285. ファンコニ貧血 Fanconi anemia Clinical trials / Disease details


臨床試験数 : 59 薬物数 : 118 - (DrugBank : 32) / 標的遺伝子数 : 30 - 標的パスウェイ数 : 154

  
1 trial found
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agemin
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PhaseCountries
1NCT01331018
(ClinicalTrials.gov)
February 22, 201216/3/2011Gene Therapy for Fanconi AnemiaGene Transfer for Patients With Fanconi Anemia Complementation Group A (FANCA)Fanconi AnemiaProcedure: Bone Marrow Aspiration;Biological: Filgrastim;Biological: Genetically Engineered Hematopoietic Stem Progenitor Cells;Other: Laboratory Biomarker Analysis;Procedure: Leukapheresis;Drug: Methylprednisolone;Drug: Plerixafor;Drug: PrednisoneFred Hutchinson Cancer Research CenterNational Heart, Lung, and Blood Institute (NHLBI);Rocket Pharma LimitedActive, not recruiting4 YearsN/AAll3Phase 1United States