DDrare: Database of Drug Development for Rare Diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
285	ファンコニ貧血	1

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	Fred Hutchinson Cancer Research Center	National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI);Rocket Pharma Limited	Active, not recruiting	4 Years	N/A	All	3	Phase 1	United States

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

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 Research Center

National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI);Rocket Pharma Limited

Active, not recruiting

4 Years

N/A

All

Phase 1

United States