Hypoxic Red Blood Cells in Sickle Cell Anemia

Официальное название

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

Обзор

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.

Подробное описание

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.

Вмешательства

• Устройство Hemanext ONE System
  Hypoxic red blood cells
• Устройство Conventional RBCs
  Conventional red blood cells

Первичные конечные точки

• %HbA Rate of Decline [Срок оценки: Through study completion, an average of 14 months]

Вторичные конечные точки (12)

• Volume of blood transfused [Срок оценки: Through study completion, an average of 14 months]
• HgbS Rate of Increase [Срок оценки: Through study completion, an average of 14 months]
• Incidence rate of vaso-occlusive crisis. [Срок оценки: Through study completion, an average of 14 months]
• Incidence rate of acute chest syndrome [Срок оценки: Through study completion, an average of 14 months]
• Duration (days) of any hospitalization for vaso-occlusive crisis [Срок оценки: Through study completion, an average of 14 months]
• Intravascular hemolysis [Срок оценки: Through study completion, an average of 14 months]
• Serum ferritin [Срок оценки: Through study completion, an average of 14 months]
• Changes in hepatic iron content [Срок оценки: Through study completion, an average of 14 months]
• Change in QoL [Срок оценки: Through study completion, an average of 14 months]
• Total hemoglobin before and after RCE [Срок оценки: Through study completion, an average of 14 months]
• Total hematocrit before and after RCE [Срок оценки: Through study completion, an average of 14 months]
• Red Cell Exchange events [Срок оценки: Through study completion, an average of 14 months]

Критерии участия

Критерии включения

• 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;

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Перекрёстный дизайн

Маскирование

Простое слепое

Основная цель

Лечение

Центры проведения

США · 6 центров

• 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

Идентификаторы

NCT: NCT06743113 · PRO-CLIN-0017

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗