Hydroxyurea for Sickle Cell Anemia

(ENHANCE Trial)

Overseen By Teresa Latham, Research Director, DrPH

Age: Any Age

Sex: Any

Trial Phase: Phase 4

Sponsor: Children's Hospital Medical Center, Cincinnati

Must be taking: Hydroxyurea

Disqualifiers: Regular transfusions, HbSC, HbSβ+

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 10 years

50 Participants Needed

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. ^{1 2 3 4 5}

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. ^{1 2 4 5 6}

Who 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

I have been diagnosed with sickle cell anemia or sickle-β0-thalassemia.

I am at least 6 months old.

I have decided with my family and doctor to start hydroxyurea treatment.

Exclusion Criteria

I have either HbSC disease or HbSβ+ thalassemia.

I am currently receiving regular blood transfusions.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive hydroxyurea with PK-optimized dosing to study its effects on sickle cell anemia

10 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Long-term monitoring

Long-term monitoring of organ systems and hematological parameters to assess the sustained effects of hydroxyurea

10 years

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?

Group I: Observational Treatment Group (Single Arm) Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Children's Hospital Medical Center, Cincinnati

Lead Sponsor

Trials

844

Recruited

6,566,000+

Greater Cincinnati Foundation

Collaborator

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11546997/

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-hydroxyurea

Evaluating 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-scd

Hydroxyurea 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-z

Role 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.

6. nejm.org nejm.org/doi/full/10.1056/NEJMp2511272

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.