Sickle Cell DiseasePhase 2REACHNCT06171217
Realizing Effectiveness Across Continents With Hydroxyurea
A Phase 2 study testing Hydroxyurea in people with Sickle Cell Disease and Children, run by Children's Hospital Medical Center, Cincinnati at 4 sites in 4 countries.
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… Read more about this study
- Who is it for?
- Ages 3 to 10 years
- Conditions the study is for
- Sickle Cell Disease, Children
- What is being tested?
- Hydroxyurea
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 811 participants
- Where
- 4 sitesin 4 countries
- Study dates
- 27 Oct 2023to 4 Oct 2033
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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.
- Pre-existing severe hematological toxicity (temporary exclusions)
- 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
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- Thrombocytopenia: Platelets <80 x 109/L
- Neutropenia: ANC <1.0 x 109/L
- Blood transfusion within 60 days before enrollment (temporary exclusion)
- 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.
Trial locations and contacts
4 sites in 4 countries.
4 sites in 4 countries
Angola1 site
Hospital Pediátrico David Bernardino
Luanda
Democratic Republic of the Congo1 site
Centre Hospitalier Monkole
Kinshasa
About this study
Official title: Realizing Effectiveness Across Continents With Hydroxyurea: A Phase I/II Prospective Trial of Hydroxyurea for Children With Sickle Cell Anemia
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.
Read the detailed 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.
- Conditions
- Sickle Cell Disease, Children
- Being tested
- Hydroxyurea
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HydroxyureaDrug
Hydroxyurea, approximately 20-30 mg/kg/day, with modifications for toxicity or for mild marrow suppression
HydroxyureaDrug
Hydroxyurea 15-35 mg/kg/day based on PK-guided dosing, with modifications for toxicity for mild marrow suppression
| Study group | Role | What they receive |
|---|---|---|
| Original Cohort | Experimental | The original REACH cohort continuing study treatment per the protocol schedule of evaluations. Receives: Drug: Hydroxyurea |
| New Cohort | Experimental | Newly enrolled REACH participants consent, 3 months screening, and treatment per the protocol schedule of evaluations Receives: Drug: Hydroxyurea |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Assessed every 6 ± 1 months up to 204 months
Secondary outcomes (2)
Reduction of malaria incidents while on hydroxyurea at MTD.
Clinical malaria infections to identify associations of risk or protection comparing treated and untreated incidents.
Measured: Assessed every 4 ± 1 weeks, then every 3 ± 1months up to 204 months
Success of PK-guided dosing of hydroxyurea
Fetal Hemoglobin changes from hydroxyurea at MTD
Measured: Assessed every 6 ± 1 months up to 204 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Dec 2023
- Study start27 Oct 2023
- Primary completion4 Oct 2027 (estimated)
- Study completion4 Oct 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- 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.
- Masking
- None (open label)
- Enrolment
- 811 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Russell Ware, MD, PhD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06171217
- Sponsor's protocol ID
- 2013-4221b
- Other IDs
- U01HL133883
- Acronym
- REACH
- Status last verified
- Apr 2026
- First submitted
- 21 Aug 2023
- Last updated
- 21 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Investigators whose proposed use of the data has been approved by a review committee identified for this purpose, will be provided individual participant data that underlie the results reported from this study, after de-identification and publication. The data can be obtained via email to Dr. Ware as the corresponding author.
Publications
Backeljauw P, Tomlinson G, Smart LR, Tshilolo L, Williams TN, Santos B, Olupot-Olupot P, Stuber S, Lane A, Latham T, Ware RE. Growth and puberty in African children with sickle cell anemia treated with hydroxyurea. Blood Adv. 2026 Jul 14;10(13):4483-4494. doi: 10.1182/bloodadvances.2025019511.
PubMed 41954645
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Realizing Effectiveness Across Continents With Hydroxyurea: A Phase I/II Prospective Trial of Hydroxyurea for Children With Sickle Cell Anemia
- ClinicalTrials.gov ID
- NCT06171217
- Acronym / sponsor ID
- REACH / 2013-4221b
- Conditions
- Sickle Cell Disease, Children
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 811 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 27 Oct 2023 (actual)
- Primary completion
- 4 Oct 2027 (estimated)
- Study completion
- 4 Oct 2033 (estimated)
- First posted
- 14 Dec 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| Original Cohort | Experimental | Drug: HydroxyureaThe original REACH cohort continuing study treatment per the protocol schedule of evaluations. |
| New Cohort | Experimental | Drug: HydroxyureaNewly enrolled REACH participants consent, 3 months screening, and treatment per the protocol schedule of evaluations |
Who may qualify
- Ages
- 3 to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (10 listed)
- 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.
- Pre-existing severe hematological toxicity (temporary exclusions)
- Blood transfusion within 60 days before enrollment (temporary exclusion)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HydroxyureaHydroxyurea, approximately 20-30 mg/kg/day, with modifications for toxicity or for mild marrow suppression | Drug | Original Cohort |
| HydroxyureaHydroxyurea 15-35 mg/kg/day based on PK-guided dosing, with modifications for toxicity for mild marrow suppression | Drug | New Cohort |
Primary outcome
Efficacy of long-term Hydroxyurea treatment at MTD
Time frame: Assessed every 6 ± 1 months up to 204 months
The efficacy of hydroxyurea will be primarily assessed through fetal hemoglobin (HbF), comparing treatment with baseline values.
2 secondary outcomes
Reduction of malaria incidents while on hydroxyurea at MTD.
Time frame: Assessed every 4 ± 1 weeks, then every 3 ± 1months up to 204 months
Success of PK-guided dosing of hydroxyurea
Time frame: Assessed every 6 ± 1 months up to 204 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Apr 2026.
- Derived from this study: PubMed 41954645Backeljauw P, Tomlinson G, Smart LR, Tshilolo L, Williams TN, Santos B, Olupot-Olupot P, Stuber S, Lane A, Latham T, Ware RE. Growth and puberty in African children with sickle cell anemia treated with hydroxyurea. Blood Adv. 2026 Jul 14;10(13):4483-4494. doi: 10.1182/bloodadvances.2025019511.
- Individual participant dataSharing planned: Yes. Investigators whose proposed use of the data has been approved by a review committee identified for this purpose, will be provided individual participant data that underlie the results reported from this study, after de-identification and publication. The data can be obtained via email to Dr. Ware as the corresponding author.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Russell Ware, MD, PhD principal investigator
Children's Hospital Medical Center, Cincinnati
4 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Hydroxyurea in people with Sickle Cell Disease and Children, run by Children's Hospital Medical Center, Cincinnati at 4 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 21 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Angola, Democratic Republic of the Congo, Kenya, Uganda. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy of long-term Hydroxyurea treatment at MTD (time frame: Assessed every 6 ± 1 months up to 204 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06171217, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Apr 2026.