Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants
Status:
Recruiting
Trial end date:
2034-08-01
Target enrollment:
Participant gender:
Summary
SCID-X1 is a genetic disorder of blood cells caused by DNA changes in a gene that is required
for the normal development of the human immune system. The purpose of this study is to
determine if a new method, called lentiviral gene transfer, can be used to treat SCID-X1.
This method involves transferring a normal copy of the common gamma chain gene into the
participant's bone marrow stem cells. The investigators want to determine if the procedure is
safe, whether it can be done according to the methods they have developed, and whether the
procedure will provide a normal immune system for the patient. It is hoped that this type of
gene transfer may offer a new way to treat children with SCID-X1 that do not have a brother
or sister who can be used as a donor for stem cell transplantation.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
St. Jude Children's Research Hospital
Collaborators:
Assisi Foundation California Institute for Regenerative Medicine (CIRM) National Heart, Lung, and Blood Institute (NHLBI)