Effectiveness of Nontraditional Hydroxyurea Algorithms: Novel and Clinical Evaluations (ENHANCE)

Conditions

• Sickle Cell Anemia (HbSS)
• Sickle-β0-thalassemia (HbSβ0)

Eligibility Criteria

Sex

ALL

Age

6 Months - N/A

Healthy Volunteers

Not accepted

Interventions

• PK-optimized oral hydroxyurea at MTD until 15 years of age. — DRUG
  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).

Study Details

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.

Key Dates

Start date

Dec 19, 2024

Status verified

Aug 2025

Primary completion

Dec 31, 2026

Completion

Dec 31, 2028

Study Design

Enrollment

50 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

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.

Primary Outcome Measure

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

Central Contacts

• Wendi L. Long, Sr. Regulatory Specialist, BS, CCRC
  513-803-3064
  [email protected]
• Teresa Latham, Research Director, DrPH
  (513) 803-7922
  [email protected]

Locations (1)

Find similar trials in Cincinnati, OH

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