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 : 31 - 標的パスウェイ数 : 155

  
2 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT03157804
(ClinicalTrials.gov)		January 7, 201626/4/2017Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype AClinical Trial Phase I / II to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34 + Cells Transduced With a Lentiviral Vector Carrying the Gene FANCA in Patients With FA Subtype A (FANCOLEN-1)Fanconi AnemiaProcedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs);Biological: Genetically Engineered Hematopoietic Stem/Progenitor Cells;Other: Laboratory Biomarker Analysis;Biological: Filgrastim;Drug: Plerixafor;Procedure: Bone Marrow AspirationHospital Infantil Universitario Niño Jesús, Madrid, SpainCentro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT);Centro de Investigación en Red de Enfermedades Raras (CIBERER);Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz;Hospital Vall d'Hebron;Universitat Autonoma de BarcelonaActive, not recruiting1 Year21 YearsAll9Phase 1/Phase 2Spain
2NCT01331018
(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