# Realizing Effectiveness Across Continents with Hydroxyurea(REACH): A Phase I/II Pilot Study of Hyroxyurea for Children with Sickle Cell Anemia

> **NIH NIH U01** · CINCINNATI CHILDRENS HOSP MED CTR · 2020 · $676,152

## Abstract

ABSTRACT
Sickle cell anemia (SCA) is among the world’s most common inherited blood disorders, and causes both morbidity and early
mortality. SCA is highly prevalent in Africa, affecting over 300,000 births annually, with projections for an increasing burden in
the next generation. In the US, infants with SCA are identified by newborn screening; simple interventions have dramatically
reduced morbidity and mortality. In Africa, by contrast, neonatal screening is not available, and most affected infants will
die before age 5 years, without proper diagnosis and treatment of SCA. The World Health Organization (WHO) notes SCA
contributes substantially to under-5 mortality for many African countries. Several pilot SCA screening programs have begun in
sub-Saharan Africa, which document a large burden (10-25% sickle trait and 1-2% SCA). With better screening and early care
and treatment programs, more children with SCA in Africa will likely survive, but their medical needs will strain the limited
healthcare resources. Accordingly, there is an urgent need to investigate the role of hydroxyurea for children with SCA in
Africa, since hydroxyurea is the only realistic and affordable disease-modifying therapy in this setting. To date, hydroxyurea
has been studied only in developed countries, with virtually no data available regarding its safe and effective use in Africa. To
address this critical unmet need, we developed and launched the REACH (Realizing Effectiveness Across Continents with
Hydroxyurea) trial, ClinicalTrials.gov NCT01966731, a prospective study of hydroxyurea designed to gather critical data
regarding the safety, feasibility, and benefits of hydroxyurea for young children in sub-Saharan Africa. REACH is also designed
to gather important data about why children have different responses to hydroxyurea treatment; using novel and innovative
approaches, we will collect data regarding the inter-patient variability by analysis of hydroxyurea pharmacokinetics,
pharmacodynamics, and pharmacogenomics. A total of 600 children with SCA between 1-10 years of age will enroll and first
receive fixed dose hydroxyurea, then dose escalation to the maximum tolerated dose (MTD), followed by maintenance
treatment to a common study termination date. We have obtained all drug supply in donation from Bristol-Myers Squibb,
performed a rigorous site selection process to identify sites able to perform high-quality research, and four locations (Luanda
Angola, Kinshasa DRC, Kilifi, Kenya; and Mbale, Uganda) are actively enrolling children and providing protocol-directed
hydroxyurea treatment. Using an adaptive study design and the REDCap electronic database system through our Clinical &
Translational Science Award program, we are collecting data on the safety, feasibility, and benefits of hydroxyurea for SCA
within Africa. We will also collect novel information about inter-patient variability by creating a hydroxyurea pharmacokinetics
profile for each participant, and then...

## Key facts

- **NIH application ID:** 10001581
- **Project number:** 5U01HL133883-04
- **Recipient organization:** CINCINNATI CHILDRENS HOSP MED CTR
- **Principal Investigator:** Russell E Ware
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $676,152
- **Award type:** 5
- **Project period:** 2017-08-01 → 2022-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10001581

## Citation

> US National Institutes of Health, RePORTER application 10001581, Realizing Effectiveness Across Continents with Hydroxyurea(REACH): A Phase I/II Pilot Study of Hyroxyurea for Children with Sickle Cell Anemia (5U01HL133883-04). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10001581. Licensed CC0.

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