Plain-English translation of NCT03264989 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Sickle cell disease research guide →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 studied , a medication given intravenously, to understand how it moves through the body and how it affects sickle cell disease. Researchers followed participants for up to five years to examine whether this treatment was safe and whether it reduced the frequency of vaso-occlusive crises—the painful episodes that people with sickle cell disease experience. The medication was tested at two different doses to see which worked best.
Sickle cell disease causes severe, unpredictable pain crises that can be life-altering for patients. This trial investigated whether this new medication could prevent or reduce these painful episodes by targeting a specific mechanism involved in the disease.
Participants received intravenous infusions of the medication at clinic visits over the course of the study. Blood samples were drawn regularly to measure how the body processed the medication and how it was affecting the disease, with additional samples taken when participants experienced pain crises, fever, or infection. The trial provided five years of follow-up data to thoroughly evaluate the safety and effectiveness of the treatment.
AI-generated summary from trial data · Oct 12, 2026 · Not medical advice
United States