Recruiting

Testing a new medication for sickle cell disease

Plain-English translation of NCT06481306 on ClinicalTrials.gov ↗ · Source last updated August 2026 · Translation generated August 2026 · How we translate trials

Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.

This is one of the first times this treatment has been tested in people.

What is this trial?

This is a first-in-human trial testing a new medication called to see if it can help people with sickle cell disease. The study will first test the medication in healthy volunteers to make sure it's safe, then test it in people who have sickle cell disease to see if it reduces pain crises and improves their condition. Researchers will carefully monitor how your body handles the medication and watch for any side effects.

Sickle cell disease causes painful crises and serious health complications. This trial exists to test whether this new medication could be a better treatment option by reducing these crises and helping people manage their condition more effectively.

Do you qualify?

You likely qualify if…

you are a healthy adult with a normal body weight, or you have been diagnosed with sickle cell disease
if you have sickle cell disease, you have experienced recent pain crises (at least 2 to 4 in the past 6 to 12 months, depending on which part of the study you join)
you are in good enough health to participate in the study, as determined by blood tests and physical exams
if you have sickle cell disease, your hemoglobin levels fall within the range expected for your condition
you have not received a blood transfusion within the past 4 weeks

You likely don't qualify if…

you have any serious medical condition that would make it unsafe for you to participate
you have had more than 6 severe pain crises requiring hospitalization in the past year
you have had acute chest syndrome (a serious lung complication) within the last 6 months
you have kidney problems or severely reduced kidney function
you are currently receiving regular blood transfusions or had a transfusion very recently

What participation looks like

If you join this study, you will either take the experimental medication or a placebo (inactive pill) in increasing doses over time. You will visit the study site multiple times for blood tests, physical exams, and to report how you're feeling. The study team will monitor your blood counts, hemoglobin levels, and whether you experience pain crises. Depending on which group you're in, the study may last several weeks to months as researchers carefully evaluate whether the medication is safe and whether it helps reduce sickle cell symptoms.

AI-generated summary from trial data · Aug 30, 2026 · Not medical advice

Trial locations(32 sites)

United States

University of Alabama at Birmingham, Birmingham, Alabama

University of California San Diego - La Jolla, La Jolla, California

UCSF Benioff Children's Hospital Oakland, Oakland, California

Yale-New Haven Hospital, New Haven, Connecticut

Winship Cancer Institute of Emory University, Atlanta, Georgia