Hydroxyurea and EPO in Sickle Cell Disease

• Status: Not yet recruiting
• Trial end date: 2024-08-01
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Julia Xu
• Collaborators: American Society of Hematology; Carnegie Mellon University
• Treatments: Epoetin Alfa; Hematinics; Hydroxyurea

Criteria

Inclusion Criteria:

• 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 ≥ 100 ng/mL

• Must be on stable-dose hydroxyurea treatment (i.e., no changes in dose within 60 days prior to screening) 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

Exclusion Criteria:

• Hemoglobin SC (HbSC), S/β+-thalassemia, or other compound heterozygous SCD genotypes

• 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 screening

• Received voxelotor or EPO within 60 days of screening

• Untreated iron deficiency, or had initiation or change in dose of supplemental iron within 30 days of screening

• Ongoing acute illness, infection, or VOC within 2 weeks of screening

• Arterial or venous thrombosis within 180 days of screening

• 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

• 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 the screening visit or plans to participate in another investigational drug trial