65. 原発性免疫不全症候群 Primary immunodeficiency Clinical trials / Disease details

臨床試験数 : 500 / 薬物数 : 614 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 217

31 trials found

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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)	April 19, 2022	21/6/2021	Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of ...	Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Mar ...	Bone Marrow Failure Syndrome;Congenital Amegakaryocytic Thrombocytopenia;Congenital Pure Red Cell Ap ...	Drug: Treosulfan;Drug: Fludarabine Phosphate;Drug: Tacrolimus;Drug: Methotrexate;Biological: Lapine T ...	Fred Hutchinson Cancer Center	Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow ...	Recruiting	1 Year	49 Years	All	40	Phase 2	United States
2	NCT03597594 (ClinicalTrials.gov)	September 8, 2021	19/6/2018	Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infus ...	Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infus ...	Severe Combined Immunodeficiency	Drug: Anti-thymocyte globulin (rabbit);Drug: Busulfan;Drug: Fludarabine;Drug: Thiotepa;Device: Clini ...	St. Jude Children's Research Hospital	NULL	Recruiting	2 Months	N/A	All	42	Phase 1/Phase 2	United States
3	NCT04339777 (ClinicalTrials.gov)	September 22, 2020	8/4/2020	Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity	A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of ...	Lymphoproliferative Disorders;Autoimmune Lymphoproliferative;Immune System Diseases;Common Variable ...	Drug: Busulfan test dose;Drug: Fludarabine;Drug: Busulfan;Drug: Alemtuzumab;Radiation: Total body Irr ...	National Cancer Institute (NCI)	NULL	Recruiting	4 Years	69 Years	All	66	Phase 2	United States
4	EUCTR2018-003842-18-IT (EUCTR)	08/01/2019	19/11/2018	Gene therapy study using a frozen formulation of OTL-103 in patients with Wiskott-Aldrich Syndrome ( ...	A Single Arm, Open Label Clinical Study of Haematopoietic Stem Cell Gene Therapy with Cryopreserved ...	Wiskott-Aldrich Syndrome MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodef ...	Product Name: OTL-103 Dispersion for Infusion Product Code: OTL-103 INN or Proposed INN: Other hematolo ...	Orchard Therapeutics Ltd.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	6	Phase 3	Italy
5	EUCTR2018-001489-41-SE (EUCTR)	02/07/2018	08/05/2018	A clinical trial using cell transplantation as treatment for bone marrow failure due to dyskeratosis ...	Radiation- and alkylator-free hematopoietic cell transplantation for bone marrow failure due to dysk ...	Dyskeratosis congenita / telomere disease MedDRA version: 20.0;Level: PT;Classification code 1000175 ...	Trade Name: Lemtrada INN or Proposed INN: Alemtuzumab Other descriptive name: ALEMTUZUMAB Trade Name: F ...	Karolinska Universitetssjukhuset	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	40	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - ( ...	United States;Sweden
6	NCT03513328 (ClinicalTrials.gov)	June 15, 2018	19/4/2018	Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation	PEDS024, Phase I/II Feasibility Study of Busulfan Fludarabineand Thiotepa Conditioning Regimen for A ...	Bone Marrow Failure Syndrome;Thalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;Acquired Neutro ...	Drug: Thiotepa --single daily dose;Drug: Thiotepa --escalated dose	University of Florida	Live Like Bella Pediatric Cancer Research	Active, not recruiting	3 Months	39 Years	All	6	Phase 1/Phase 2	United States
7	JPRN-UMIN000030806	2018/01/17	15/01/2018	A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome	A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome - H ...	Wiskott-Aldrich syndrome	WASP cDNA-transduced autologous hematopoietic stem cells are administered to patients affected by WA ...	National Center for Child Heath and Development	NULL	Complete: follow-up continuing	Not applicable	Not applicable	Male	3	Phase 1,2	Japan
8	NCT03333486 (ClinicalTrials.gov)	December 7, 2017	2/11/2017	FludarabinePhosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Tr ...	A Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripher ...	Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Acute Leukemia in Remission;Acute ...	Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Procedure: Pe ...	Roswell Park Cancer Institute	NULL	Active, not recruiting	1 Year	75 Years	All	31	Phase 2	United States
9	JPRN-UMIN000019532	2015/12/01	01/12/2015	Clinical Phase II Study of hematopoietic stem ctell transplantation for ataxia telangiectasia and re ...	Clinical Phase II Study of hematopoietic stem ctell transplantation for ataxia telangiectasia and re ...	ataxia telangiectasiaDNA damage response disorder	fludarabine 30 mg/m2x5, cyclophosphamide 5mg/kgx4, ATG 1.25mg/kgx4	Graduate Schoool,Dept. of Community Pediatrics,Perinatal and Maternal Medicine,Tokyo Medical and Den ...	NULL	Recruiting	Not applicable	Not applicable	Male and Female	5	Phase 2	Japan
10	NCT02162420 (ClinicalTrials.gov)	January 10, 2015	10/6/2014	Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia	Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia	Dyskeratosis Congenita;Aplastic Anemia	Drug: Alemtuzumab;Drug: Fludarabine;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Biologi ...	Masonic Cancer Center, University of Minnesota	NULL	Recruiting	0 Years	70 Years	All	50	N/A	United States

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