65. Primary immunodeficiency Clinical trials / Disease details

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

7 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	NCT04561115 (ClinicalTrials.gov)	October 6, 2020	10/9/2020	A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Immune Globulin (Human) 10% (Gamunex-C) PEG Process (IVIG-PEG) Compared to Gamunex-C in Participants With Primary Humoral Immunodeficiency	A Phase 3, Multicenter, Open-label, Single-sequence, Cross-over, Bioequivalence Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of IVIG-PEG Compared to Gamunex-C in Subjects With Primary Humoral Immunodeficiency	Primary Immunodeficiency	Biological: Gamunex-C;Biological: IVIG-PEG	Grifols Therapeutics LLC	NULL	Active, not recruiting	18 Years	75 Years	All	36	Phase 3	United States
2	NCT03765632 (ClinicalTrials.gov)	January 3, 2018	8/8/2018	Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID	Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1a Short Form (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency (SCID) Due to Adenosine Deaminase Deficiency	Severe Combined Immunodeficiency Due to ADA Deficiency	Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: Busulfan;Drug: Peg-Ada	Great Ormond Street Hospital for Children NHS Foundation Trust	Orchard Therapeutics	Active, not recruiting	N/A	17 Years	All	13	Phase 1/Phase 2	United Kingdom
3	NCT02999984 (ClinicalTrials.gov)	December 16, 2016	19/12/2016	Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID	Efficacy and Safety of Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With EFS Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to ADA Deficiency	Severe Combined Immunodeficiency Due to ADA Deficiency	Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: busulfan;Drug: PEG -ADA ERT	Orchard Therapeutics	California Institute for Regenerative Medicine (CIRM);University of California, Los Angeles	Completed	N/A	17 Years	All	10	Phase 1/Phase 2	United States
4	NCT01852071 (ClinicalTrials.gov)	August 2, 2013	7/5/2013	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With EFS Lentiviral Vector Encoding for the Human ADA Gene	Autologous Transplantation of Bone Marrow CD34+ Stem/Progenitor Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID)	ADA-SCID	Genetic: Infusion of autologous EFS-ADA LV CD34+ (OTL-101);Drug: busulfan;Drug: PEG -ADA ERT	Orchard Therapeutics	National Institute of Allergy and Infectious Diseases (NIAID);National Human Genome Research Institute (NHGRI);National Heart, Lung, and Blood Institute (NHLBI);University of California, Los Angeles	Completed	1 Month	17 Years	All	20	Phase 1/Phase 2	United States
5	NCT01380990 (ClinicalTrials.gov)	November 15, 2012	23/6/2011	Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency	Phase I/II, Historical Controlled, Open-label, Non-randomised, Single-centre Trial to Assess the Safety and Efficacy of EF1aS-ADA Lentiviral Vector Mediated Gene Modification of Autologous CD34+ Cells From ADA-deficient Individuals	Adenosine Deaminase Deficiency;Severe Combined Immunodeficiencies (SCID)	Genetic: Infusion of autologous EFS-ADA LV CD34+ cells;Other: Haematopoietic Stem Cell Transplantation (HSCT);Drug: Busulfan;Drug: Peg-Ada	Great Ormond Street Hospital for Children NHS Foundation Trust	Orchard Therapeutics	Completed	N/A	15 Years	All	36	Phase 1/Phase 2	United Kingdom
6	NCT00794508 (ClinicalTrials.gov)	November 2008	19/11/2008	MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID	MND-ADA Transduction of CD34+ Cells From the Bone Marrow Of Children With Adenosine Deaminase (ADA)-Deficient Severe Combined Immunodeficiency (SCID): Effect of Discontinuation of PEG-ADA and Marrow Cytoreduction With Busulfan	Severe Combined Immunodeficiency	Biological: ADA gene transfer	Donald B. Kohn, M.D.	FDA Office of Orphan Products Development;National Institutes of Health (NIH)	Completed	1 Month	18 Years	All	10	Phase 2	United States
7	NCT00004695 (ClinicalTrials.gov)	September 1997	24/2/2000	Randomized Study of Polyethylene-Glycol-Conjugated Interleukin 2 in Patients With Common Variable Immunodeficiency		Common Variable Immunodeficiency	Drug: PEG -interleukin-2	Mount Sinai School of Medicine	NULL	Completed	2 Years	N/A	Both	48	N/A	NULL