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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
5 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
1NCT02678533
(ClinicalTrials.gov)
February 10, 20175/2/2016Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and PlerixaforPilot Study Assessing the Feasibility of CD34+ Cells Mobilization and Collection After Treatment With G-CSF and Plerixafor in Patients With Fanconi Anemia for Subsequent Treatment by Gene TherapyFanconi AnemiaDrug: G-CSF;Drug: PlerixaforAssistance Publique - Hôpitaux de ParisEuroFancolenRecruiting2 Years17 YearsAll8Phase 1/Phase 2France
2NCT03157804
(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
3EUCTR2014-004272-29-GB
(EUCTR)
30/03/201515/07/2015Phase II clinical trial to evaluate safety and efficacy of mobilisation and collection of CD34+ cells after treatment with plerixafor and filgrastim in patients with Fanconi anaemia for subsequent transduction with a lentiviral vector carrying the FANCA gene and reinfusion into the patient - Fancostem-Plerixafor & Filgrastim mobilisation in Fanconi AnaemiaPhase II clinical trial to evaluate safety and efficacy of mobilisation and collection of CD34+ cells after treatment with plerixafor and filgrastim in patients with Fanconi anaemia for subsequent transduction with a lentiviral vector carrying the FANCA gene and reinfusion into the patient - Fancostem-Plerixafor & Filgrastim mobilisation in Fanconi Anaemia Fanconi Anaemia;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Great Ormond Street Hospital NHS foundation TrustNULLNot Recruiting Female: yes
Male: yes
20 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;Spain;United Kingdom
4NCT02931071
(ClinicalTrials.gov)
September 20136/9/2016Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1Clinical Phase II Trial to Evaluate Efficacy and Safety of CD34+ Cells Mobilization and Collection After Treatment With Plerixafor and Filgrastim in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene and Reinfusion in the PatientFanconi AnemiaDrug: filgrastim;Drug: plerixaforHospital Universitari Vall d'Hebron Research InstituteCIEMAT;CIBERERCompleted2 Years64 YearsAll13Phase 2Spain
5NCT01331018
(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 Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI);Rocket Pharma LimitedActive, not recruiting4 YearsN/AAll3Phase 1United States