Sickle Cell Anaemia, Sickle Cell Anemia Crisis, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Sickle Cell Anemia in Children
Conditions
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.
Interventions
DEVICEHemanext ONE System
Hypoxic red blood cells
DEVICEConventional RBCs
Conventional red blood cells
Sponsors
Hemanext
Emory University
University of Connecticut
University of Pittsburgh Medical Center
Johns Hopkins University
University of Illinois at Chicago
National Heart, Lung, and Blood Institute (NHLBI)
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
SINGLE (Subject)
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.
Eligibility
Sex/Gender
ALL
Age
7 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| %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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| Red Cell Exchange events | Through study completion, an average of 14 months | Mean number of RCE events over the course of the study |
| Safety assessment | Through study completion, an average of 14 months | Frequency of adverse event reactions and device deficiencies over the course of the study |
Countries
United States
Contacts
CONTACTJill Bagdasarian
PRINCIPAL_INVESTIGATORBiree Andemariam, MD
New England Sickle Cell Institute, University of Connecticut
PRINCIPAL_INVESTIGATORLaurel Omert, MD, FACS
Hemanext Inc.
Outcome results
None listed