Experimental gene therapy targets rare immune disorder
NCT ID NCT01855685
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This trial tested a gene therapy for X-linked chronic granulomatous disease (X-CGD), a rare genetic disorder that leaves boys unable to fight off infections. The treatment involved taking the patient's own blood stem cells, adding a working copy of the faulty gene, and returning them to the body. The goal was to restore immune function and reduce severe infections and inflammation. Only 3 patients were enrolled before the study was terminated.
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 (lentiviral vector with GP91PHOX gene)
- What this could lead to
- If successful, this gene therapy could provide a long-term treatment option for X-CGD patients who lack a donor for bone marrow transplant, reducing severe infections and inflammation.
- What could go wrong
- This was a very early, small trial (only 3 participants) that was terminated, so results are limited. Gene therapy carries risks like immune reactions or the treatment not lasting.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
-
3 people
The number who actually took part.
- Started
-
Jun 2013
- Finished
-
Oct 2025
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
6 months and older
- Sex
-
Male participants only
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Male X-CGD patients * Molecular diagnosis confirmed by DNA sequencing * At least one prior ongoing or resistant severe infection and/or inflammatory complications requiring hospitalisation despite conventional therapy * No HLA-matched donor available after 3 months search unless the risk of waiting for a potential match or for performing an allogeneic transplant is considered unacceptable by the investigator Exclusion Criteria: * Contraindication for leukapheresis * Contraindication for administration of conditioning medication * Administration of gammainterferon within 30 days before the infusion of transduced autologous CD34+ cells
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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
-
Great Ormond Street Hospital NHS Foundation Trust
London, United Kingdom
-
University College London Hospital (UCLH)
London, NW1 2PG, United Kingdom
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Gene therapy hope for rare immune disease fails to launch
- Gene fix trial aims to tame rare immune disease
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- Gene therapy aims to fix immune system in rare disease