Hypoxic Red Blood Cells in Sickle Cell Anemia

NCT ID: NCT06743113

Last Updated: 2024-12-19

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

NA

Total Enrollment

48 participants

Study Classification

INTERVENTIONAL

Study Start Date

2026-01-31

Study Completion Date

2028-06-30

Brief Summary

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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

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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.

Conditions

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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

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 Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Participants

Study Groups

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Treatment A (Hypoxic RBCs)

Transfusion of hypoxic red blood cells manufactured with Hemanext ONE system

Group Type EXPERIMENTAL

Hemanext ONE System

Intervention Type DEVICE

Hypoxic red blood cells

Treatment B (Conventional RBCs)

Transfusion of conventional red blood cells

Group Type ACTIVE_COMPARATOR

Conventional RBCs

Intervention Type DEVICE

Conventional red blood cells

Interventions

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Hemanext ONE System

Hypoxic red blood cells

Intervention Type DEVICE

Conventional RBCs

Conventional red blood cells

Intervention Type DEVICE

Eligibility Criteria

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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
Minimum Eligible Age

7 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Emory University

OTHER

Sponsor Role collaborator

University of Connecticut

OTHER

Sponsor Role collaborator

University of Pittsburgh Medical Center

OTHER

Sponsor Role collaborator

Johns Hopkins All Children's Hospital

OTHER

Sponsor Role collaborator

Johns Hopkins University

OTHER

Sponsor Role collaborator

Hemanext

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Enrico Novelli, MD, MS

Role: PRINCIPAL_INVESTIGATOR

University of Pittsburgh Medical Center

Biree Andemariam, MD

Role: PRINCIPAL_INVESTIGATOR

New England Sickle Cell Institute, University of Connecticut

Laurel Omert, MD, FACS

Role: PRINCIPAL_INVESTIGATOR

Hemanext Inc.

Locations

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New England Sickle Cell Institute, University of Connecticut

Farmington, Connecticut, United States

Site Status

Johns Hopkins All Children's Hospital

St. Petersburg, Florida, United States

Site Status

Emory University School of Medicine

Atlanta, Georgia, United States

Site Status

John Hopkins University School of Medicine

Baltimore, Maryland, United States

Site Status

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Site Status

Countries

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United States

Central Contacts

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Jill Bagdasarian

Role: CONTACT

Phone: (781) 301-7474

Email: [email protected]

Facility Contacts

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Biree Andemariam, MD

Role: primary

Cassandra Josephson, MD

Role: primary

Ross Fasano, MD

Role: primary

Elizabeth Crowe, MD, PhD

Role: primary

Alesia Kaplan, MD

Role: primary

Enrico Novelli, MD, MS

Role: primary

Other Identifiers

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PRO-CLIN-0017

Identifier Type: -

Identifier Source: org_study_id