DDrare
Disease
Drug
Target Gene/Pathway
問合せ先
EN
285. ファンコニ貧血
Fanconi anemia
Clinical trials
/
Disease details
臨床試験数
:
59
/
薬物数
:
118
- (
DrugBank
:
32
) /
標的遺伝子数
:
31
-
標的パスウェイ数
:
155
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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
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
Procedure:
Bone Marrow Aspiration
;Biological:
Filgrastim
;Biological:
Genetically Engineered Hematopo
...
Fred Hutchinson Cancer Research Center
National Heart, Lung, and Blood Institute (NHLBI);Rocket Pharma Limited
Active, not recruiting
4 Years
N/A
All
3
Phase 1
United States