Gene therapy offers hope for 'Bubble Boy' disease without a donor

NCT ID NCT04797260

Terminated Disease control Sponsor: Videja B.V. Source: ClinicalTrials.gov ↗

First seen Jul 13, 2026 · Last updated Jul 17, 2026 · Updated 3 times

Summary

This trial tests a gene therapy for infants under 2 years old with RAG1-deficient severe combined immunodeficiency (SCID), a life-threatening condition where the immune system barely works. The therapy uses the child's own blood stem cells, modified with a corrected gene, to try to rebuild a functioning immune system. It is designed for those who need a stem cell transplant but lack a matched donor. The study will monitor participants for at least 5 years to check safety and whether the treatment restores T and B cell immunity.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: gene therapy (RAG1 LV CD34+ cells)
What this could lead to: If successful, this could provide a life-saving treatment option for infants with RAG1 SCID who have no matched donor, potentially restoring their immune system without lifelong medication.
What could go wrong: This is an early-phase trial with only 10 participants, so results may not apply to all. Risks include adverse events, insertional mutagenesis, and the possibility that the therapy may not fully restore immunity.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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