Realizing Effectiveness Across Continents With Hydroxyurea

NCT06171217 | Active Not Recruiting Phase 2 DMC

• 2 other identifiers: 2013-4221bU01HL133883
• Study Type: interventional
• Target: 811
• Locations: 4 countries
• Sites: 4

Brief Summary

REACH is a prospective, phase I/II open-label dose escalation trial of hydroxyurea for children with confirmed SCA between 3 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.

Conditions

Sickle Cell Disease; Children

Outcome Measures

Primary Outcomes (1)

• Efficacy of long-term Hydroxyurea treatment at MTD

  The efficacy of hydroxyurea will be primarily assessed through fetal hemoglobin (HbF), comparing treatment with baseline values.

  Assessed every 6 ± 1 months up to 204 months

Secondary Outcomes (2)

• Reduction of malaria incidents while on hydroxyurea at MTD.

  Assessed every 4 ± 1 weeks, then every 3 ± 1months up to 204 months

• Success of PK-guided dosing of hydroxyurea

  Assessed every 6 ± 1 months up to 204 months

Study Arms (2)

Original Cohort

EXPERIMENTAL

The original REACH cohort continuing study treatment per the protocol schedule of evaluations.

Drug: Hydroxyurea

New Cohort

EXPERIMENTAL

Newly enrolled REACH participants consent, 3 months screening, and treatment per the protocol schedule of evaluations

Drug: Hydroxyurea

Interventions

Hydroxyurea DRUG

Hydroxyurea, approximately 20-30 mg/kg/day, with modifications for toxicity or for mild marrow suppression

Original Cohort

Eligibility Criteria

• Age: 3 Years - 10 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Pediatric patients with documented sickle cell anemia (typically HbSS supported by hemoglobin electrophoresis, complete blood count, and peripheral blood smear)
• In the Original Cohort, age range of 1.00-9.99 years, inclusive, at the time of enrollment (now age 5.5 - 17.5 years); age range 3.0-10.0 years for the New Cohort
• Weight at least 10.0 kg at the time of enrollment
• Parent or guardian willing and able to provide written informed consent, with child's verbal assent as per local IRB/Ethics Board requirements
• Willingness to comply with all study-related treatments, evaluations, and follow-up

You may not qualify if:

• Known medical condition making participation ill-advised (e.g., acute or chronic infectious disease, HIV, or malignancy)
• Acute or chronic severe malnutrition determined by impaired growth parameters as defined by WHO (weight for length/height or height for age \>3 z-scores below the median WHO growth standards.
• Anemia: Hb \<4.0 g/dL
• Anemia: Hb \<6.0 g/dL with ARC \<100 x 109/L
• Reticulocytopenia: ARC \<80 x 109/L with Hb \<7.0 g/dL
• Thrombocytopenia: Platelets \<80 x 109/L
• Neutropenia: ANC \<1.0 x 109/L

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Sites (4)

Hospital Pediátrico David Bernardino

Luanda, Angola

Location

Centre Hospitalier Monkole

Kinshasa, Democratic Republic of the Congo

Location

KEMRI/Wellcome Trust Research

Kilifi, Kenya

Location

Mbale Regional Hospital

Mbale, Uganda

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Hydroxyurea

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Urea; Amides; Organic Chemicals

Study Officials

• Russell Ware, MD, PhD

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: The original cohort will continue treatment per planned protocol schedule of evaluations, and a new cohort will join with consent, a 3-month screening period, and treatment per planned protocol schedule of evaluations.

Study Record Dates

• First Submitted: August 21, 2023
• First Posted: December 14, 2023
• Study Start: October 27, 2023
• Primary Completion (Estimated): October 4, 2027
• Study Completion (Estimated): October 4, 2033
• Last Updated: April 21, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: Nine months following publication ending 36 months following publication.
• Access Criteria: For individual participant data meta-analysis after approval of a proposal and with a signed data access agreement.

Locations

KE (1); AN (1); DE (1); UG (1)