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 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:19] 

Searched query = "Osteopetrosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
1EUCTR2016-003226-16-IT03/02/20175 February 2018Follow-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 disordersFollow-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 HSCTHematological 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.AuthorisedFemale: yes
Male: yes
175Phase 1;Phase 2Italy
2NCT02666768February 22, 201615 July 2019ACTIMMUNE in Intermediate OsteopetrosisOpen-label Early Phase 2 Study With a Single Arm of Interferon Gamma-1b Treatment of OsteopetrosisOsteopetrosisDrug: Interferon gamma-1bLos Angeles Biomedical Research InstituteUniversity of Minnesota - Clinical and Translational Science Institute;Horizon Pharma Ireland, Ltd., Dublin IrelandNot recruiting1 YearN/AAll5Phase 2United States
3NCT02584608January 201620 August 2018Use of ACTIMMUNE in Patients With ADO2Phase 2a Study of Interferon Gamma-1b for the Treatment of Autosomal Dominant Type 2 OsteopetrosisAutosomal Dominant Osteopetrosis Type 2Drug: ACTIMMUNEIndiana UniversityHorizon Pharma Ireland, Ltd., Dublin IrelandNot recruiting3 Years65 YearsAll12Phase 2United States
4NCT02171104July 10, 20147 October 2019MT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisMT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATGMucopolysaccharidosis 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 DisordersBiological: 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 MinnesotaRecruitingN/A55 YearsAll100Phase 2United States
5NCT02065869April 20144 November 2019Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell TransplantPhase 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 DisordersAcute 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 CellBiological: BPX-501 T cells;Drug: rimiducidBellicum PharmaceuticalsNot recruiting1 Month18 YearsAll193Phase 1/Phase 2Italy;United Kingdom;Germany;Spain;United States
No.TrialIDDate_
enrollement
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
6NCT03301168April 20144 November 2019Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell TransplantPhase 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 DisordersAcute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndromes;Primary Immune Deficiency Disorder;Osteopetrosis;Cytopenia;Hemoglobinopathy in Children;Anemia, AplasticBiological: BPX-501 T cells;Drug: AP1903Bellicum PharmaceuticalsNot recruiting1 Month26 YearsAll120Phase 1/Phase 2United States;Italy
7NCT03333200January 11, 201211 November 2019Longitudinal Study of Neurodegenerative DisordersLongitudinal Study of Neurodegenerative DisordersMLD;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 DiseaseOther: Palliative Care;Biological: Hematopoetic Stem Cell TransplantationUniversity of PittsburghRecruitingN/AN/AAll1500Phase 1United States
8NCT01200017September 201018 December 2018Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell TransplantAn 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 RecipientsAcute 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 AbnormalityBiological: CD34+ enriched, T Cell Depleted donor stem cell productUniversity of California, San FranciscoNot recruiting2 Months30 YearsAllPhase 2United States
9NCT01087398September 200919 February 2015Hematopoietic Stem Cell Transplantation for Malignant Infantile OsteopetrosisHematopoietic Stem Cell Transplantation for Malignant Infantile OsteopetrosisOsteopetrosisDrug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen);Procedure: Stem Cell Transplantation;Drug: Cyclosporin, Methotrexate (GVHD prophylaxis)Tehran University of Medical SciencesRecruitingN/A5 YearsBoth10Phase 2/Phase 3Iran, Islamic Republic of
10NCT00775931August 20084 May 2015Allogeneic Transplantation For Severe OsteopetrosisAllogeneic Hematopoietic Stem Cell Transplantation For Severe OsteopetrosisSevere OsteopetrosisProcedure: umbilical cord blood transplantation;Drug: Campath-1H;Radiation: Total Lymphoid Irradiation;Drug: Cyclophosphamide;Drug: Busulfan;Drug: Fludarabine monophosphate;Procedure: marrow graft transplantationMasonic Cancer Center, University of MinnesotaNot recruitingN/A45 YearsBoth23Phase 2/Phase 3United States
No.TrialIDDate_
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11NCT00638820September 200719 October 2017Reduced Intensity AlloTransplant For OsteopetrosisReduced Intensity Allogeneic Transplantation For Severe Osteopetrosis Incorporating A Second Cd34 Selected GraftOsteopetrosisProcedure: Stem Cell or Umbilical Cord Blood Transplantation;Drug: Campath, Busulfan, Clofarabine;Procedure: Total Lymphoid IrradiationMasonic Cancer Center, University of MinnesotaNot recruitingN/A45 YearsAll3Phase 2United States
12NCT00145587July 200416 December 2017Stem Cell Transplantation for Children Affected With OsteopetrosisAllogeneic Hematopoietic Stem Cell Transplantation for Children Affected With Malignant Osteopetrosis: A Pilot StudyOsteopetrosisProcedure: Stem Cell Transplantation;Device: Miltenyi Biotec CliniMACS;Drug: Systemic chemotherapy and antibodiesSt. Jude Children's Research HospitalNot recruitingN/AN/AAll15N/AUnited States
13NCT00145886February 200318 January 2016rhPTH Therapy for Low Turnover Bone FragilityEffect 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 DisordersOsteopetrosisDrug: rhPTHUniversity of ChicagoNot recruiting20 YearsN/ABoth2Phase 1United States
14NCT01019876June 200219 February 2015Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant DiseasesRisk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant DiseasesBone Marrow Failure;Osteopetrosis;Fanconi Anemia;Severe Combined ImmunodeficiencyDrug: Fludarabine;Drug: Cyclophosphamide;Drug: Cyclophosphamide 40;Drug: Cyclophosphamide 30Columbia UniversityRecruitingN/A30 YearsBoth50Phase 2/Phase 3United States
15NCT00043329January 200219 February 2015Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant OsteopetrosisPost-Marketing Surveillance Study of Actimmune (Interferon Gamma-1b) in Patients With Severe Malignant OsteopetrosisOsteopetrosisDrug: Actimmune RegistryInterMuneNot recruitingN/AN/ABoth6Phase 4United States
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
16NCT00004402November 19997 April 2015Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital OsteopetrosisOsteopetrosisDrug: calcitriol;Drug: interferon gammaFDA Office of Orphan Products DevelopmentMedical University of South CarolinaNot recruitingN/A10 YearsBoth30Phase 3

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