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
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
Who can I contact for more information?
You can email or call the study team at 206-884-1068.