326. 大理石骨病 Osteopetrosis Clinical trials / Disease details
臨床試験数 : 20 / 薬物数 : 51 - (DrugBank : 14) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 81
Showing 1 to 10 of 20 diseases
No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
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1 | NCT04525352 (ClinicalTrials.gov) | February 1, 2021 | 11/8/2020 | A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteope ... | A Phase I Clinical Trial for Gene Therapy in Infantile Malignant Osteopetrosis (IMO) to Evaluate the Safety and Preliminary Efficacy of Autologous CD34+ Enriched Cells Transduced With a LV Vector Encoding the TCIRG1 Gene A Phase I Clinical Trial for Gene Therapy in Infantile Malignant Osteopetrosis(IMO) to Evaluate the ... | Infantile Malignant Osteopetrosis | Biological: RP-L401 | Rocket Pharmaceuticals Inc. | California Institute for Regenerative Medicine (CIRM) | Active, not recruiting | 1 Month | N/A | All | 2 | Phase 1 | United States |
2 | EUCTR2014-000584-41-ES (EUCTR) | 08/06/2017 | 21/03/2017 | 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 Phase II extension study of CaspaCIDe T cells (BPX-501) from an HLA-partially matched family donor a ... | 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 - CaspaCide TCR aß haplo HSCT Phase II extension study of CaspaCIDe T cells (BPX-501) from an HLA-partially matched family donor a ... | Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplasticsyndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopathies) MedDRA version: 19.1;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] Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplasticsyndromes;Congenital immune def ... | Product Name: BPX-501 cells INN or Proposed INN: rivogenleucleucel Other descriptive name: BPX-501 Product Name: AP1903 INN or Proposed INN: Rimiducid Other descriptive name: AP1903 Product Name: BPX-501cells INN or Proposed INN: rivogenleucleucel Other descriptive name: BPX-501 Produ ... | Bellicum Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 175 | Phase 2 | Spain;United Kingdom;Italy | ||
3 | EUCTR2016-003226-16-IT (EUCTR) | 03/02/2017 | 17/11/2016 | 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 ... | 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 Follow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after ... | Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplasticsyndromes;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] Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplasticsyndromes;Congenital immune def ... | Product Name: AP1903 Other descriptive name: AP1903 | Bellicum Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 175 | Phase 1;Phase 2 | Italy | ||
4 | NCT02666768 (ClinicalTrials.gov) | February 22, 2016 | 23/6/2015 | 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 | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | University of Minnesota;Horizon Pharma Ireland, Ltd., Dublin Ireland | Completed | 1 Year | N/A | All | 5 | Phase 2 | United States |
5 | NCT02584608 (ClinicalTrials.gov) | January 1, 2016 | 20/10/2015 | 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 | Completed | 3 Years | 65 Years | All | 12 | Phase 2 | United States |
6 | EUCTR2014-000584-41-IT (EUCTR) | 23/10/2014 | 15/04/2014 | 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 Phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative sele ... | 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 Phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative sele ... | Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplasticsyndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopathies) MedDRA version: 16.1;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] Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplasticsyndromes;Congenital immune def ... | Product Name: BPX-501 cells Product Name: AP1903 | Bellicum Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 1;Phase 2 | Italy;United Kingdom;Spain | ||
7 | NCT02171104 (ClinicalTrials.gov) | July 10, 2014 | 20/6/2014 | 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 MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Sever ... | 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 Mucopolysaccharidosis Disorders;Hurler Syndrome;Hunter Syndrome;Maroteaux Lamy Syndrome;Sly Syndrome ... | 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) Biological: Stem Cell Transplantation;Drug: IMDPreparative Regimen;Drug: Osteopetrosis Only Preparat ... | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 55 Years | All | 100 | Phase 2 | United States |
8 | NCT03301168 (ClinicalTrials.gov) | April 2014 | 29/9/2017 | Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant ... | 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 Phase I/II Study of CaspaCIDe® T Cells From an HLA-Partially Matched Family Donor After Negative Sel ... | Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndromes;Primary Immune Deficiency Disorder;Osteopetrosis;Cytopenia;Hemoglobinopathy in Children;Anemia, Aplastic Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplas ... | Biological: BPX-501 T cells;Drug: Rimiducid | Bellicum Pharmaceuticals | NULL | Active, not recruiting | 1 Month | 26 Years | All | 120 | Phase 1/Phase 2 | United States;Italy |
9 | NCT02065869 (ClinicalTrials.gov) | April 2014 | 13/2/2014 | 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 Phase II Extension Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor A ... | 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 Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplas ... | Biological: BPX-501 T cells;Drug: rimiducid | Bellicum Pharmaceuticals | NULL | Active, not recruiting | 1 Month | 18 Years | All | 193 | Phase 2 | Italy;United Kingdom;Germany;Spain;United States |
10 | NCT03333200 (ClinicalTrials.gov) | January 11, 2012 | 24/4/2017 | 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 MLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;T ... | Other: Palliative Care;Biological: Hematopoetic Stem Cell Transplantation | University of Pittsburgh | NULL | Recruiting | N/A | N/A | All | 1500 | United States |