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

  
5 trials found
No.TrialIDDate_
enrollment
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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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