess muscle morphological and electromechanical properties in patients affected with Marfan syndrome (MFS) and Ehlers Danlos syndrome (EDS).
- Conditions
- Rare Diseases, Marfan Syndrome, Ehlers-Danlos Syndrome
- Trial name
- MUSCLE (NCT05720923)
- Testing
- Analysis with Ergometer
- Where
- Italy
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- IRCCS Policlinico S. Donato
Chronic Myeloproliferative Disorders, Leukemia
Ultraviolet-B Light Therapy and Allogeneic Stem Cell Transplantation in Treating Patients With Hematologic Malignancies
RATIONALE: Peripheral stem cell transplantation may be able to replace immune cells that were destroyed by chemotherapy. Sometimes the transplanted cells from a donor are rejected by the body's normal cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00068523
- Testing
- anti-thymocyte globulin, cyclophosphamide, cyclosporine
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Case Comprehensive Cancer Center
Proteus Syndrome, PIK3CA Related Overgrowth Spectrum
Study of Proteus Syndrome and Related Congenital Disorders
This study will examine rare congenital disorders that involve malformations and abnormal growth. It will focus on patients with Proteus syndrome, whose physical features are characterized by overgrowth, benign tumors of fatty…
- Trial name
- NCT00001403
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Cystic Fibrosis
Monocytes as Predictors of Cystic Fibrosis-related Bone Disease
Hypothesis: Circulating monocytes RANK and MCSF-R expression is predictive of Cystic Fibrosis-related Bone Disease. Study design: Single-center comparative cross-sectional study Population: Patients with a CFTR channel mutation…
- Trial name
- MucOs (NCT04877223)
- Testing
- No intervention
- Where
- France
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Université de Reims Champagne-Ardenne
Fructose Metabolism, Inborn Errors, Glycogen Storage Disease
Biomarker for Glycogen Storage Diseases (BioGlycogen)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Glycogen Storage Diseases from plasma. Testing for clinical robustness, specificity and long-term stability of the biomarker.
- Conditions
- Fructose Metabolism, Inborn Errors, Glycogen Storage Disease, Glycogen Storage Disease Type I, Glycogen Storage Disease Type II
- Trial name
- BioGlycogen (NCT02385162)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Leukemia
Azacitidine in Treating Patients With Newly Diagnosed Previously Untreated or Secondary Acute Myeloid Leukemia Who Are Unsuitable For Intensive Chemotherapy
RATIONALE: Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00739388
- Testing
- azacytidine
- Where
- 11 sites in Switzerland
- Volunteers
- 47 people
- Sponsor
- Swiss Cancer Institute
Osteogenesis Imperfecta, Osteoporosis
REMS25: Study on the Use of REMS Technology in Diseases Commonly Associated With Reduced Bone Mineral Density (BMD)
This study evaluates bone mineral density (BMD) in pediatric patients aged 5-18 years with conditions negatively affecting bone health, using REMS (Radiofrequency Echographic Multi Spectrometry), a non-invasive and radiation-free…
- Conditions
- Osteogenesis Imperfecta, Osteoporosis, Hypogonadisms, Neoplasia
- Trial name
- NCT07412782
- Testing
- REMS
- Where
- Italy
- Volunteers
- 100 people
- Sponsor
- Meyer Children's Hospital IRCCS
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Non-Hodgkin Lymphoma
Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)
This phase II Pediatric MATCH screening and multi-sub-trial studies how well treatment that is directed by genetic testing works in pediatric patients with solid tumors, non-Hodgkin lymphomas, or histiocytic disorders that have…
- Conditions
- Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Non-Hodgkin Lymphoma, Ann Arbor Stage IV Non-Hodgkin Lymphoma, Histiocytic Sarcoma
- Trial name
- NCT03155620
- Testing
- Biopsy Procedure, Biospecimen Collection, Bone Marrow Aspiration and Biopsy
- Where
- 172 sites in United States, Australia and 2 more
- Volunteers
- 1,377 people
- Sponsor
- National Cancer Institute (NCI)
Leukemia, Myelodysplastic Syndromes
Intensive Compared With Nonintensive Chemotherapy in Treating Older Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. It is not yet known if stronger doses of chemotherapy given over a longer period of time are as well…
- Conditions
- Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT00005823
- Testing
- cytarabine, daunorubicin hydrochloride, etoposide
- Where
- 3 sites in United Kingdom
- Volunteers
- 2,000 people
- Sponsor
- Leukemia Research Fund
Intellectual Disability, Neurodevelopmental Disorders
Physical Activity and Community EmPOWERment Project
Purpose: Conduct a wait-list randomized controlled trial (RCT) of an inclusive physical activity program called PACE for adults with intellectual disability (ID) who are not yet showing signs of Alzheimer's Disease…
- Conditions
- Intellectual Disability, Neurodevelopmental Disorders, Autism Spectrum Disorder, Down Syndrome
- Trial name
- PACE (NCT06740162)
- Testing
- PACE Program
- Where
- 2 sites in United States
- Volunteers
- 376 people, healthy volunteers welcome
- Sponsor
- University of North Carolina, Chapel Hill
Osteogenesis Imperfecta
An Efficacy and Safety Trial of Intravenous Zoledronic Acid in Infants Less Than One Year of Age, With Severe Osteogenesis Imperfecta
The investigators have currently finished conducting an international multi-center trial that compares the efficacy and safety of pamidronate and zoledronate in the treatment of moderate to severe forms of Osteogenesis Imperfecta…
- Trial name
- INFOI (NCT00982124)
- Testing
- Zoledronic Acid
- Where
- Canada
- Volunteers
- 14 people
- Sponsor
- Shriners Hospitals for Children
Periodontal Disease, Gingivitis and Periodontal Diseases
Salivary Biomarkers in Periodontal Disease Progression
This study aims to evaluate salivary biomarkers associated with bone remodeling during the transition from periodontal health to disease.
- Conditions
- Periodontal Disease, Gingivitis and Periodontal Diseases, Periodontitis
- Trial name
- PERIO-BIO (NCT07661784)
- Testing
- Saliva Sampling
- Where
- Turkey (Türkiye)
- Volunteers
- 240 people, healthy volunteers welcome
- Sponsor
- Suleyman Demirel University
Osteosarcoma
Development of Indices Predicting Response to Pre-operative Chemotherapy in Osteosarcoma Patients
The purpose of this study is to develop indices predicting response to pre-operative chemotherapy in osteosarcoma patients. Histologic response to pre-operative chemotherapy is very important for the ultimate outcome of…
- Trial name
- NCT00686738
- Where
- South Korea
- Volunteers
- 20 people
- Sponsor
- National Cancer Center, Korea
Scoliosis, Idiopathic Scoliosis
Validation of a New Optimized Nighttime Providence Brace for Personalized Treatment of Adolescent Idiopathic Scoliosis
Adolescent Idiopathic Scoliosis affects 3-4% of the Canadian population, of which about 10% will need a brace treatment during pubertal growth spurt.
- Trial name
- NCT05001568
- Testing
- Immediate in-brace correction: Conventional Brace, Immediate in-brace correction: Optimized Brace, Long-term bracing: Conventional Brace
- Where
- Canada
- Volunteers
- 58 people
- Sponsor
- St. Justine's Hospital
Hurler Syndrome, Fanconi Anemia
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem…
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01917708
- Testing
- Abatacept
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Emory University
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
Rebeccamycin Analog in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, Acute Lymphoblastic Leukemia, or Chronic Myelogenous Leukemia
This phase I trial is studying the side effects and best dose of rebeccamycin analog in treating patients with relapsed or refractory acute myeloid leukemia, myelodysplastic syndrome, acute lymphoblastic leukemia, or chronic…
- Conditions
- Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)
- Trial name
- NCT00087204
- Testing
- becatecarin, laboratory biomarker analysis
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Cancer Institute (NCI)
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Mechanical Stimulation in Preventing Bone Density Loss in Patients Undergoing Donor Stem Cell Transplant
This pilot clinical trial studies mechanical stimulation in preventing bone density loss in patients undergoing donor stem cell transplant.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01558778
- Testing
- management of therapy complications, musculoskeletal complications management/prevention
- Sponsor
- Roswell Park Cancer Institute
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