Gene therapy offers hope for children with SCID-X1
NCT ID NCT03311503
First seen Jan 04, 2026 · Last updated Jun 23, 2026 · Updated 34 times
Summary
This trial tests a gene therapy for children with X-linked severe combined immunodeficiency (SCID-X1), also known as 'bubble boy' disease. Participants receive a low dose of busulfan followed by an infusion of their own blood stem cells that have been genetically corrected with a lentiviral vector. The goal is to restore a working immune system and reduce the need for lifelong treatments. The study is recruiting up to 12 children aged 5 or younger.
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This is a summary of
the original study
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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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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Boston Childrens Hospital
RECRUITINGBoston, Massachusetts, 02115, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Cincinnati Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Emory University/Childrens Healthcare of Atlanta
RECRUITINGAtlanta, Georgia, 30322, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Mattel Children's Hospital - UCLA
RECRUITINGLos Angeles, California, 90095, United States
Contact Phone: •••-•••-••••
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
autologous CD34+ cells transduced with G2SCID lentiviral vector
What this could lead to
If successful, this could provide a one-time gene therapy that restores immune function in children with SCID-X1, reducing the need for lifelong treatments.
What could go wrong
This is an early-phase trial with only 12 participants, so results may not apply to all patients. Risks include failure to restore immunity, side effects from busulfan, or need for a stem cell transplant.
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.