Gene therapy offers hope for 'Bubble Boy' disease, but leukemia risk remains
NCT ID NCT01129544
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This trial tested a gene therapy for children with SCID-X1, a severe immune disorder, who do not have a matched bone marrow donor. The treatment involves harvesting the child's own bone marrow cells, fixing the broken gene in the lab, and infusing the corrected cells back after mild chemotherapy. Of the 8 children enrolled, some saw immune improvement, but the therapy carries a risk of leukemia, as seen in earlier studies.
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 (retrovirus-mediated gene transfer)
What this could lead to
If successful, this could offer a treatment option for SCID-X1 patients who lack a matched bone marrow donor, potentially restoring immune function without the need for lifelong medication.
What could go wrong
This is an early-phase trial with only 8 participants. Previous similar trials caused leukemia in 5 out of 20 children, and one child died. The procedure may not work for everyone, and long-term risks are still unknown.
Disclaimer
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the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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Locations
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Children's Hospital Boston
Boston, Massachusetts, 02116, United States
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Cincinnati Children's Medical Center
Cincinnati, Ohio, 45229, United States
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Mattel Children's Hospital - UCLA
Los Angeles, California, 90095, United States