Plain-English translation of NCT05431088 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Sickle Cell Disease research guide →Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.
This trial is testing a medication called to see if it can help people with sickle cell disease. The study has three phases: first, researchers will test different doses to find the safest and most effective amount; then, they will compare the medication to a placebo (a dummy pill) in a larger group of teens and adults over 48 weeks; finally, people who complete the main study can continue taking the medication for up to 2 years to see how safe and effective it is over the long term.
Sickle cell disease causes painful crises when blood vessels become blocked, and current treatments don't work well enough for everyone. Researchers are testing this new medication to see if it can reduce how often these painful episodes happen and improve overall blood health.
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If you join, you'll take a daily oral medication (a pill) for several weeks to months, depending on which part of the study you're in. Some participants will receive the new medication at different doses, while others will receive a placebo to compare results. You'll have regular visits with the study team to check how you're doing, answer questions about any side effects, and have your blood checked. The main comparison phase lasts 48 weeks, and if you complete it, you may have the option to continue taking the medication for up to 2 years to monitor long-term safety.
AI-generated summary from trial data · Jun 6, 2026 · Not medical advice
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