NCT04797260View on ClinicalTrials.gov

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

NARecruitingINTERVENTIONAL
Enrollment

10

Participants

Timeline

Start Date

July 23, 2021

Primary Completion Date

December 31, 2029

Study Completion Date

December 31, 2029

Conditions
Severe Combined Immunodeficiency Due to RAG1 Deficiency
Interventions
GENETIC

Gene therapy

Patients will be infused with autologous CD34+ cells transduced with the pCCL.MND.coRAG1.wpre lentiviral vector (RAG1 LV CD34+ cells).

Trial Locations (7)

3052

NOT_YET_RECRUITING

The Royal Childrens Hospital, Melbourne

Unknown

NOT_YET_RECRUITING

Ospedale Pediatrico Bambino Gesù, Roma

RECRUITING

Erciyes Üniversitesi TIP Fakültesi, Kayseri

NOT_YET_RECRUITING

University College London Great Ormond Street, London

2300RC

RECRUITING

Leiden University Medical Center, Leiden

50-556

RECRUITING

Wroclaw Medical University, Wroclaw

08035

RECRUITING

Hospital Universitari Vall d'Hebron, Barcelona

All Listed Sponsors
collaborator

ZonMw: The Netherlands Organisation for Health Research and Development

OTHER

collaborator

Horizon 2020 - European Commission

OTHER

lead

Leiden University Medical Center

OTHER