65. Primary immunodeficiency Clinical trials / Disease details

Clinical trials : 482 / Drugs : 653 - (DrugBank : 119) / Drug target genes : 92 - Drug target pathways : 212

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	NCT04965597 (ClinicalTrials.gov)	March 2022	21/6/2021	Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)	Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases	Bone Marrow Failure Syndrome;Congenital Amegakaryocytic Thrombocytopenia;Congenital Pure Red Cell Aplasia;Hereditary Sideroblastic Anemia;Myeloid Neoplasms With Germline GATA2 Mutation;Paroxysmal Nocturnal Hemoglobinuria;Shwachman-Diamond Syndrome	Drug: Treosulfan;Drug: Fludarabine Phosphate;Drug: Tacrolimus;Drug: Methotrexate;Biological: Lapine T-Lymphocyte Immune Globulin;Procedure: Peripheral Blood Stem Cell Transplantation;Procedure: Allogeneic Bone Marrow Transplantation;Other: Quality-of-Life Assessment	Fred Hutchinson Cancer Research Center	Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI)	Recruiting	1 Year	49 Years	All	40	Phase 2	United States
2	NCT00885833 (ClinicalTrials.gov)	February 2007	21/4/2009	Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS)	Phase I/II Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome	Wiskott-Aldrich Syndrome	Drug: Fludarabine , Busulfan, Thymoglobulin	The Korean Society of Pediatric Hematology Oncology	NULL	Completed	1 Year	25 Years	Both	5	Phase 1/Phase 2	Korea, Republic of