Plain-English translation of NCT05213572 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
This study is examining whether a stem cell transplant or gene therapy helps repair organ damage caused by sickle cell disease. Researchers will compare people who receive these potentially curative treatments to those who continue with standard care, looking at how the heart, lungs, kidneys, liver, and brain function over 2 years.
Sickle cell disease damages multiple organs over time and can shorten life span. While stem cell transplants and gene therapy show promise as cures, doctors need to understand exactly how these treatments affect organ recovery and whether people truly benefit in the long term.
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You will visit the NIH Clinical Center for detailed testing of your heart, lungs, kidneys, liver, and brain function at the start of the study and again 2 years later. Testing includes heart and lung scans, blood work, kidney function assessments, brain imaging, and cognitive (thinking and memory) evaluations. If you are receiving a stem cell transplant or gene therapy, researchers will track how your organs recover; if you are on standard care, you will serve as a comparison group to help show the benefit of these newer treatments.
AI-generated summary from trial data · Jul 13, 2026 · Not medical advice
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