326. Osteopetrosis Clinical trials / Disease details


Clinical trials : 20 Drugs : 51 - (DrugBank : 14) / Drug target genes : 17 - Drug target pathways : 81

  
4 trials found
No.TrialIDDate_
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PhaseCountries
1EUCTR2014-000584-41-ES
(EUCTR)
08/06/201721/03/2017Phase 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 disordersPhase 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 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]
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
Bellicum Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
175Phase 2Spain;United Kingdom;Italy
2NCT02065869
(ClinicalTrials.gov)
April 201413/2/2014Safety 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 PharmaceuticalsNULLActive, not recruiting1 Month18 YearsAll193Phase 2Italy;United Kingdom;Germany;Spain;United States
3NCT03301168
(ClinicalTrials.gov)
April 201429/9/2017Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell TransplantPhase 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 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: RimiducidBellicum PharmaceuticalsNULLActive, not recruiting1 Month26 YearsAll120Phase 1/Phase 2United States;Italy
4EUCTR2014-000584-41-GB
(EUCTR)
15/07/2015Phase I/II study of CaspaCide T cells in children following aß- depleted mis-matched family donor stem cell transplantationPhase 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: 20.0;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: BPX-501 cells
INN or Proposed INN: Rivogenlecleucel
Other descriptive name: BPX-501
Product Name: rimiducid
INN or Proposed INN: Rimiducid
Other descriptive name: AP1903 A594
Bellicum Pharmaceuticals, Inc.NULLNAFemale: yes
Male: yes
175Phase 2Spain;Italy;United Kingdom