285. ファンコニ貧血
[臨床試験数:56,薬物数:111(DrugBank:31),標的遺伝子数:30,標的パスウェイ数:151

Searched query = "Fanconi anemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

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1 trial found
No.TrialIDDate_
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agemin
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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