Phase 2 N=12 Use of ACTIMMUNE in Patients With ADO2
Autosomal Dominant Osteopetrosis Type 2
Primary: Changes in Bone Resorption Markers From Baseline to 14 Weeks. — 2.2; -2.1 percentage of change
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 3 N=7 Allogeneic Transplantation For Severe Osteopetrosis
Severe Osteopetrosis
Primary: Number of Patients Who Achieved Donor Cell Engraftment — 5; 0 Participants
Phase 3 N=34 Pamidronate to Treat Osteogenesis Imperfecta in Children
Osteogenesis Imperfecta
Primary: Change in Bone Mineral Density in Response to Pamidronate — -3.54; -4.98 Z-score
Phase 2 N=5 ACTIMMUNE in Intermediate Osteopetrosis
Osteopetrosis
Primary: Number of Participants With Treatment Related Adverse Events CTCAE v4.0 Grade 3 or Higher — 0 Participants
N/A N=29 Calcium Absorption in Patients With Rothmund-Thomson Syndrome
Rothmund-Thomson Syndrome
Primary: Bone Density (Low Areal Bone Mineral Density (aBMD)) — -1.3; -0.9; -2.4; -1.2 z-score
Phase 1 N=11 Safety of Fresolimumab in the Treatment of Osteogenesis Imperfecta
Osteogenesis Imperfecta
Primary: Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0 — 2; 4; 2; 1 Participants
N/A N=26 Trial of Lithium Carbonate for Treatment of Osteoporosis-pseudoglioma Syndrome
Osteoporosis Pseudoglioma
Primary: pQCT of Lower Leg — -2.19; 0.04; -2.09; -0.30 Z-score — p=0.0003
Phase 2 N=112 A Study in Adult Patients With Type I, III or IV Osteogenesis Imperfecta Treated With BPS804
Osteogenesis Imperfecta, Type I · Osteogenesis Imperfecta Type III · Osteogenesis Imperfecta Type IV
Primary: Change From Baseline in Radial Trabecular Volumetric Bone Mineral Density (Tr vBMD) at Month 12 — 1.004; 0.993; 0.992 ratio — p=0.644
Phase 3 N=143 Safety and Efficacy of Risedronate in the Treatment of Osteogenesis Imperfecta in Children
Osteogenesis Imperfecta
Primary: Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 12, ITT Population — 7.592; 16.159 Percent Change — p=<0.0001
N/A N=22 Prevention of Post Operative Bone Loss in Children
Osteoporosis · Cerebral Palsy · Spina Bifida
Primary: Amount of Bone Density Lost (%) From pre-to Post-operative DXA Scan — 0.043; -0.025 percent change
Phase 2 N=58 An Open-Label Extension Study of Palovarotene Treatment in Fibrodysplasia Ossificans Progressiva (FOP)
Fibrodysplasia Ossificans Progressiva
Primary: Parts A and B: Percentage of Flare-ups With No New Heterotopic Ossification (HO) at Week 12 — 64.3; 72.5 percentage of flare-ups
Phase 4 N=18 Bisphosphonate Therapy for Osteogenesis Imperfecta
Osteogenesis Imperfecta · Osteoporosis · Paget Disease of Bone
Primary: Bone Mineral Density — -1.3; -1.1 z-score
Phase 4 N=78 Study of Teriparatide (FORTEO) to Treat Adults With Osteogenesis Imperfecta
Osteogenesis Imperfecta
Primary: Spine Bone Mineral Density (BMD) — 6.1; 2.8 percentage of change in g/cm2
Phase 3 N=221 An Efficacy and Safety Study of Palovarotene for the Treatment of Fibrodysplasia Ossificans Progressiva.
Fibrodysplasia Ossificans Progressiva
Primary: Annualized New Heterotopic Ossification (HO) — 9427.1; 23720.2 cubic millimeters (mm^3)
Phase 2 N=38 HSCT for High Risk Inherited Inborn Errors
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
Phase 2 N=10 Phase II Study of Alendronate Sodium in Children With High-Turnover Idiopathic Juvenile Osteoporosis
Osteoporosis
Primary: Number of Participants With Increased Bone Mineral Density — 10 participants
Phase 3 N=42 Growth Hormone Therapy in Osteogenesis Imperfecta
Osteogenesis Imperfecta
Primary: Proportion of Subjects Who Met Criteria of Increase in Growth Rate Since Baseline. — 25 Participants
Phase 2 N=63 Prevention of Bone Loss After Pediatric Hematopoietic Cell Transplantation
Osteopenia · Osteoporosis
Primary: Lumbar Spine Bone Mineral Content — 41.4; 38.7 Average grams
Phase 4 N=12 Growth and Development Study of Alglucosidase Alfa
Pompe Disease · Glycogen Storage Disease Type II (GSD-II) · Acid Maltase Deficiency Disease
Primary: Recumbent Height/Length of Participants in Centimeters (cm) — 80.4; 93.8; 67.7; 91.1 cm
Phase 2 N=40 An Efficacy and Safety Study of Palovarotene to Treat Preosseous Flare-ups in FOP Subjects
Fibrodysplasia Ossificans Progressiva
Primary: Percentage of Responders at Week 6 — 100; 88.9; 88.9 percentage of subjects — p=0.1664
N/A N=10 Effect of Thiazolidinediones on Human Bone
Osteoblast · Adipocytes · Bone Density
Primary: Percent Change in Number of Osteoblast and Adipocyte Colony Forming Units Cultured From Bone Marrow Stem Cells Harvested 6 Months After Treatment With Study Drug…
Phase 2 N=41 Forteo Trial on Idiopathic Osteoporosis in Premenopausal Women
Adult Idiopathic Generalized Osteoporosis
Primary: Change in Lumbar Spine Bone Mineral Density (LS-BMD) on Active Medication — 10.1; 4.1 percentage of change
Phase 3 N=13 A Long-term Administration Study of OPC-41061 in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD) (2) [Extension of Study 156-05-002]
Autosomal Dominant Polycystic Kidney Disease
Primary: Total Kidney Volume — 1428; 1675; 1728; 1775 mL
Phase 2 N=59 Zoledronate in Preventing Osteoporosis in Patients With Primary Malignant Glioma
Brain Tumors · Osteoporosis · Central Nervous System(CNS)Malignancies
Primary: Percent of Patients With Change in Combined Bone Mass Density T-score <= -0.5. — 3.7; 10.5 percentage of patients
N/A N=14 Post-market Observational Study on JTIN Telescopic Nail in Osteogenesis Imperfecta Pediatric Patients
Osteogenesis Imperfecta
Primary: Percentage of Procedures With at Least One Serious/Not Serious Adverse Event Certainly or Possibly Related to JTIN — 4.0 percentage of procedures
Phase 3 N=24 Safety and Efficiency of Denosumab in Pediatric Subjects With Glucocorticoid-induced Osteoporosis
Evaluate the Safety and Efficacy of Denosumab in Pediatric Subjects With · Glucocorticoid-induced Osteoporosis
Primary: Change From Baseline in Lumbar Spine BMD Z-score as Assessed by Dual-energy X-ray Absorptiometry (DXA) at 12 Months — 0.23; 0.11 Z-score — p=0.68
Phase 2 N=33 Denosumab for Prevention of Post-Teriparatide Bone Loss in Premenopausal Women With IOP
Adult Idiopathic Generalized Osteoporosis
Primary: Percent Change in Lumbar Spine Areal BMD by DXA — 5.2 percentage of change
N/A N=144 OPALINE : A Study Of Morbidity And Mortality At 2 Years
Pancreatic Neuroendocrine Tumor, Well Differentiated and Progressive
Primary: Progression-Free Survival (PFS) at 2 Years Assessed by Investigator Per Response Evaluation Criteria in Solid Tumors (RECIST) Version (v) 1.1 - Based on Type of…
Phase 4 N=21 Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease
Gaucher Disease
Primary: Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-Score up to End of Study (EOS) (Week 103) — 0.17 Z-score
N/A N=3 Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
Lysosomal Storage Disease · Peroxisomal Disorder
Primary: Donor (Allogeneic) Hematopoietic Engraftment — 1 participants