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

  
5 trials found
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PhaseCountries
1NCT04561115
(ClinicalTrials.gov)
October 6, 202010/9/2020A 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 ImmunodeficiencyA 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 ImmunodeficiencyPrimary ImmunodeficiencyBiological: Gamunex-C;Biological: IVIG-PEGGrifols Therapeutics LLCNULLCompleted18 Years75 YearsAll36Phase 3United States
2NCT03765632
(ClinicalTrials.gov)
January 3, 20188/8/2018Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCIDEfficacy 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 DeficiencySevere Combined Immunodeficiency Due to ADA DeficiencyGenetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: Busulfan;Drug: Peg-AdaGreat Ormond Street Hospital for Children NHS Foundation TrustOrchard TherapeuticsCompleted30 Days17 YearsAll13Phase 1/Phase 2United Kingdom
3NCT01380990
(ClinicalTrials.gov)
November 15, 201223/6/2011Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) DeficiencyPhase 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 IndividualsAdenosine 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-AdaGreat Ormond Street Hospital for Children NHS Foundation TrustOrchard TherapeuticsCompletedN/A15 YearsAll36Phase 1/Phase 2United Kingdom
4NCT00794508
(ClinicalTrials.gov)
November 200819/11/2008MND-ADA Transduction of CD34+ Cells From Children With ADA-SCIDMND-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 BusulfanSevere Combined ImmunodeficiencyBiological: ADA gene transferDonald B. Kohn, M.D.FDA Office of Orphan Products Development;National Institutes of Health (NIH)Completed1 Month18 YearsAll10Phase 2United States
5NCT00004695
(ClinicalTrials.gov)
September 199724/2/2000Randomized Study of Polyethylene-Glycol-Conjugated Interleukin 2 in Patients With Common Variable ImmunodeficiencyCommon Variable ImmunodeficiencyDrug: PEG-interleukin-2Mount Sinai School of MedicineNULLCompleted2 YearsN/ABoth48N/ANULL