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
65	原発性免疫不全症候群	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	NCT03538899 (ClinicalTrials.gov)	May 31, 2018	3/5/2018	Autologous Gene Therapy for Artemis-Deficient SCID	A Phase I/II Feasibility Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) Using a Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells	Severe Combined Immunodeficiency	Drug: AProArt;Device: CliniMACS® CD34 Reagent System cell sorter device;Drug: Busulfan	University of California, San Francisco	NULL	Recruiting	2 Months	N/A	All	15	Phase 1;Phase 2	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

NCT03538899
(ClinicalTrials.gov)

May 31, 2018

3/5/2018

Autologous Gene Therapy for Artemis-Deficient SCID

A Phase I/II Feasibility Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) Using a Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells

Severe Combined Immunodeficiency

Drug: AProArt;Device: CliniMACS® CD34 Reagent System cell sorter device;Drug: Busulfan

University of California, San Francisco

NULL

Recruiting

2 Months

N/A

All

Phase 1;Phase 2

United States