Gene therapy breakthrough for SCID-X1: a step toward a cure?
NCT ID NCT01410019
First seen Nov 01, 2025 · Last updated Jun 19, 2026 · Updated 31 times
Summary
This trial tested a gene therapy for X-linked severe combined immunodeficiency (SCID-X1), a rare genetic disorder that leaves boys without a working immune system. Five boys received a single infusion of their own blood stem cells, modified with a corrected gene. The goal was to see if this could rebuild their immune defenses safely and effectively.
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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.
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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Locations
-
Hopital Necker
Paris, 75015, France
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 with a self-inactivating gammaretroviral vector
What this could lead to
If successful, this could provide a one-time gene therapy to restore immune function in boys with SCID-X1, reducing the need for lifelong treatments.
What could go wrong
This is an early-phase trial with only 5 participants, so results may not apply to all patients. Risks include potential side effects from gene therapy, such as immune reactions or long-term safety issues.
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.