Gene therapy aims to fix immune system in rare disease
NCT ID NCT03645486
First seen Apr 25, 2026 · Last updated Jun 23, 2026 · Updated 9 times
Summary
This trial tests a gene therapy for chronic granulomatous disease (CGD), a rare immune disorder that causes severe infections. The treatment uses a lentivirus to deliver a corrected gene into the patient's own stem cells, which are then infused back. Ten participants will be followed for up to 15 years to see if the therapy improves immune function and reduces infections.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for CHRONIC GRANULOMATOUS DISEASE are added.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Shenzhen Geno-immune Medical Institute
RECRUITINGShenzhen, Guangdong, 518000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
lentiviral gene therapy (TYF-CGD-modified stem cells)
What this could lead to
If successful, this could provide a long-term treatment option for chronic granulomatous disease by correcting the genetic defect and reducing severe infections.
What could go wrong
This is an early-phase trial with only 10 participants, so results may not apply to all patients. Risks include potential side effects from the gene therapy or failure to achieve lasting immune recovery.
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.