Gene therapy for rare blood disease passes 15-Year safety watch
NCT ID NCT04437771
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study follows 9 people with Fanconi Anemia who already received a gene therapy that adds a working FANCA gene to their blood stem cells. Researchers will check their health and blood counts for 15 years to see if the treatment remains safe and keeps working. No new treatment is given during this follow-up.
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 (autologous CD34+ cells with FANCA gene)
What this could lead to
If successful, this could show that a one-time gene therapy safely stabilizes blood counts and reduces cancer risk for people with Fanconi Anemia.
What could go wrong
This is a very small, early-phase follow-up study with only 9 patients. It cannot prove the therapy works for everyone, and long-term risks like cancer or side effects from the gene insertion are still unknown.
Disclaimer
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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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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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Hospital Infantil Universitario Niño Jesús (HIUNJ)
Madrid, 28009, Spain