DDrare: Database of Drug Development for Rare Diseases

Procedure: iv administration of genetically engineered hematopoietic stem/progenitors cells (hspcs) (DrugBank: -)

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	NCT03157804 (ClinicalTrials.gov)	January 7, 2016	26/4/2017	Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients 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 Gene FANCA in Patients With FA Subtype A (FANCOLEN-1)	Fanconi Anemia	Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs);Biological: Genetically Engineered Hematopoietic Stem/Progenitor Cells;Other: Laboratory Biomarker Analysis;Biological: Filgrastim;Drug: Plerixafor;Procedure: Bone Marrow Aspiration	Hospital Infantil Universitario Niño Jesús, Madrid, Spain	Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT);Centro de Investigación en Red de Enfermedades Raras (CIBERER);Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz;Hospital Vall d'Hebron;Universitat Autonoma de Barcelona	Active, not recruiting	1 Year	21 Years	All	9	Phase 1;Phase 2	Spain

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

NCT03157804
(ClinicalTrials.gov)

January 7, 2016

26/4/2017

Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients 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 Gene FANCA in Patients With FA Subtype A (FANCOLEN-1)

Fanconi Anemia

Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs);Biological: Genetically Engineered Hematopoietic Stem/Progenitor Cells;Other: Laboratory Biomarker Analysis;Biological: Filgrastim;Drug: Plerixafor;Procedure: Bone Marrow Aspiration

Hospital Infantil Universitario Niño Jesús, Madrid, Spain

Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT);Centro de Investigación en Red de Enfermedades Raras (CIBERER);Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz;Hospital Vall d'Hebron;Universitat Autonoma de Barcelona

Active, not recruiting

1 Year

21 Years

All

Phase 1;Phase 2

Spain