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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06171217
Other study ID # 2013-4221b
Secondary ID U01HL133883
Status Recruiting
Phase Phase 2
First received
Last updated
Start date October 27, 2023
Est. completion date October 4, 2033

Study information

Verified date December 2023
Source Children's Hospital Medical Center, Cincinnati
Contact Russell Ware, MD, PhD
Phone 513-803-4597
Email russell.ware@cchmc.org
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Description:

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. In the REACH prospective trial, the Original Cohort will receive long-term treatment while for the New Cohort, treatment will continue at least 4 years using PK-guided dosing after an initial 3-month screening period.


Recruitment information / eligibility

Status Recruiting
Enrollment 822
Est. completion date October 4, 2033
Est. primary completion date October 4, 2027
Accepts healthy volunteers No
Gender All
Age group 3 Years to 10 Years
Eligibility Inclusion Criteria 1. Pediatric patients with documented sickle cell anemia (typically HbSS supported by hemoglobin electrophoresis, complete blood count, and peripheral blood smear) 2. 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 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 Exclusion Criteria 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 height for age >3 z-scores below the median WHO growth standards. 3. Pre-existing severe hematological toxicity (temporary exclusions) 1. Anemia: Hb <4.0 g/dL 2. Anemia: Hb <6.0 g/dL with ARC <100 x 109/L 3. Reticulocytopenia: ARC <80 x 109/L with Hb <7.0 g/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. In the Original Cohort, hydroxyurea use within 6 months before enrollment (temporary exclusion). In the New Cohort, the children should be hydroxyurea naïve, without any prior treatment exposure.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Hydroxyurea
Hydroxyurea, approximately 20-30 mg/kg/day, with modifications for toxicity or for mild marrow suppression
Hydroxyurea
Hydroxyurea 15-35 mg/kg/day based on PK-guided dosing, with modifications for toxicity for mild marrow suppression

Locations

Country Name City State
Angola Hospital Pediátrico David Bernardino Luanda
Congo, The Democratic Republic of the Centre Hospitalier Monkole Kinshasa
Kenya KEMRI/Wellcome Trust Research Kilifi
Uganda Mbale Regional Hospital Mbale

Sponsors (2)

Lead Sponsor Collaborator
Children's Hospital Medical Center, Cincinnati National Heart, Lung, and Blood Institute (NHLBI)

Countries where clinical trial is conducted

Angola,  Congo, The Democratic Republic of the,  Kenya,  Uganda, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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 Reduction of malaria incidents while on hydroxyurea at MTD. Clinical malaria infections to identify associations of risk or protection comparing treated and untreated incidents. Assessed every 4 ± 1 weeks, then every 3 ± 1months up to 204 months
Secondary Success of PK-guided dosing of hydroxyurea Fetal Hemoglobin changes from hydroxyurea at MTD Assessed every 6 ± 1 months up to 204 months
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