Current Research Studies

Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants (LVXSCID-ND)

Condition or Therapy:

X-linked severe combined immunodeficiency (SCID-X1); gene therapy

Category:

What is the goal of this study?

Researchers at Seattle Children’s are testing whether gene therapy can be used to treat male infants with X-linked Severe Combined Immunodeficiency (X-linked SCID).

Who can join the study?

This study might be a good fit if your child:

Has X-linked SCID
Is over the age of 2 months
Does not have a matched sibling donor

What will happen if my child takes part in this study?

If you decide to have your child take part in this study and your child is a good candidate for this trial, your child would:

Undergo a bone marrow cell harvest
Receive a reduced amount of chemotherapy to create space for gene modified cells to grow
Receive his own gene modified cells
Have regular evaluations to monitor response to therapy