285. Fanconi anemia Clinical trials / Disease details
Clinical trials : 62 / Drugs : 93 - (DrugBank : 30) / Drug target genes : 30 - Drug target pathways : 144
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02678533 (ClinicalTrials.gov) | February 10, 2017 | 5/2/2016 | Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor | Pilot 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 Therapy | Fanconi Anemia | Drug: G-CSF;Drug: Plerixafor | Assistance Publique - Hôpitaux de Paris | EuroFancolen | Completed | 2 Years | 17 Years | All | 4 | Phase 1/Phase 2 | France |
| 2 | NCT03157804 (ClinicalTrials.gov) | January 7, 2016 | 26/4/2017 | Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A | Clinical 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 Anemia | Procedure: 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 Aspiration | Hospital Infantil Universitario Niño Jesús, Madrid, Spain | Centro 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 Barcelona | Active, not recruiting | 1 Year | 21 Years | All | 9 | Phase 1/Phase 2 | Spain |
| 3 | EUCTR2014-004272-29-GB (EUCTR) | 30/03/2015 | 15/07/2015 | Phase 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 | Phase 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] | Trade Name: Mozobil Product Name: Mozobil INN or Proposed INN: Mozobil Other descriptive name: Plerixafor Trade Name: Neupogen Product Name: Neupogen INN or Proposed INN: Neupogen Other descriptive name: Filgrastim | Great Ormond Street Hospital NHS foundation Trust | NULL | Not 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): no | France;Spain;United Kingdom | ||
| 4 | NCT02931071 (ClinicalTrials.gov) | September 2013 | 6/9/2016 | Clinical 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-1 | Clinical 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 Patient | Fanconi Anemia | Drug: filgrastim;Drug: plerixafor | Hospital Universitari Vall d'Hebron Research Institute | CIEMAT;CIBERER | Completed | 2 Years | 64 Years | All | 13 | Phase 2 | Spain |
| 5 | NCT01331018 (ClinicalTrials.gov) | February 22, 2012 | 16/3/2011 | Gene Therapy for Fanconi Anemia | Gene Transfer for Patients With Fanconi Anemia Complementation Group A (FANCA) | Fanconi Anemia | Procedure: Bone Marrow Aspiration;Biological: Filgrastim;Biological: Genetically Engineered Hematopoietic Stem Progenitor Cells;Other: Laboratory Biomarker Analysis;Procedure: Leukapheresis;Drug: Methylprednisolone;Drug: Plerixafor;Drug: Prednisone | Fred Hutchinson Cancer Center | National Heart, Lung, and Blood Institute (NHLBI);Rocket Pharma Limited | Active, not recruiting | 4 Years | N/A | All | 3 | Phase 1 | United States |