Plain-English translation of NCT04201210 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.
This trial is testing a new approach to stem cell transplantation for people with sickle cell disease who don't have a perfectly matched sibling donor. Instead of waiting for a fully matched donor, researchers are using stem cells from a half-matched family member (usually a parent) after specially treating those cells to reduce rejection risk. The goal is to see if this treatment can cure sickle cell disease with fewer serious side effects compared to traditional transplants.
Many people with severe sickle cell disease could benefit from a stem cell transplant, but only about 25% have a perfectly matched sibling donor. This trial exists to expand transplant options for the majority of patients who lack a matched family member, potentially offering them a cure they might not otherwise have access to.
You likely qualify if…
You likely don't qualify if…
If you qualify, you would be assigned to either the experimental group (using a half-matched family donor) or the control group (if you happen to have a matched sibling). You would undergo preparation with chemotherapy and other conditioning treatments, then receive the transplanted stem cells. After transplant, you would have frequent medical visits and monitoring for months to a year or longer to watch for side effects, check that your new immune system is developing, and confirm that the sickle cell disease is cured. The trial will track your health, fertility, and quality of life over time.
AI-generated summary from trial data · Jun 3, 2026 · Not medical advice
Austria
Germany