NCT05451940

Hydroxyurea and EPO in Sickle Cell Disease

Assessing Combination Hydroxyurea and Exogenous Erythropoietin in Sickle Cell Disease

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: Julia Xu · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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).

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

Interventions

Type	Name	Description
DRUG	Hydroxyurea	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	Epoetin Alfa-BioSimilar	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.

Timeline

• Start date: 2023-05-25
• Primary completion: 2024-12-13
• Completion: 2025-02-27
• First posted: 2022-07-11
• Last updated: 2026-01-07
• Results posted: 2026-01-07

Locations

2 sites across 2 countries: United States, Nigeria

Regulatory

• FDA-regulated drug study