326. Osteopetrosis Clinical trials / Disease details
Clinical trials : 18 / Drugs : 43 - (DrugBank : 14) / Drug target genes : 17 - Drug target pathways : 82
| 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 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02065869 (ClinicalTrials.gov) | April 2014 | 13/2/2014 | Safety Study of Gene Modified Donor T-cells Following TCRaß+ Depleted Stem Cell Transplant | Phase I/II Study of CaspaCIDe T Cells (BPX-501; Rivogenlecleucel) From an HLA Partially Matched Family Donor After Negative Selection of TCRaß+ T Cells in Paediatric Patients Affected by Haematological 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 | NULL | Active, not recruiting | 1 Month | 18 Years | All | 187 | Phase 1/Phase 2 | Italy;United Kingdom;Germany;Spain;United States |
| 2 | EUCTR2014-000584-41-GB (EUCTR) | 15/07/2015 | Phase I/II study of CaspaCide T cells in children following aß- depleted mis-matched family donor stem cell transplantation | 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: 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. | NULL | NA | Female: yes Male: yes | 175 | Phase 2 | Spain;Italy;United Kingdom |