Gene therapy could free kids from lifelong blood transfusions
NCT ID NCT07476183
First seen Jun 27, 2026 · Last updated Jul 22, 2026 · Updated 2 times
Summary
This early-stage trial tests a one-time gene therapy called APR-2020 in 4 children with a severe form of Diamond-Blackfan anemia. The treatment uses the child's own blood stem cells, modified to correct the genetic defect, and given back as an infusion. The goal is to see if it is safe and can reduce or end the need for regular blood transfusions.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- APR-2020 (gene-modified stem cells)
- What this could lead to
- If it works, this could reduce or eliminate the need for blood transfusions in children with Diamond-Blackfan anemia by correcting the genetic defect in their own stem cells.
- What could go wrong
- This is a very early phase 1 trial with only 4 participants, so safety and effectiveness are not yet proven. There are risks from the stem cell harvest and conditioning regimen, and the treatment may not work or could cause side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
-
Boston Children's Hospital
RECRUITINGBoston, Massachusetts, 02215, United States
-
Stanford University - Stanford Children's Health
RECRUITINGStanford, California, 94304, United States