Clinical trials: Skeletal Dysplasia
Osteopetrosis
Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis
The purpose of this study is to establish a registry of all children with severe, malignant osteopetrosis who are treated with Actimmune (IFN-g 1b or Interferon gamma-1b) to monitor the effects of IFN-g 1b on preventing…
- Trial name
- NCT00043329
- Testing
- Actimmune Registry
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- InterMune
Lung Cancer (Diagnosis), Osteochondroma
Clonal Hematopoiesis Chemotherapy and Radiation Effects Study
The goal of the Clonal Hematopoiesis Chemotherapy and Radiation Effects (CH CARE) Study is to understand how the presence or absence of clonal hematopoiesis (CH) influences outcomes in people receiving chemotherapy and radiation…
- Conditions
- Lung Cancer (Diagnosis), Osteochondroma, Spitz Nevus, Solid Cancers
- Trial name
- CH CARE (NCT07675967)
- Testing
- Samples
- Where
- United States
- Volunteers
- 5,000 people
- Sponsor
- Dana-Farber Cancer Institute
Hepatocellular Carcinoma
A Study of LY2157299 in Participants With Advanced Hepatocellular Carcinoma
The main purpose of this study is to evaluate the safety and effectiveness of the study drug known as LY2157299 in participants with hepatocellular carcinoma.
- Trial name
- NCT02178358
- Testing
- LY2157299, Sorafenib
- Where
- 16 sites in China, Hong Kong and 3 more
- Volunteers
- 132 people
- Sponsor
- Eli Lilly and Company
Bone Marrow Failure, Osteopetrosis
Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases
This study proposes the use of a reduced intensity chemotherapy/radiation therapy regimen followed by stem cell transplantation, as compared to standard ablative chemotherapy regimens associated with stem cell transplantation, in…
- Conditions
- Bone Marrow Failure, Osteopetrosis, Fanconi Anemia, Severe Combined Immunodeficiency
- Trial name
- NCT01019876
- Testing
- Fludarabine, Cyclophosphamide, Busulfan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Columbia University
Proliferative Vitreoretinopathy in Rhegmatogenous Retinal Detachment
Intravitreal Triamcinolone and Biomarkers in Rhegmatogenous Retinal Detachment
This prospective, randomized, controlled clinical study evaluates the effects of preoperative intravitreal triamcinolone acetonide (TA) on growth factors and inflammatory mediators in patients with rhegmatogenous retinal…
- Trial name
- NCT07774182
- Testing
- Standard Vitrectomy, Triamcinolone acetonide (TA)
- Where
- Indonesia
- Volunteers
- 60 people
- Sponsor
- Anggun Rama Yudantha
McCune Albright Syndrome, Polyostotic Fibrous Dysplasia
Effects of Letrozole on Precocious Puberty Due to McCune Albright Syndrome
This study will test the safety and effectiveness of letrozole in treating precocious (early) puberty in girls with McCune-Albright syndrome (MAS).
- Conditions
- McCune Albright Syndrome, Polyostotic Fibrous Dysplasia, Precocious Puberty
- Trial name
- NCT00006174
- Testing
- Letrozole
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
Osteogenesis Imperfecta
A First-in-Human Study Evaluating AGA2115 in Adult Healthy Volunteers
To understand if AGA2115 is safe and well tolerated in healthy adult volunteers.
- Trial name
- NCT06086613
- Testing
- AGA2115
- Where
- United States
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Angitia Incorporated Limited
Osteogenesis Imperfecta
Epigenetic Regulation of Osteogenesis Imperfecta Severity : miROI Study
Osteogenesis Imperfecta (OI) is a heterogeneous group of rare connective tissue hereditary diseases responsible for fragility and bone deformity.
- Trial name
- miROI (NCT04009733)
- Testing
- Blood sample
- Where
- France
- Volunteers
- 66 people
- Sponsor
- Hospices Civils de Lyon
Achondroplasia
Lifetime Impact Study for Achondroplasia
Observational study looking at the burden of illness in achondroplasia subjects aged 3 and above. The study will include a 3 year review of historical clinical data as well as a single point collection of questionnaire data to…
- Trial name
- LISA (NCT03872531)
- Where
- 4 sites in Argentina, Brazil and 1 more
- Volunteers
- 173 people
- Sponsor
- BioMarin Pharmaceutical
Exostoses, Multiple Hereditary
Gene Mutations and Orthopaedic Symptoms Correlation of Multiple Hereditary Exostoses: Multicentre Project
The investigator's goal for this project is to examine the causes of the wide variability of the expression of Hereditary Multiple Exostoses (HME).
- Trial name
- NCT00474331
- Where
- Canada
- Volunteers
- 2,000 people
- Sponsor
- University of British Columbia
Osteogenesis Imperfecta
An Exploratory Study of BPS804 Treatment in Adult Patients With Type I, III or IV Osteogenesis Imperfecta
The purpose of this study is to investigate the effect of BPS804 on strength/quality of bone in patients with Type I, III or IV Osteogenesis imperfecta using a special type of CT scanner. Participants will be treated for 1 year.
- Trial name
- Meteoroid (NCT03216486)
- Testing
- BPS804
- Where
- 2 sites in United States
- Sponsor
- Ultragenyx Pharmaceutical Inc
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)
Bone Marrow Diseases
Cytokine Gene Polymorphisms in Bone Marrow Failure
This study will examine whether cytokine gene polymorphisms affect the progression or response to therapy of bone marrow failure disorders.
- Trial name
- NCT00085670
- Where
- United States
- Volunteers
- 79 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Hypochondroplasia
Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia
Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with…
- Trial name
- NCT07393373
- Testing
- Infigratinib
- Where
- 24 sites in United States, Australia and 8 more
- Volunteers
- 135 people
- Sponsor
- QED Therapeutics, a BridgeBio company