Can a Decades-Old sickle cell drug also fix the lungs?
NCT ID NCT00350844
First seen Sep 24, 2026 · Last updated Sep 25, 2026 · Updated 1 time
Summary
Children and young adults with sickle cell disease often develop high blood pressure in the lungs, which raises the risk of heart problems. This small trial tests whether hydroxyurea, a drug already used for sickle cell disease, can lower that lung pressure. Participants take hydroxyurea at increasing doses while researchers track heart ultrasound measurements and blood markers over a year.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- hydroxyurea, a drug already used to treat sickle cell disease
- What this could lead to
- If it works, hydroxyurea could become a targeted way to treat high lung blood pressure in young people with sickle cell disease, a complication with few good options.
- What could go wrong
- This is a tiny early-phase study of six people, so any signal may not hold up in larger trials. Hydroxyurea can lower blood counts and carries other risks that need monitoring.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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6 people
The number who actually took part.
- Start date
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Jul 2006
- Finished
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Jun 2008
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
10 to 25 years
- Sex
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Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Age between 10 and 25 years old * Sickle cell disease with hemoglobin SS, SC or S-B\^0 thalassemia confirmed on hemoglobin electrophoresis * Tricuspid regurgitant jet velocity (TRJV) equal to or greater than 2.5 m/sec on 2 baseline Doppler echocardiograms at least 3 months apart Exclusion Criteria: * Patients already being treated with hydroxyurea * Patients on a chronic transfusion protocol * Patients with evidence of hepatic (alanine aminotransferase \[ALT\] equal to or greater than 2 SD above normal) or renal dysfunction (creatinine \[Cr\] equal to or greater than 2 SD above normal) * Patients who are pregnant * Patients with documented causes of severe pulmonary hypertension other than from SCD
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Ann & Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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