Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID

NCTID	NCT04797260 (View at clinicaltrials.gov)
Description	This study is a prospective, non-randomized, open-label, two-centre phase I/II intervention study designed to treat children up to 24 months of age with RAG1-deficient SCID with an indication for allogeneic hematopoietic stem cell transplantation but lacking an HLA-matched donor. The study involves infusion of autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector (hereafter called RAG1 LV CD34+ cells) in five patients with RAG1-deficient SCID. (Show More)
Development Status	Active
Indication	Severe Combined Immunodeficiency Due to RAG1 Deficiency
Disease Ontology Term	DOID:0060011
Compound Name	Autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector
Sponsor	Leiden University Medical Center
Funder Type	Other
Recruitment Status	Recruiting
Enrollment Count	10
Results Posted	Not Available

Target Gene/Variant	RAG1
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	LV
Editor Type	none
Dose 1	Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	7
Locations	Netherlands,Turkey,Poland,Italy,United Kingdom,Australia,Spain