326. 大理石骨病 ［臨床試験数：16，薬物数：33（DrugBank：11），標的遺伝子数：15，標的パスウェイ数：55］
Searched query = "Osteopetrosis"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2016-003226-16-IT | 03/02/2017 | 5 February 2018 | Follow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR aß+ T cells in pediatric patients affected by hematological disorders | Follow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR aß+ T cells in pediatric patients affected by hematological disorders - CaspaCide TCR aß haplo HSCT | Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplastic syndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopathies) MedDRA version: 19.0 Level: HLGT Classification code 10018849 Term: Haematological disorders NEC System Organ Class: 10005329 - Blood and lymphatic system disorders ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: AP1903 Pharmaceutical Form: Solution for infusion CAS Number: 195514-63-7 Current Sponsor code: AP1903 Other descriptive name: AP1903 Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 0.4- | Bellicum Pharmaceuticals, Inc. | Authorised | Female: yes Male: yes | 175 | Phase 1;Phase 2 | Italy | |||
| 2 | NCT02666768 | February 22, 2016 | 15 July 2019 | ACTIMMUNE in Intermediate Osteopetrosis | Open-label Early Phase 2 Study With a Single Arm of Interferon Gamma-1b Treatment of Osteopetrosis | Osteopetrosis | Drug: Interferon gamma-1b | Los Angeles Biomedical Research Institute | University of Minnesota - Clinical and Translational Science Institute;Horizon Pharma Ireland, Ltd., Dublin Ireland | Not recruiting | 1 Year | N/A | All | 5 | Phase 2 | United States |
| 3 | NCT02584608 | January 2016 | 20 August 2018 | Use of ACTIMMUNE in Patients With ADO2 | Phase 2a Study of Interferon Gamma-1b for the Treatment of Autosomal Dominant Type 2 Osteopetrosis | Autosomal Dominant Osteopetrosis Type 2 | Drug: ACTIMMUNE | Indiana University | Horizon Pharma Ireland, Ltd., Dublin Ireland | Not recruiting | 3 Years | 65 Years | All | 12 | Phase 2 | United States |
| 4 | NCT02171104 | July 10, 2014 | 7 October 2019 | MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis | MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG | Mucopolysaccharidosis Disorders;Hurler Syndrome;Hunter Syndrome;Maroteaux Lamy Syndrome;Sly Syndrome;Alpha-Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Glycoprotein Metabolic Disorders;Sphingolipidoses;Recessive Leukodystrophies;Globoid Cell Leukodystrophy;Metachromatic Leukodystrophy;Niemann-Pick B;Niemann-Pick C Subtype 2;Sphingomyelin Deficiency;Peroxisomal Disorders;Adrenoleukodystrophy With Cerebral Involvement;Zellweger Syndrome;Neonatal Adrenoleukodystrophy;Infantile Refsum Disease;Acyl-CoA Oxidase Deficiency;D-Bifunctional Enzyme Deficiency;Multifunctional Enzyme Deficiency;Alpha-methylacyl-CoA Racmase Deficiency;Mitochondrial Neurogastrointestingal Encephalopathy;Severe Osteopetrosis;Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation);Inherited Metabolic Disorders | Biological: Stem Cell Transplantation;Drug: IMD Preparative Regimen;Drug: Osteopetrosis Only Preparative Regimen;Drug: Osteopetrosis Haploidentical Only Preparative Regimen;Drug: cALD SR-A (Standard-Risk, Regimen A);Drug: cALD SR-B (Standard-Risk, Regimen B);Drug: cALD HR-D (High-Risk, Regimen C);Drug: cALD HR-D (High-Risk, Regimen D) | Masonic Cancer Center, University of Minnesota | Recruiting | N/A | 55 Years | All | 100 | Phase 2 | United States | |
| 5 | NCT02065869 | April 2014 | 4 November 2019 | Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell Transplant | Phase II Extension Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological Disorders | Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndrome;Primary Immunodeficiency;Anemia, Aplastic;Osteopetrosis;Hemoglobinopathies;Cytopenia;Fanconi Anemia;Diamond Blackfan Anemia;Thalassemia;Anemia, Sickle Cell | Biological: BPX-501 T cells;Drug: rimiducid | Bellicum Pharmaceuticals | Not recruiting | 1 Month | 18 Years | All | 193 | Phase 1/Phase 2 | Italy;United Kingdom;Germany;Spain;United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT03301168 | April 2014 | 4 November 2019 | Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant | Phase II Extension Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological Disorders | Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndromes;Primary Immune Deficiency Disorder;Osteopetrosis;Cytopenia;Hemoglobinopathy in Children;Anemia, Aplastic | Biological: BPX-501 T cells;Drug: AP1903 | Bellicum Pharmaceuticals | Not recruiting | 1 Month | 26 Years | All | 120 | Phase 1/Phase 2 | United States;Italy | |
| 7 | NCT03333200 | January 11, 2012 | 11 November 2019 | Longitudinal Study of Neurodegenerative Disorders | Longitudinal Study of Neurodegenerative Disorders | MLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;Tay-Sachs Disease;NP Deficiency;Osteopetrosis;Alpha-Mannosidosis;Sandhoff Disease;Niemann-Pick Diseases;MPS IV;Gaucher Disease;GAN;GM1 Gangliosidoses;Morquio Disease;S-Adenosylhomocysteine Hydrolase Deficiency;Batten Disease;Pelizaeus-Merzbacher Disease;Leukodystrophy;Lysosomal Storage Diseases;Purine Nucleoside Phosphorylase Deficiency;Multiple Sulfatase Deficiency Disease | Other: Palliative Care;Biological: Hematopoetic Stem Cell Transplantation | University of Pittsburgh | Recruiting | N/A | N/A | All | 1500 | Phase 1 | United States | |
| 8 | NCT01200017 | September 2010 | 18 December 2018 | Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant | An Expanded Access Study of the Feasibility of Using the CliniMACS® Device for CD34+ Cell Selection and T Cell Depletion for Graft-versus-Host Disease Prophylaxis in Alternative Donor Stem Cell Transplant Recipients | Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Chronic Myeloid Leukemia;Myelodysplastic Syndrome;Lymphomas;Bone Marrow Failure;Hemoglobinopathy;Immune Deficiency;Osteopetrosis;Cytopenias;Leukocyte Disorders;Anemia Due to Intrinsic Red Cell Abnormality | Biological: CD34+ enriched, T Cell Depleted donor stem cell product | University of California, San Francisco | Not recruiting | 2 Months | 30 Years | All | Phase 2 | United States | ||
| 9 | NCT01087398 | September 2009 | 19 February 2015 | Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis | Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis | Osteopetrosis | Drug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen);Procedure: Stem Cell Transplantation;Drug: Cyclosporin, Methotrexate (GVHD prophylaxis) | Tehran University of Medical Sciences | Recruiting | N/A | 5 Years | Both | 10 | Phase 2/Phase 3 | Iran, Islamic Republic of | |
| 10 | NCT00775931 | August 2008 | 4 May 2015 | Allogeneic Transplantation For Severe Osteopetrosis | Allogeneic Hematopoietic Stem Cell Transplantation For Severe Osteopetrosis | Severe Osteopetrosis | Procedure: umbilical cord blood transplantation;Drug: Campath-1H;Radiation: Total Lymphoid Irradiation;Drug: Cyclophosphamide;Drug: Busulfan;Drug: Fludarabine monophosphate;Procedure: marrow graft transplantation | Masonic Cancer Center, University of Minnesota | Not recruiting | N/A | 45 Years | Both | 23 | Phase 2/Phase 3 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT00638820 | September 2007 | 19 October 2017 | Reduced Intensity AlloTransplant For Osteopetrosis | Reduced Intensity Allogeneic Transplantation For Severe Osteopetrosis Incorporating A Second Cd34 Selected Graft | Osteopetrosis | Procedure: Stem Cell or Umbilical Cord Blood Transplantation;Drug: Campath, Busulfan, Clofarabine;Procedure: Total Lymphoid Irradiation | Masonic Cancer Center, University of Minnesota | Not recruiting | N/A | 45 Years | All | 3 | Phase 2 | United States | |
| 12 | NCT00145587 | July 2004 | 16 December 2017 | Stem Cell Transplantation for Children Affected With Osteopetrosis | Allogeneic Hematopoietic Stem Cell Transplantation for Children Affected With Malignant Osteopetrosis: A Pilot Study | Osteopetrosis | Procedure: Stem Cell Transplantation;Device: Miltenyi Biotec CliniMACS;Drug: Systemic chemotherapy and antibodies | St. Jude Children's Research Hospital | Not recruiting | N/A | N/A | All | 15 | N/A | United States | |
| 13 | NCT00145886 | February 2003 | 18 January 2016 | rhPTH Therapy for Low Turnover Bone Fragility | Effect of 12 Months Treatment With rhPTH on Calcium Balance, Bone Turnover, Bone Mineral Density, and Bone Micro-architecture in Patients With Fractures Associated With Low Bone Turnover and Sclerosing Bone Disorders | Osteopetrosis | Drug: rhPTH | University of Chicago | Not recruiting | 20 Years | N/A | Both | 2 | Phase 1 | United States | |
| 14 | NCT01019876 | June 2002 | 19 February 2015 | Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases | Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant Diseases | Bone Marrow Failure;Osteopetrosis;Fanconi Anemia;Severe Combined Immunodeficiency | Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Cyclophosphamide 40;Drug: Cyclophosphamide 30 | Columbia University | Recruiting | N/A | 30 Years | Both | 50 | Phase 2/Phase 3 | United States | |
| 15 | NCT00043329 | January 2002 | 19 February 2015 | Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis | Post-Marketing Surveillance Study of Actimmune (Interferon Gamma-1b) in Patients With Severe Malignant Osteopetrosis | Osteopetrosis | Drug: Actimmune Registry | InterMune | Not recruiting | N/A | N/A | Both | 6 | Phase 4 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT00004402 | November 1999 | 7 April 2015 | Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis | Osteopetrosis | Drug: calcitriol;Drug: interferon gamma | FDA Office of Orphan Products Development | Medical University of South Carolina | Not recruiting | N/A | 10 Years | Both | 30 | Phase 3 |