Bubble boy disease gene therapy trial launches
NCT ID NCT03217617
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This trial tests a gene therapy given by IV for X-linked severe combined immunodeficiency (SCID-X1), a rare immune disorder. Ten participants with no matched donor will receive a lentiviral vector to correct the genetic defect. The goal is to restore immune function and improve survival.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- lentiviral vector (ivlv-X1)
- What this could lead to
- If successful, this could provide a one-time gene therapy treatment for SCID-X1, potentially restoring immune function without the need for a bone marrow donor.
- What could go wrong
- This is an early-phase trial with only 10 participants, so results may not apply to everyone. Risks include immune reactions or the gene therapy not working as expected.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Shenzhen Geno-immune Medical Institute
RECRUITINGShenzhen, Guangdong, 518000, China