Recruiting

Testing a new medication to reduce pain crises in sickle cell disease

Plain-English translation of NCT06975865 on ClinicalTrials.gov ↗ · Source last updated October 2026 · Translation generated August 2026 · 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.

What is this trial?

This study tests whether a medication called can help reduce the painful crises that people with sickle cell disease experience. About 192 people will take part in this 52-week trial, where some will receive the medication and others will receive a placebo (a dummy pill with no active ingredient). The study is designed to see if this treatment is safe and effective at preventing the pain episodes that are a hallmark of sickle cell disease.

Sickle cell disease causes recurring episodes of severe pain when blood cells block blood vessels. Doctors want to find new ways to prevent or reduce these painful crises and improve patients' quality of life.

Do you qualify?

You likely qualify if…

you have been diagnosed with sickle cell disease
you have had between 2 and 10 documented pain crisis episodes in the past 12 months
you are between 10 and 65 years old
you are either not taking hydroxyurea or L-glutamine, or you have been on a stable dose of these medications for at least 6 months
you are in generally good health (ECOG performance status of 2 or lower)

You likely don't qualify if…

you have a history of cancer (except some skin cancers) in the past 5 years
you have a history of stroke or abnormal brain blood vessel screening
you have an active or untreated infection (hepatitis B, hepatitis C, HIV, or tuberculosis)
you recently took certain other sickle cell medications like crizanlizumab (within 90 days) or voxelotor (within 30 days)
you have significant heart problems or an abnormal heart rhythm

What participation looks like

You will visit the study clinic regularly over 52 weeks, during which you'll take either the new medication or a placebo by mouth. You won't know which one you're receiving. The study team will monitor you with blood tests, exams, and check-ins to see how the treatment affects your pain crises and overall health. If you complete the first 52 weeks and want to continue, you may be invited to join an open-label extension period where you'll receive the active medication for an additional year.

AI-generated summary from trial data · Aug 2, 2026 · Not medical advice

Testing a new medication to reduce pain crises in sickle cell disease

What is this trial?

Do you qualify?

What participation looks like

Trial locations(63 sites)