Recruiting

Testing a New Medication for Sickle Cell Disease

Plain-English translation of NCT01245179 on ClinicalTrials.gov ↗ · Source last updated January 2026 · Translation generated June 2026 · How we translate trials

Read our Sickle Cell Disease research guide →

Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.

What is this trial?

This is an early-stage study testing a medication called to see if it's safe and tolerable for adults with sickle cell disease. The study focuses on people whose disease hasn't improved with standard therapy (hydroxyurea) or who cannot tolerate that treatment. Researchers will gradually increase the dose of this medication to find the safest and most effective level.

Many people with sickle cell disease don't respond well to current standard treatments or experience side effects that make them unable to continue. This medication may work through a different mechanism to help reduce the complications of sickle cell disease, offering a new option for patients who need better treatment choices.

Do you qualify?

You likely qualify if…

you are at least 18 years old
you have been diagnosed with sickle cell disease (homozygous SS or S-beta0 thalassemia)
you cannot tolerate hydroxyurea, have chosen to refuse it, or it has not worked for you
you have had significant sickle cell complications in the past year, such as at least two hospitalizations, three or more severe pain crises, leg ulcers, acute chest syndrome, stroke, or priapism
you are able to give written informed consent and understand the study

You likely don't qualify if…

you have taken other medications that raise fetal hemoglobin levels within the past 60 days (except if you stopped more than 60 days ago)
you have had a severe pain crisis requiring injectable pain medication within the past 2 weeks
you have stomach or intestinal problems that would prevent you from absorbing oral medication properly
you are currently on a chronic blood transfusion schedule or have received a recent transfusion
you have significant heart problems, including low heart function, irregular heartbeats, or a pacemaker

What participation looks like

You will attend a screening visit where the research team will perform blood tests, heart tests (ECG and echocardiogram), and physical exams to make sure you're eligible. If you qualify, you'll enter a 12-week treatment phase during which you'll take the medication three times per week (Monday, Wednesday, and Friday) at a dose assigned by the research team. You'll have regular follow-up visits to monitor how you're tolerating the medication and to watch for any side effects, followed by a post-treatment follow-up period.

AI-generated summary from trial data · Jun 6, 2026 · Not medical advice

Testing a New Medication for Sickle Cell Disease

What is this trial?

Do you qualify?

What participation looks like

Trial locations