Hypoxic Red Blood Cells in Sickle Cell Anemia

Not Applicable

Not yet recruiting

• Conditions: Sickle Cell Anaemia; Sickle Cell Anemia Crisis; Sickle Cell Anemia in Children; Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)

• Registration Number: NCT06743113
• Lead Sponsor: Hemanext

Brief Summary

The overall objective of this study is to evaluate the effectiveness and safety of transfusing hypoxic red blood cells manufactured with the Hemanext ONE system in patients with sickle cell anemia. The Hemanext ONE device was cleared through the De Novo process in September 2023.

Detailed Description

In this Direct-to-Phase II study, Hemanext Inc. will carry out a prospective, multi-center, single-blind, randomized, cross-over study in patients with Sickle Cell Anemia, comparing the efficacy of transfusion of hypoxic red blood cells (HRBCs) to transfusions with conventional RBCs. The primary efficacy objective is to demonstrate an increase in %HbA between red cell exchange transfusions (RCE) of HRBCs compared to conventional RBCs. The increases in %HbA (normal Hb) from RCE will be accompanied by a concomitant decrease in sickle Hb (%HbS). The persistence of %HbA will allow for a decrease in the volume of RBCs transfused with an overall decrease in the number of units consumed, which in turn can result in an increase in time (number of days) between transfusions.

Study Timeline

• Status Verified: 2024-12
• Study First Submitted: 2024-12-12
• Study First Submitted QC: 2024-12-16
• Last Update Submitted: 2024-12-16
• Study First Posted: 2024-12-19
• Last Update Posted: 2024-12-19
• Study Start: 2026-01
• Primary Completion: 2028-02
• Study Completion: 2028-06

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 48

Inclusion Criteria

1. Male or female at least 7 years of age;
2. Are able to provide informed consent, and assent as applicable, to participate in the study;
3. Diagnosis of Sickle Cell Anemia (SCA) (HbSS, HbSβ0 thalassemia) with participation in a chronic transfusion program and have undergone regular transfusions during at least 6 months prior to Screening;
4. Have had an average interval of at least 14 days between RBC transfusions over the past 6 months;
5. If on iron chelation therapy, have been on a stable dose for ≥3 months prior to screening;

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Exclusion Criteria

1. Are not exclusively transfused at the site;
2. Have a diagnosis of HbSC disease, HbSβ+ thalassemia or another SCD variant (excluding HbSS and HbSβ0 thalassemia)
3. Are routinely transfused with washed, packed RBC units;
4. Have received hemoglobin inducers (e.g. erythropoietin) in the 30 days prior to Screening;
5. Are currently being evaluated for gene therapy;
6. Have any clinically significant pulmonary, cardiovascular, endocrine, hepatic, gastrointestinal, renal, infectious, immunological (including significant allo- or auto-immunization) disease, considered not adequately controlled prior to the study;
7. Are a female of child-bearing potential who is pregnant or planning to become pregnant in the next 14 months;
8. Have a history of allo-immunization that cannot be managed by the local blood bank;
9. Patients who, in the opinion of the Investigator, would not be able or willing to comply with the protocol;
10. Is a ward of the state, prisoner, or transient

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Primary Outcome Measures

Name	Time	Method
%HbA Rate of Decline	Through study completion, an average of 14 months	The primary objective is to evaluate the decreased rate of decline of %HbA between post-transfusion RCE and the subsequent pre-transfusion RCE over 6 transfusion cycles in the hypoxic RBC group compared to the conventional group.

Secondary Outcome Measures

Name	Time	Method
Red Cell Exchange events	Through study completion, an average of 14 months	Mean number of RCE events over the course of the study
Volume of blood transfused	Through study completion, an average of 14 months	The mean volume of blood per patient transfused with hypoxic RBCs and with standard RBC units will be analyzed and compared
HgbS Rate of Increase	Through study completion, an average of 14 months	Average rate of increase of the HgbS measurement between automated red cell exchange (RCE) of hypoxic RBCs compared to conventional RBCs.
Incidence rate of vaso-occlusive crisis.	Through study completion, an average of 14 months	Incidence rate of vaso-occlusive crisis events through the duration of the study
Incidence rate of acute chest syndrome	Through study completion, an average of 14 months	Incidence rate of acute chest syndrome events accompanied by fever and/or respiratory symptoms through the duration of the study
Duration (days) of any hospitalization for vaso-occlusive crisis	Through study completion, an average of 14 months	Mean duration (days) of any hospitalization for vaso-occlusive crisis
Intravascular hemolysis	Through study completion, an average of 14 months	Level of intravascular hemolysis (measured with free plasma hemoglobin) between each procedure, before and after each Red Cell Exchange.
Serum ferritin	Through study completion, an average of 14 months	Mean change from baseline in serum ferritin
Changes in hepatic iron content	Through study completion, an average of 14 months	Mean changes in hepatic iron content
Change in QoL	Through study completion, an average of 14 months	Mean change in QoL, as measured by validated QoL questionnaires
Total hemoglobin before and after RCE	Through study completion, an average of 14 months	Mean change before and after transfusions of hypoxically stored RBCs compared to that with conventionally stored RBCs.
Total hematocrit before and after RCE	Through study completion, an average of 14 months	Mean change before and after transfusions of hypoxically stored RBCs compared to that with conventionally stored RBCs.
Safety assessment	Through study completion, an average of 14 months	Frequency of adverse event reactions and device deficiencies over the course of the study

Trial Locations

Locations (6)

New England Sickle Cell Institute, University of Connecticut; 🇺🇸 Farmington, Connecticut, United States

Johns Hopkins All Children's Hospital; 🇺🇸 Saint Petersburg, Florida, United States

Emory University School of Medicine; 🇺🇸 Atlanta, Georgia, United States

John Hopkins University School of Medicine; 🇺🇸 Baltimore, Maryland, United States

University of Pittsburgh; 🇺🇸 Pittsburgh, Pennsylvania, United States

University of Pittsburgh Medical Center; 🇺🇸 Pittsburgh, Pennsylvania, United States