Hypoxic Red Blood Cells in Sickle Cell Anemia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Hemanext ONE System, Conventional RBCs.
- Who it may be relevant to
- Registry conditions: Sickle Cell Anaemia, Sickle Cell Anemia Crisis, Sickle Cell Anemia in Children, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0). Basic parameters: from 7 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Multi-Center, Randomized, Controlled, Cross-Over Study to Evaluate the Effectiveness of Hypoxic Red Blood Cells Processed With the Hemanext ONE® System Versus Conventional Red Blood Cells in Patients With Transfusion Dependent Sickle Cell Anemia
Overview
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.
Interventions
- Device Hemanext ONE System
Hypoxic red blood cells - Device Conventional RBCs
Conventional red blood cells
Primary outcome measures
- %HbA Rate of Decline [Time frame: Through study completion, an average of 14 months]
Secondary outcome measures (12)
- Volume of blood transfused [Time frame: Through study completion, an average of 14 months]
- HgbS Rate of Increase [Time frame: Through study completion, an average of 14 months]
- Incidence rate of vaso-occlusive crisis. [Time frame: Through study completion, an average of 14 months]
- Incidence rate of acute chest syndrome [Time frame: Through study completion, an average of 14 months]
- Duration (days) of any hospitalization for vaso-occlusive crisis [Time frame: Through study completion, an average of 14 months]
- Intravascular hemolysis [Time frame: Through study completion, an average of 14 months]
- Serum ferritin [Time frame: Through study completion, an average of 14 months]
- Changes in hepatic iron content [Time frame: Through study completion, an average of 14 months]
- Change in QoL [Time frame: Through study completion, an average of 14 months]
- Total hemoglobin before and after RCE [Time frame: Through study completion, an average of 14 months]
- Total hematocrit before and after RCE [Time frame: Through study completion, an average of 14 months]
- Red Cell Exchange events [Time frame: Through study completion, an average of 14 months]
Eligibility criteria
Inclusion criteria
- Male or female at least 7 years of age;
- Are able to provide informed consent, and assent as applicable, to participate in the study;
- 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;
- Have had an average interval of at least 14 days between RBC transfusions over the past 6 months;
- If on iron chelation therapy, have been on a stable dose for ≥3 months prior to screening;
Exclusion criteria
- Are not exclusively transfused at the site;
- Have a diagnosis of HbSC disease, HbSβ+ thalassemia or another SCD variant (excluding HbSS and HbSβ0 thalassemia)
- Are routinely transfused with washed, packed RBC units;
- Have received hemoglobin inducers (e.g. erythropoietin) in the 30 days prior to Screening;
- Are currently being evaluated for gene therapy;
- 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;
- Are a female of child-bearing potential who is pregnant or planning to become pregnant in the next 14 months;
- Have a history of allo-immunization that cannot be managed by the local blood bank;
- Patients who, in the opinion of the Investigator, would not be able or willing to comply with the protocol;
- Is a ward of the state, prisoner, or transient
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Crossover
- Masking
- Single blind
- Primary purpose
- Treatment
Study locations
United States · 6 centers
- New England Sickle Cell Institute, University of Connecticut — Farmington
- Johns Hopkins All Children's Hospital — St. Petersburg
- Emory University School of Medicine — Atlanta
- John Hopkins University School of Medicine — Baltimore
- University of Pittsburgh — Pittsburgh
- University of Pittsburgh Medical Center — Pittsburgh
Identifiers
NCT: NCT06743113 · PRO-CLIN-0017