Hydroxyurea for Sickle Cell Anemia
(ENHANCE Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial focuses on learning more about hydroxyurea, a medication for treating sickle cell anemia. Researchers aim to find better methods for determining the right dose and monitoring its long-term effects. The study specifically examines how children with sickle cell anemia absorb the medication to optimize dosing. It is suitable for children diagnosed with sickle cell anemia who are beginning hydroxyurea treatment. As a Phase 4 trial, this research helps to understand how this FDA-approved and effective treatment can benefit more patients.
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.
What is the safety track record for hydroxyurea?
Research has shown that hydroxyurea has been safely used for many years to treat sickle cell anemia (SCA). Studies have found it reduces emergency room visits and hospital stays for children with this condition. Long-term use of hydroxyurea has also been linked to lower death rates in patients. Although some uncertainty exists about its safety for certain patients, such as those with a specific type of sickle cell disease called HbSC, many others have tolerated it well. Overall, hydroxyurea has a strong record of safety and effectiveness in managing SCA.12345
Why are researchers enthusiastic about this study treatment?
Hydroxyurea is unique because it optimizes dosing specifically for children with sickle cell anemia, focusing on their age group of 6 months to 5 years. While most treatments for sickle cell anemia, such as blood transfusions and pain management, address symptoms, hydroxyurea helps increase fetal hemoglobin levels, which can reduce the frequency of painful episodes and other complications. Researchers are excited about this treatment because it targets the root cause of the disease more effectively in young children, potentially improving their quality of life significantly from an early age.
What is the effectiveness track record for hydroxyurea in treating sickle cell anemia?
Research has shown that hydroxyurea effectively treats sickle cell anemia (SCA). Studies have found that it increases hemoglobin levels, which are crucial for oxygen transport in the blood. Hydroxyurea also reduces emergency room visits and hospital stays for children with SCA. Long-term use has demonstrated lasting benefits, making it a reliable treatment option. Additionally, higher doses have been linked to significant improvements in blood tests related to SCA. Overall, hydroxyurea has a strong track record for managing symptoms and complications of sickle cell anemia.
In this trial, children with sickle cell anemia who start hydroxyurea for clinical indicators between 6 months and 5 years of age can participate in this observational study with PK-optimized hydroxyurea dosing.12456Who Is on the Research Team?
Charles T. Quinn, Professor of Pediatrics, M.D., M.S.
Principal Investigator
Children's Hospital Medical Center, Cincinnati
Are You a Good Fit for This Trial?
This trial is for individuals with sickle cell anemia or disease. Participants should be willing to take hydroxyurea orally and be monitored until they are 15 years old. Specific eligibility criteria details were not provided.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive hydroxyurea with PK-optimized dosing to study its effects on sickle cell anemia
Follow-up
Participants are monitored for safety and effectiveness after treatment
Long-term monitoring
Long-term monitoring of organ systems and hematological parameters to assess the sustained effects of hydroxyurea
What Are the Treatments Tested in This Trial?
Interventions
- Hydroxyurea
Trial Overview
The study tests a new dosing method of hydroxyurea, optimized based on individual absorption rather than just weight. The goal is to improve treatment outcomes for those with sickle cell anemia over time.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
All children with sickle cell anemia who are started on hydroxyurea for clinical indicators between 6 months and 5 years of age can do so on this observational study with PK-optimized hydroxyurea dosing.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Children's Hospital Medical Center, Cincinnati
Lead Sponsor
Greater Cincinnati Foundation
Collaborator
Citations
The Current Role of Hydroxyurea in the Treatment of Sickle ...
This study showed that the MTD achieved greater increases in HbF and total hemoglobin levels, as well as a more significant reduction in white ...
2.
ashpublications.org
ashpublications.org/bloodadvances/article/9/14/3585/536727/Evaluating-the-long-term-benefits-of-hydroxyureaEvaluating the long-term benefits of hydroxyurea in pediatric ...
This study demonstrates that hydroxyurea has sustained clinical benefits in reducing ED visits and hospital days across years of use in children with SCA.
3.
hematology.org
hematology.org/newsroom/press-releases/2025/hydroxyurea-effective-long-term-in-children-living-with-scdHydroxyurea Effective Long Term in Children Living With ...
Hydroxyurea remains effective long-term in reducing emergency department visits and hospital days for children living with sickle cell disease (SCD), according ...
4.
hematologyadvisor.com
hematologyadvisor.com/features/hydroxyurea-for-sickle-cell-anemia-in-children/Hydroxyurea for Sickle Cell Anemia in Children
Researchers determined long-term treatment with hydroxyurea is beneficial for children with sickle cell anemia (SCA).
5.
systematicreviewsjournal.biomedcentral.com
systematicreviewsjournal.biomedcentral.com/articles/10.1186/s13643-024-02461-zRole of hydroxyurea therapy in the prevention of organ ...
Our study found that a hydroxyurea dose above 20 mg/kg/day with a mean rise in HbF by 18.46% post-hydroxyurea therapy had a beneficial role.
Thirty Years of Hydroxyurea for Sickle Cell Anemia
Additional follow-up research involving patients in the MSH trial found that, after 9 years, hydroxyurea was associated with reduced mortality.
Unbiased Results
We believe in providing patients with all the options.
Your Data Stays Your Data
We only share your information with the clinical trials you're trying to access.
Verified Trials Only
All of our trials are run by licensed doctors, researchers, and healthcare companies.