Phase 3 N=7 Allogeneic Transplantation For Severe Osteopetrosis
Severe Osteopetrosis
Primary: Number of Patients Who Achieved Donor Cell Engraftment — 5; 0 Participants
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
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
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
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=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
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
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=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 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
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
N/A N=28 Sepsis-Associated Purpura Fulminans International Registry - Europe
Sepsis
Primary: Mortality — 11 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
N/A N=48 A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
Hypophosphatasia (HPP)
Primary: Survival — 270.5 days
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=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=424 A Severity-Adapted Clinical Trial of Diminished Bone Mineral Density in Acute Lymphoblastic Leukemia Survivors
Leukemia, Lymphoblastic, Acute · Osteoporosis
Primary: Effect of Taking Calcium and Vitamin D Supplements on Bone Mineral Density (BMD) — -0.95; -0.97; -0.87; -1.04 Z-Score — p=0.86
N/A N=61 Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Dyskeratosis Congenita · Aplastic Anemia
Primary: Incidence of Neutrophil Engraftment — 100; 100; 96; 100 Percentage of participants
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
N/A N=545 Patterns of Care and Outcomes in Patients With Metastatic Bone Tumors (METABONE)
Metastatic Bone Tumor
Primary: Overall Survival (OS) — 21.5; 12.7; 26.6 months
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 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
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
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
Phase 2 N=47 Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)
Hemophagocytic Lymphohistiocytosis · Chronic Active Epstein-Barr Virus Infection · Chronic Granulomatous Disease
Primary: Percentage of Participants With Overall Survival (OS) — 80.4; 66.7 percentage of 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 4 N=14 Imaging of Osteonecrosis With Ferumoxytol-Enhanced MRI
Osteonecrosis
Primary: Signal to Noise Ratio (SNR) — 33.82; 129.56 dB — p=0.002
N/A N=262 Acinetobacter Baumannii-related Osteomyelitis: Clinical and Epidemiological Characterization
Osteomyelitis
Primary: Description of the Clinical and Epidemiological Profile of Patients With Infection — 92; 26; 25; 21 Participants
Phase 3 N=68 Bone Properties in Hypoparathyroidism: Effects of PTH
Hypoparathyroidism
Primary: Requirements for Calcium Supplementation — 3.03; 1.66 grams per day of calcium supplementation
Phase 2 N=6 Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
Sickle Cell Disease · Thalassemia · Diamond-Blackfan Anemia
Primary: Count of Participants With Stable Engraftment Post Hematopoietic Cell Transplantation (HCT) — 3 Participants
Phase 2 N=21 Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia
Severe Thalassemia
Primary: Primary Objective: Event-free Survival at 1 Year. — 17 Participants