Realizing Effectiveness Across Continents With Hydroxyurea (REACH): A Phase I/II Pilot Study Of Hydroxyurea For Children With Sickle Cell Anemia

Locations

1 Angola site

1 Kenya site

1 Congo, The Democratic Republic of the site

1 Uganda site

Age

1 to 10 Years

Genotypes

HbSS

Phase

Phase 1/Phase 2

Study type

Interventional

Gender

All

Interventions

Drug

Compensation

Unknown

About the study

REACH is a prospective, phase I/II open-label dose escalation trial of hydroxyurea for
children with confirmed SCA between 12 months and 10 years of age. The short-term goal is to
obtain critical pilot data regarding the feasibility, safety, and benefit of hydroxyurea for
children with SCA in multiple distinct research settings in Africa. Based on that
information, the longer-term goal is to make hydroxyurea more widely available for children
with SCA in Africa, particularly those identified with SCA through expanded newborn screening
programs.

participation requirements

1. Pediatric patients with documented sickle cell anemia (typically HbSS supported by
hemoglobin electrophoresis, complete blood count, and peripheral blood smear)

2. Age range of 1.00-9.99 years, inclusive, at the time of enrollment

3. Weight at least 10.0 kg at the time of enrollment

4. Parent or guardian willing and able to provide written informed consent, with child’s
verbal assent as per local IRB/Ethics Board requirements

5. Willingness to comply with all study-related treatments, evaluations, and follow-up

participation restrictions

1. Known medical condition making participation ill-advised, (e.g., acute or chronic
infectious disease, HIV, or malignancy)

2. Acute or chronic severe malnutrition determined by impaired growth parameters as
defined by WHO (weight for length/height or weight-for-length/height >3 z-scores below
the median WHO growth standards, as defined in Appendix I)

3. Pre-existing severe hematological toxicity (temporary exclusions)

1. Anemia: Hb <4.0 gm/dL 2. Anemia: Hb <6.0 gm/dL with ARC <100 x 109/L 3. Reticulocytopenia: ARC <80 x 109/L with Hb <7.0 gm/dL 4. Thrombocytopenia: Platelets <80 x 109/L 5. Neutropenia: ANC <1.0 x 109/L 4. Blood transfusion within 60 days before enrollment (temporary exclusion) 5. Hydroxyurea use within 6 months before enrollment

Locations

  • Luanda, Angola, Hospital Pedi√°trico David Bernardino
  • Kinshasa, Congo, The Democratic Republic of the, Centre Hospitalier Monkole
  • Kilifi, Kenya, KEMRI/Wellcome Trust Research
  • Mbale, Uganda, Ministry of Health Mbale Regional Hospital