Recruiting

Hydroxyurea

Sponsor:

Children's Hospital Medical Center, Cincinnati

Code:

NCT07177300

Conditions

Sickle Cell Anemia (HbSS)

Sickle-β0-thalassemia (HbSβ0)

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Interventions

PK-optimized oral hydroxyurea at MTD until 15 years of age.

Study Details

Brief summary:

The main reason for this research study is to learn more about hydroxyurea and the treatment of sickle cell anemia (SCA). Hydroxyurea is a medication that has been studied for many years and has been shown to provide benefits for people with SCA.

In this research study, the investigators hope to learn more about how to improve the dosing and monitoring of hydroxyurea and learn more about the long-term effects of hydroxyurea over time. Hydroxyurea is usually dosed based only on your weight. Our study will use a new way to select a starting dose that is based on how each patient absorbs hydroxyurea.

Conditions

Sickle Cell Anemia (HbSS)

Sickle-β0-thalassemia (HbSβ0)

Study ID

NCT07177300

Start date

Dec 19, 2024

Status verified date

Aug, 2025

Completion date

Dec, 2028

Anticipated

Primary completion date

Dec, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Diagnosis of sickle cell anemia (HbSS) or sickle-β0-thalassemia (HbSβ0)
  • Age 6 months at the time of enrollment
  • Clinical decision by patient, family, and healthcare provider to initiate hydroxyurea therapy

Exclusion Criteria:

  • Current treatment with regularly scheduled blood transfusions
  • Sickle-hemoglobin C disease (HbSC), sickle-β+-thalassemia (HbSβ+)

Study Design

Enrollment

50 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Observational Treatment Group (Single Arm)

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.

Interventions

PK-optimized oral hydroxyurea at MTD until 15 years of age.

Because people are different, we will measure how each participant's body absorbs and eliminates the medicine, hydroxyurea, using blood tests. This information will be used to determine the best dose for each participant (rather than using the same weight-based dose for everyone).

Primary outcome measure

  • Composite Organ Injury [ Time Frame: Through study completion, an average of 10 years ]

Central Contacts and Locations

Central contacts

Wendi L. Long, Sr. Regulatory Specialist, BS, CCRC

513-803-3064wendi.long@cchmc.org

Teresa Latham, Research Director, DrPH

(513) 803-7922teresa.latham@cchmc.org

Locations

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229

Contacts

Charles T. Quinn, M.D., M.S.

513-803-3086charles.quinn@cchmc.org

Megan Metcalf, CRC, III

megan.metcalf@cchmc.org

More Information

Sponsor

Children's Hospital Medical Center, Cincinnati

Last update posted

Sep 16, 2025

Last verified

Aug, 2025

Keywords

  • sickle cell anemia
  • SCD
  • sickle cell
  • hydroxyurea

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Children's Hospital Medical Center, Cincinnati on 2025-09-16.