Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency

PHASE1RecruitingINTERVENTIONAL
Enrollment

5

Participants

Timeline

Start Date

December 21, 2018

Primary Completion Date

August 31, 2026

Study Completion Date

August 31, 2026

Conditions
Severe Combined Immunodeficiency, X-Linked
Interventions
DRUG

Lentiviral vector transduced CD34+ cells

Gene therapy for X-linked Severe Combined Immunodeficiency will be performed by introduction a normal copy of the IL2RG gene into the blood forming stem cells (CD34+ cells) of the patient's bone marrow by using a type of gene delivery system (in this trial called a lentiviral vector). The gene corrected cells are then transplanted back into the patient.

Trial Locations (1)

WC1N 3JH

RECRUITING

Great Ormond Street Hospital for Children NHS Foundation Trust, London

All Listed Sponsors
lead

Great Ormond Street Hospital for Children NHS Foundation Trust

OTHER

NCT03601286 - Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency | Biotech Hunter | Biotech Hunter