Anemia, Sickle CellPhase 1/2ACHiEvE-SCDNCT05451940

Hydroxyurea and EPO in Sickle Cell Disease

A Phase 1/2 study testing Hydroxyurea and Epoetin Alfa-BioSimilar in people with Anemia, Sickle Cell and Sickle Cell Disease, run by Julia Xu at 2 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The proposed study is a Phase 1/2 multi-center study evaluating the safety and efficacy of erythropoietin (EPO) in combination with hydroxyurea in the treatment of chronic anemia in patients with sickle cell disease (SCD). Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Anemia, Sickle Cell, Sickle Cell Disease
What is being tested?: Hydroxyurea and Epoetin Alfa-BioSimilar
Type of treatment: Pill or oral medicine, Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 17 participants
Where: 2 sitesin 2 countries
Study dates: 25 May 2023to 27 Feb 2025
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Aged ≥ 18 years
Confirmed diagnosis of SCD (HbSS or HbS/β0-thalassemia genotypes)
Screening Hb ≤ 9.0 g/dL
Screening transferrin saturation ≥ 20% and ferritin ≥ 50 ng/mL
Must be on stable-dose hydroxyurea treatment (i.e., no changes in dose within 60 days prior to start of study drug) and plan to continue taking hydroxyurea at the same dose and schedule during the study
If receiving L-glutamine or crizanlizumab, must have been receiving the drug at a stable dose for at least 60 days prior to screening and plan to continue taking the drug at the same dose and schedule during the study

Who cannot join

Participating in a chronic transfusion program (pre-planned series of transfusions for prophylactic purposes) and/or planning on undergoing an exchange transfusion during the duration of the study; episodic transfusion in response to worsened anemia or VOC is permitted, but participant should not have received a blood transfusion within 60 days of start of study drug
Received voxelotor or EPO within 30 days of start of study drug
Untreated iron deficiency, or had initiation or change in dose of supplemental iron within 30 days of start of study drug
Ongoing acute illness, infection, or VOC within 2 weeks of start of study drug
Arterial or venous thrombosis within 180 days of start of study drug
Grade 3 hypertension (defined as systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥100 mmHg; medical intervention indicated; more than one drug or more intensive therapy than previously used indicated) on two consecutive measurements

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Unstable angina, uncontrolled seizure disorder, or active malignancy
End-stage renal disease requiring hemodialysis
Current pregnancy or breastfeeding
Received active treatment on another investigational trial within 30 days (or 5 half-lives of that agent, whichever is greater) prior to start of study drug or plans to participate in another investigational drug trial

Trial locations and contacts

2 sites in 2 countries.

Country

City, postcode or hospital

2 sites in 2 countries

United States1 site

UPMC
Pittsburgh, Pennsylvania, 15213

Other trials in United States

Nigeria1 site

Lagos University Teaching Hospital
Lagos, 102215

Other trials in Nigeria

About this study

Official title: Assessing Combination Hydroxyurea and Exogenous Erythropoietin in Sickle Cell Disease

Read the detailed description

Sickle cell disease (SCD) is a devastating inherited hemoglobin disorder characterized by recurrent episodes of pain and chronic hemolytic anemia. Chronic anemia contributes to multi-organ damage and decreased life expectancy in SCD. However, there are limited treatment options for anemia in SCD. Erythropoietin (EPO) is the standard of care for treatment of anemia related to chronic kidney disease (CKD) and is also used ad hoc in patients with SCD. However, there is limited data on the safety and efficacy of EPO in patients with SCD, especially in combination with hydroxyurea. Therefore, this study aims to treat patients on stable hydroxyurea therapy with subcutaneous EPO, with the goal of assessing the safety of EPO therapy and its effect on chronic anemia in SCD.

(Note: Outcome measure changes were in place prior to study initiation.)

Conditions: Anemia, Sickle Cell, Sickle Cell Disease
Being tested: Hydroxyurea, Epoetin Alfa-BioSimilar
Keywords: Sickle cell disease, Anemia, Erythropoietin, Hydroxyurea, Sickle cell anemia
Medical subject headings: Anemia, Sickle Cell, Anemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

HydroxyureaDrug
Also called Droxia, Hydrea, and hydroxycarbamide.
Hydroxyurea is an orally available antimetabolite medication that has been shown to reduce the frequency of painful crises and acute chest syndrome in adults and children with sickle cell disease. Hydroxyurea treats sickle cell disease by a number of different mechanisms, including increasing the expression of fetal hemoglobin (HbF), which reduces sickling of red blood cells.
Epoetin Alfa-BioSimilarDrug
Also called EPO, epoetin, erythropoietin, erythropoiesis-stimulating agent, ESA, haematopoietin, haemopoietin
Epoetin alfa and its biosimilars are first-generation erythropoiesis-stimulating agents (ESAs), which are recombinant versions of erythropoietin (EPO) produced using recombinant DNA technology. Erythropoietin (EPO) is a glycoprotein hormone, naturally produced mainly in the kidneys in response to hypoxia and stimulates red blood cell production (erythropoiesis) in the bone marrow.

Study groups in NCT05451940
Study group	Role	What they receive
Erythropoietin	Experimental	Subjects on a stable dose of hydroxyurea will be treated with increasing doses of subcutaneous erythropoietin (EPO) as tolerated for an initial 12 weeks, during which the main safety and efficacy endpoints (including the primary endpoint of hemoglobin response) will be assessed. Subjects may continue on treatment for an additional 12 weeks as clinically indicated, with assessment of additional endpoints at the end of the 24-week study period. Receives: Drug: Hydroxyurea; Drug: Epoetin Alfa-BioSimilar

What is being measured

The primary outcome is the main question the study is designed to answer.

Percentage of Participants With Hemoglobin (Hb) Response
Hb response is defined as a Hb increase of ≥ 1.0 g/dL at 12 weeks compared to baseline
Measured: Baseline to 12 weeks

Secondary outcomes (1)

Change in Number of Blood Transfusions Per Year
Annualized number of units of simple red blood cell transfusions received in the 12 months before treatment initiation, compared to during the treatment period
Measured: Baseline to 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

25 May 202327 Feb 2025

First posted11 Jul 2022
Study start25 May 2023
Primary completion13 Dec 2024
Study completion27 Feb 2025

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Anemia, Sickle CellStudies 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 1/2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 17 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05451940
Sponsor's protocol ID: STUDY21120027
Acronym: ACHiEvE-SCD
Status last verified: Dec 2025
First submitted: 24 Jun 2022
Last updated: 7 Jan 2026
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Yes: Data collected may be shared with secondary researchers within or outside the participating centers upon request. The shared data will be deidentified.

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
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Active, not recruitingObservational study
Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease
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Enrolling by invitationPhase 4
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
Pyruvate Kinase Deficiency, Anemia, Hemolytic
Active, not recruitingPhase 2
A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)
Sickle Cell Disease
RecruitingPhase 3
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
Sickle Cell Disease

All Anemia, Sickle Cell trials

Study at a glance

From the registry record, last updated by the sponsor on 7 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Assessing Combination Hydroxyurea and Exogenous Erythropoietin in Sickle Cell Disease
ClinicalTrials.gov ID: NCT05451940
Acronym / sponsor ID: ACHiEvE-SCD / STUDY21120027
Conditions: Anemia, Sickle Cell, Sickle Cell Disease
Keywords: Sickle cell disease, Anemia, Erythropoietin, Hydroxyurea, Sickle cell anemia
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 17 participants (actual)
Lead sponsor: Julia XuUniversity, hospital or other
Collaborators: Carnegie Mellon University, American Society of Hematology
Study start: 25 May 2023 (actual)
Primary completion: 13 Dec 2024 (actual)
Study completion: 27 Feb 2025 (actual)
First posted: 11 Jul 2022 (actual)
Results: Posted 7 Jan 2026

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

(Note: Outcome measure changes were in place prior to study initiation.)

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Erythropoietin	Experimental	Drug: Hydroxyurea, Drug: Epoetin Alfa-BioSimilarSubjects on a stable dose of hydroxyurea will be treated with increasing doses of subcutaneous erythropoietin (EPO) as tolerated for an initial 12 weeks, during which the main safety and efficacy endpoints (including the primary endpoint of hemoglobin response) will be assessed. Subjects may continue on treatment for an additional 12 weeks as clinically indicated, with assessment of additional endpoints at the end of the 24-week study period.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (6 listed)

Aged ≥ 18 years
Confirmed diagnosis of SCD (HbSS or HbS/β0-thalassemia genotypes)
Screening Hb ≤ 9.0 g/dL
Screening transferrin saturation ≥ 20% and ferritin ≥ 50 ng/mL
Must be on stable-dose hydroxyurea treatment (i.e., no changes in dose within 60 days prior to start of study drug) and plan to continue taking hydroxyurea at the same dose and schedule during the study

Key exclusion criteria (10 listed)

Participating in a chronic transfusion program (pre-planned series of transfusions for prophylactic purposes) and/or planning on undergoing an exchange transfusion during the duration of the study; episodic transfusion in response to worsened anemia or VOC is permitted, but participant should not have received a blood transfusion within 60 days of start of study drug
Received voxelotor or EPO within 30 days of start of study drug
Untreated iron deficiency, or had initiation or change in dose of supplemental iron within 30 days of start of study drug
Ongoing acute illness, infection, or VOC within 2 weeks of start of study drug
Arterial or venous thrombosis within 180 days of start of study drug

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
HydroxyureaAlso: Droxia, Hydrea, and hydroxycarbamide.Hydroxyurea is an orally available antimetabolite medication that has been shown to reduce the frequency of painful crises and acute chest syndrome in adults and children with sickle cell disease. Hydroxyurea treats sickle cell disease by a number of different mechanisms, including increasing the expression of fetal hemoglobin (HbF), which reduces sickling of red blood cells.	Drug	Erythropoietin
Epoetin Alfa-BioSimilarAlso: EPO, epoetin, erythropoietin, erythropoiesis-stimulating agent, ESA, haematopoietin, haemopoietinEpoetin alfa and its biosimilars are first-generation erythropoiesis-stimulating agents (ESAs), which are recombinant versions of erythropoietin (EPO) produced using recombinant DNA technology. Erythropoietin (EPO) is a glycoprotein hormone, naturally produced mainly in the kidneys in response to hypoxia and stimulates red blood cell production (erythropoiesis) in the bone marrow.	Drug	Erythropoietin

Primary outcome

Percentage of Participants With Hemoglobin (Hb) Response
Time frame: Baseline to 12 weeks
Hb response is defined as a Hb increase of ≥ 1.0 g/dL at 12 weeks compared to baseline

1 secondary outcome

Change in Number of Blood Transfusions Per Year
Time frame: Baseline to 12 weeks

Results and safety

Results posted to the registry on 7 Jan 2026, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Erythropoietin	17	16

Primary outcome: Percentage of Participants With Hemoglobin (Hb) Response

Time frame: Baseline to 12 weeks. Count of participants. Unit: Participants

Measure	Erythropoietin n = 16
Value	15

Population analysed: All enrolled participants who have completed baseline and end of treatment testing, regardless of duration of treatment with EPO, will be included in the analysis.

1 secondary outcome is reported in the full results.

Adverse events

Time frame: From study drug initiation to the end of the treatment period (up to 12 weeks). Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Erythropoietin	2 of 17 (11.8%)	15 of 17 (88.2%)	0 of 17

Limitations and caveats (from the sponsor): The sponsor provided the study drug (EPO) for the duration of the treatment period (12 weeks). However, the sponsor was not able to provide study drug for a longer study, and participants who wanted to continue on EPO treatment during the observation period had to pay out of pocket or get insurance coverage. Thus, most participants had to discontinue EPO after the treatment period. The long-term safety of EPO treatment in patients with sickle cell disease cannot be evaluated from this study.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 7 Jan 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: Yes. Data collected may be shared with secondary researchers within or outside the participating centers upon request. The shared data will be deidentified.

Related trials for Anemia, Sickle Cell

RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
Active, not recruitingObservational study
Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease
Sickle Cell Anemia, Sickle Cell Thalassemia
Enrolling by invitationPhase 4
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study
Pyruvate Kinase Deficiency, Anemia, Hemolytic
Active, not recruitingPhase 2
A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)
Sickle Cell Disease
RecruitingPhase 3
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
Sickle Cell Disease

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.

Julia Z Xu, MD, MScGH principal investigator

University of Pittsburgh

2 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Hydroxyurea and Epoetin Alfa-BioSimilar in people with Anemia, Sickle Cell and Sickle Cell Disease, run by Julia Xu at 2 sites in 2 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 6 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: finished, as of the sponsor's last update on 7 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States, Nigeria. 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

Yes. Results are posted in the registry (first posted 7 Jan 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Percentage of Participants With Hemoglobin (Hb) Response (time frame: Baseline to 12 weeks). 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 NCT05451940.

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 7 Jan 2026.