Clinical trial NCT06743113: Hypoxic Red Blood Cells in Sickle Cell Anemia
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
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: NA
Conditions: Sickle Cell Anaemia
Sickle Cell Anemia Crisis
Sickle Cell Anemia in Children
Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Lead sponsor: Hemanext
Sponsor class: INDUSTRY
Collaborators: name: Emory University
name: University of Connecticut
name: University of Pittsburgh Medical Center
name: Johns Hopkins All Children's Hospital
name: Johns Hopkins University
First posted: 2024-12-19
Enrollment: 48
Sex: ALL
Minimum age (years): 7
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Intervention model description: Each enrolled patient will be randomized to treatment with either hypoxic RBCs or conventional RBCs after successful completion of the screening period. After 6 months in the randomized treatment arm, two "Wash Out" transfusions of conventional RBCs will be scheduled. The patient will then "cross-over" into the other treatment arm to complete another 6 months of treatment.
Primary purpose: TREATMENT

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.

Eligibility criteria: 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;

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

Primary outcomes:
measure: %HbA Rate of Decline
timeFrame: Through study completion, an average of 14 months
Secondary outcomes:
measure: Volume of blood transfused
timeFrame: Through study completion, an average of 14 months
measure: HgbS Rate of Increase
timeFrame: Through study completion, an average of 14 months
measure: Incidence rate of vaso-occlusive crisis.
timeFrame: Through study completion, an average of 14 months
measure: Incidence rate of acute chest syndrome
timeFrame: Through study completion, an average of 14 months
measure: Duration (days) of any hospitalization for vaso-occlusive crisis
timeFrame: Through study completion, an average of 14 months
measure: Intravascular hemolysis
timeFrame: Through study completion, an average of 14 months
measure: Serum ferritin
timeFrame: Through study completion, an average of 14 months
measure: Changes in hepatic iron content
timeFrame: Through study completion, an average of 14 months
measure: Change in QoL
timeFrame: Through study completion, an average of 14 months
measure: Total hemoglobin before and after RCE
timeFrame: Through study completion, an average of 14 months
measure: Total hematocrit before and after RCE
timeFrame: Through study completion, an average of 14 months
measure: Red Cell Exchange events
timeFrame: Through study completion, an average of 14 months
measure: Safety assessment
timeFrame: Through study completion, an average of 14 months
Other outcomes:
measure: Hemoglobin increment from each transfusion
timeFrame: Through study completion, an average of 14 months
Locations:
city: Farmington
country: United States
facility: New England Sickle Cell Institute, University of Connecticut
state: Connecticut
city: Saint Petersburg
country: United States
facility: Johns Hopkins All Children's Hospital
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Baltimore
country: United States
facility: John Hopkins University School of Medicine
state: Maryland
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
Study officials:
affiliation: University of Pittsburgh Medical Center
name: Enrico Novelli, MD, MS
role: PRINCIPAL_INVESTIGATOR
affiliation: New England Sickle Cell Institute, University of Connecticut
name: Biree Andemariam, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Hemanext Inc.
name: Laurel Omert, MD, FACS
role: PRINCIPAL_INVESTIGATOR
References: