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 (Click here for study contact information)
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