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 | NCT04248439 (ClinicalTrials.gov) | July 15, 2020 | 24/1/2020 | Gene Therapy for Fanconi Anemia, Complementation Group A | A Phase 2 Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A | Fanconi Anemia Complementation Group A | Biological: RP-L102 | Rocket Pharmaceuticals Inc. | NULL | Recruiting | 1 Year | N/A | All | 5 | Phase 2 | United States |
| 2 | EUCTR2018-002502-31-GB (EUCTR) | 07/02/2020 | 08/10/2019 | A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A | A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A - FANCOLEN-II | Fanconi anemia (subtype A) MedDRA version: 20.0;Level: LLT;Classification code 10055206;Term: Fanconi's anemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: RP-L102 INN or Proposed INN: CD34+CELLS Other descriptive name: CD34+CELLS | Rocket Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 2 | Spain;United Kingdom | ||
| 3 | NCT04069533 (ClinicalTrials.gov) | November 28, 2019 | 23/8/2019 | Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A | A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients With Fanconi Anemia Subtype A | Fanconi Anemia Complementation Group A | Biological: RP-L102 | Rocket Pharmaceuticals Inc. | NULL | Recruiting | 1 Year | 17 Years | All | 5 | Phase 2 | Spain |
| 4 | NCT03814408 (ClinicalTrials.gov) | January 11, 2019 | 14/1/2019 | A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A | A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A | Fanconi Anemia Complementation Group A | Biological: RP-L102 | Rocket Pharmaceuticals Inc. | NULL | Active, not recruiting | 1 Year | 12 Years | All | 2 | Phase 1 | United States |
| 5 | EUCTR2018-002502-31-ES (EUCTR) | 31/10/2018 | 02/08/2018 | A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A | A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A - FANCOLEN-II | Fanconi anemia (subtype A) MedDRA version: 20.0;Level: LLT;Classification code 10055206;Term: Fanconi's anemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: RP-L102 INN or Proposed INN: CD34+CELLS Other descriptive name: CD34+CELLS | Rocket Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 2 | Spain | ||
| 6 | 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 |
| 7 | 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 | ||
| 8 | 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 |
| 9 | EUCTR2011-006100-12-ES (EUCTR) | 12/04/2013 | 27/01/2012 | Clinical Trial Phase I / II to evaluate the safety and efficacy of the infusion of cells transduced with a therapeutic lentiviral vector for patients with Fanconi Anemia 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 FANCA gene (orphan drug) for patients with Fanconi Anemia Subtype A. - Fancolen-1 | Fanconi anemia (Subtype A) MedDRA version: 15.1;Level: LLT;Classification code 10055206;Term: Fanconi's anemia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: CD34+ Cells INN or Proposed INN: CD34+ CELLS Other descriptive name: CD34+ CELLS | FUNDACION PARA LA INVESTIGACION BIOMEDICA DEL HOSPITAL UNIVERSITARIO NIÑO JESUS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 1 | Spain | ||
| 10 | NCT00001399 (ClinicalTrials.gov) | December 3, 1993 | 3/11/1999 | Gene Therapy for the Treatment of Fanconi's Anemia Type C | Retroviral Mediated Gene Transfer of the Fanconi Anemia Complementation Group C Gene to Hematopoietic Progenitors of Group C Patients | Fanconi's Anemia;Pancytopenia | Drug: Transduced CD34+ Cells | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Completed | 5 Years | N/A | All | 9 | Phase 1 | United States |