285. ファンコニ貧血 Fanconi anemia Clinical trials / Disease details


臨床試験数 : 62 薬物数 : 93 - (DrugBank : 30) / 標的遺伝子数 : 30 - 標的パスウェイ数 : 144

  
10 trials found
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PhaseCountries
1NCT04248439
(ClinicalTrials.gov)
July 15, 202024/1/2020Gene Therapy for Fanconi Anemia, Complementation Group AA 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 AFanconi Anemia Complementation Group ABiological: RP-L102Rocket Pharmaceuticals Inc.NULLRecruiting1 YearN/AAll5Phase 2United States
2EUCTR2018-002502-31-GB
(EUCTR)
07/02/202008/10/2019A 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 AA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
5Phase 2Spain;United Kingdom
3NCT04069533
(ClinicalTrials.gov)
November 28, 201923/8/2019Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype AA 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 AFanconi Anemia Complementation Group ABiological: RP-L102Rocket Pharmaceuticals Inc.NULLRecruiting1 Year17 YearsAll5Phase 2Spain
4NCT03814408
(ClinicalTrials.gov)
January 11, 201914/1/2019A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype AA 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 AFanconi Anemia Complementation Group ABiological: RP-L102Rocket Pharmaceuticals Inc.NULLActive, not recruiting1 Year12 YearsAll2Phase 1United States
5EUCTR2018-002502-31-ES
(EUCTR)
31/10/201802/08/2018A 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 AA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
5Phase 2Spain
6NCT02678533
(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 ParisEuroFancolenCompleted2 Years17 YearsAll4Phase 1/Phase 2France
7EUCTR2014-004272-29-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]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 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
8NCT02931071
(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
9EUCTR2011-006100-12-ES
(EUCTR)
12/04/201327/01/2012Clinical 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 JESUSNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
5Phase 1Spain
10NCT00001399
(ClinicalTrials.gov)
December 3, 19933/11/1999Gene Therapy for the Treatment of Fanconi's Anemia Type CRetroviral Mediated Gene Transfer of the Fanconi Anemia Complementation Group C Gene to Hematopoietic Progenitors of Group C PatientsFanconi's Anemia;PancytopeniaDrug: Transduced CD34+ CellsNational Heart, Lung, and Blood Institute (NHLBI)NULLCompleted5 YearsN/AAll9Phase 1United States