Clinical trials: Skeletal Dysplasia
Genetic Disorder, Achondroplasia
Special Survey for Long Term Application
This study is conducted in Japan. The aim of this study is to assess the incidence rate of adverse drug reactions (ADRs) when using somatropin (Norditropin®) for treatment of for achondroplasia without epiphyseal line closure…
- Trial name
- NCT01516229
- Testing
- somatropin
- Where
- 2 sites in Japan
- Volunteers
- 395 people
- Sponsor
- Novo Nordisk A/S
Pancreatogenic Diabetes Mellitus, Type 2 Diabetes Mellitus (T2DM)
Assessment of Pancreatic Dysfunction in Patients With Type 2 Diabetes
The goal of this observational study is to better understand how the pancreas works in adults with type 2 diabetes. The study focuses on both hormone production (endocrine function) and digestive function (exocrine function) of…
- Conditions
- Pancreatogenic Diabetes Mellitus, Type 2 Diabetes Mellitus (T2DM), Exocrine Pancreatic Insufficiency (EPI)
- Trial name
- T3cDM-AKT (NCT07512934)
- Where
- Kazakhstan
- Volunteers
- 310 people
- Sponsor
- West Kazakhstan Marat Ospanov Medical University
Puberty, Precocious, McCune-Albright Syndrome
Faslodex in McCune-Albright Syndrome
The purpose of this study is to evaluate the safety, effectiveness and pharmacokinetics of a study drug called Faslodex (fulvestrant) in the treatment of progressive precocious puberty (PPP) (early puberty) in girls with…
- Trial name
- FMAS (NCT00278915)
- Testing
- Fulvestrant
- Where
- 16 sites in United States, France and 4 more
- Volunteers
- 30 people
- Sponsor
- AstraZeneca
Osteogenesis Imperfecta
Safety of Fresolimumab in the Treatment of Osteogenesis Imperfecta
Osteogenesis Imperfecta (OI) is a rare disorder that causes bones to break easily. People with OI may have broken bones with little or no trauma, dentinogenesis imperfecta (DI), and, in adult years, hearing loss.
- Trial name
- NCT03064074
- Testing
- Fresolimumab
- Where
- 2 sites in United States
- Volunteers
- 11 people
- Sponsor
- Baylor College of Medicine
Heart Transplant Rejection, Heart Transplant Infection
TTV Viral Load in Heart Transplant Recipients
This prospective, multicenter, non-interventional trial aims to study the association between TTV viral load and the occurrence of rejection or infection during the first year after transplantation.
- Conditions
- Heart Transplant Rejection, Heart Transplant Infection, Virus
- Trial name
- TTVCoeur (NCT05064462)
- Testing
- Collection of EDTA blood sample (5 to 7 ml)
- Where
- 3 sites in France
- Volunteers
- 60 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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, 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
Chronic Rejection in Lung Transplant, Cytokine Production in Bos Post Lung Transplant
Early Indicators of Chronic Rejection in Lung Transplant
The purpose of this research is to discover the different patterns of cytokine production in patients who may develop Bronchiolitis Obliterans Syndrome-0p (BOS-0p) which means ongoing rejection.
- Trial name
- BOS (NCT00449332)
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- University of Chicago
Glomerulonephritis, IGA, Hypertension
A Prospective Study of the Kidney Protective Effect of Aliskiren in Hypertensive Patients With IgA Nephropathy
The purpose of this study is to evaluate the effect of aliskiren, a novel direct rennin inhibitor, on renal function and progress of renal disease in hypertensive patients with IgA nephropathy.
- Trial name
- NCT01184599
- Testing
- aliskiren
- Where
- 4 sites in Japan
- Volunteers
- 30 people
- Sponsor
- Kagawa University
Fibrous Dysplasia/McCune-Albright Syndrome
Pain in Fibrous Dysplasia
Pain remains a common and frequently debilitating symptom, particularly during adulthood of Fibrous Dysplasia/McCune-Albright Syndrome (FD/MAS).
- Trial name
- NCT04125862
- Testing
- MRI-based Neuroimaging, Non-contrast MRI, 18F-FDG-PET/CT
- Where
- United States
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Rhizomelic Chondrodysplasia Punctata
A First-in-Human Phase 1 Study of Plasmalogen Precursor PPI-1011 in Healthy Adult Volunteers to Assess Safety, Tolerability, and Pharmacokinetics
PPI-1011 is being developed as a docosahexaenoic acid (DHA) containing plasmalogen precursor with good long-term stability, specifically for the treatment of rhizomelic chondrodysplasia punctata (RCDP), which is an ultra rare…
- Trial name
- NCT05969977
- Testing
- PPI-1011
- Where
- Canada
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- MED-LIFE DISCOVERIES LP
Genetic Disorder, Achondroplasia
A Survey Collecting Data on Adult Height in Patients With Achondroplasia Treated With Somatropin
This study is conducted in Japan. The aim of the study is to evaluate the efficacy of somatropin (Norditropin®) on adult height (cm) in patients with achondroplasia / hypochondroplasia enrolled in the GH-1941 study (NCT01516229).
- Trial name
- NCT01435629
- Testing
- somatropin
- Where
- Japan
- Volunteers
- 81 people
- Sponsor
- Novo Nordisk A/S
Rheumatoid Arthritis, Osteoarthritis
A Single Centre Study to Assess the Long-term Performance of the Pinnacle™ Cup With a Ceramic-on-ceramic Bearing in Primary Total Hip Replacement
The purpose of this study is to monitor the performance of the Pinnacle™ Cup with a ceramic-on-ceramic bearing combination in the treatment of patients with hip joint disease requiring a total hip replacement.
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Post-traumatic Arthritis, Collagen Disorders
- Trial name
- NCT00872222
- Testing
- Pinnacle™ Acetabular System
- Where
- Czechia
- Volunteers
- 98 people
- Sponsor
- DePuy International
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
Lupus Nephritis (LN)
The Role of Transforming Growth Factor- Beta (TGF- β) Expression in Renal Biopsy of Lupus Nephritis Patients
To investigate the possible role of TGF-β expression in renal biopsy and it's relation with fibrosis, activity and chronicity indices
- Trial name
- NCT07823556
- Testing
- renal biopsy
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- Ain Shams University
Slipped Capital Femoral Epiphyses, SCFE
SCFE Longitudinal International Prospective Registry
Slipped Capital Femoral Epiphysis (SCFE) is the most common disorder of the adolescent hip and diagnosis and treatment of SCFE remain areas of controversy and investigation.
- Trial name
- SLIP (NCT04117841)
- Testing
- Observational
- Where
- Canada
- Volunteers
- 2,000 people
- Sponsor
- University of British Columbia
Bone Diseases, Bone Cysts
Safety and Efficacy of Non-Setting Paste in Bone Defect Reconstruction
This study aims to evaluate the safety and efficacy of a novel bone substitute material for the treatment of bone defects in patients requiring defect filling following the excision of benign bone tumors, cysts, or similar…
- Conditions
- Bone Diseases, Bone Cysts, Bone Neoplasm, Enchondromatosis
- Trial name
- NCT07379008
- Testing
- Non-setting paste
- Where
- Poland
- Volunteers
- 29 people
- Sponsor
- Biovico Sp. z o.o.
Achondroplasia
A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
The purpose of this study is to measure how much of the study drug gets into the blood- stream and how long it takes the body to get rid of it when given as a single dose.
- Trial name
- NCT01590446
- Testing
- BMN 111, Normal Saline
- Where
- United States
- Volunteers
- 74 people, healthy volunteers welcome
- Sponsor
- BioMarin Pharmaceutical
Osteogenesis Imperfecta
Growth Hormone Therapy in Osteogenesis Imperfecta
Growth deficiency is a key feature of severe Osteogenesis Imperfecta (OI) and a frequent feature of mild to moderate forms of the disease. The reason that children with OI are short is not fully understood.
- Trial name
- NCT00001305
- Testing
- Humatrope
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)