Hypoxic Red Blood Cells in Sickle Cell Anemia

NCT06743113 | Not Yet Recruiting DMC FDA Device

• 1 other identifier: PRO-CLIN-0017
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 6

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.

Conditions

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

Outcome Measures

Primary Outcomes (1)

• %HbA Rate of Decline

  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.

  Through study completion, an average of 14 months

Secondary Outcomes (13)

• Volume of blood transfused

  Through study completion, an average of 14 months

• HgbS Rate of Increase

  Through study completion, an average of 14 months

• Incidence rate of vaso-occlusive crisis.

  Through study completion, an average of 14 months

• Incidence rate of acute chest syndrome

  Through study completion, an average of 14 months

• Duration (days) of any hospitalization for vaso-occlusive crisis

  Through study completion, an average of 14 months

Other Outcomes (1)

• Hemoglobin increment from each transfusion

  Through study completion, an average of 14 months

Study Arms (2)

Treatment A (Hypoxic RBCs)

EXPERIMENTAL

Transfusion of hypoxic red blood cells manufactured with Hemanext ONE system

Device: Hemanext ONE System

Treatment B (Conventional RBCs)

ACTIVE COMPARATOR

Transfusion of conventional red blood cells

Device: Conventional RBCs

Interventions

Hemanext ONE System DEVICE

Hypoxic red blood cells

Treatment A (Hypoxic RBCs)

Conventional RBCs DEVICE

Conventional red blood cells

Treatment B (Conventional RBCs)

Eligibility Criteria

• Age: 7 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;

You may not qualify if:

• 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

Sponsors & Collaborators

• Hemanext: lead
• Emory University: collaborator
• University of Connecticut: collaborator
• University of Pittsburgh Medical Center: collaborator
• Johns Hopkins All Children's Hospital: collaborator
• Johns Hopkins University: collaborator

Study Sites (6)

New England Sickle Cell Institute, University of Connecticut

Farmington, Connecticut, 06030, United States

Location

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States

Location

Emory University School of Medicine

Atlanta, Georgia, 30322, United States

Location

John Hopkins University School of Medicine

Baltimore, Maryland, 21287, United States

Location

University of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15260, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Vaso-Occlusive Crises

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Enrico Novelli, MD, MS

  University of Pittsburgh Medical Center

  PRINCIPAL INVESTIGATOR

• Biree Andemariam, MD

  New England Sickle Cell Institute, University of Connecticut

  PRINCIPAL INVESTIGATOR

• Laurel Omert, MD, FACS

  Hemanext Inc.

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jill Bagdasarian

CONTACT

(781) 301-7474; jill.bagdasarian@hemanext.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: December 12, 2024
• First Posted: December 19, 2024
• Study Start: March 1, 2026
• Primary Completion (Estimated): April 1, 2028
• Study Completion (Estimated): September 1, 2028
• Last Updated: January 7, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)