284. Diamond-Blackfan anemia Clinical trials / Disease details

Clinical trials : 37 / Drugs : 110 - (DrugBank : 34) / Drug target genes : 23 - Drug target pathways : 124

2 trials found

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	NCT03733249 (ClinicalTrials.gov)	January 2017	2/11/2018	Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study	Follow-up of Phase 1/2 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 Immunodeficiency;Anemia, Aplastic;Hemoglobinopathies;Cytopenia;Fanconi Anemia;Diamond Blackfan Anemia;Thalassemia;Anemia, Sickle Cell	Drug: Rimiducid;Biological: rivogenlecleucel	Bellicum Pharmaceuticals	NULL	Enrolling by invitation	1 Month	18 Years	All	193	Phase 1/Phase 2	Italy;Saudi Arabia;United Kingdom
2	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	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	193	Phase 2	Italy;United Kingdom;Germany;Spain;United States