A Phase 1 Safety Study of SANGUINATE™ In Patients With Acute Severe Anemia.

Study Overview
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Brief Summary
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In this open-label study, eligible and consenting adult patients admitted to the hospital for critical care that have acute severe anemia, defined as a blood hemoglobin level ≤ 5 g/dL, but who are unwilling or unable to receive red blood cell (RBC) transfusion, will receive one or more infusions of SANGUINATE as are deemed necessary by the Investigator for survival of the acute anemic episode.

Detailed Description
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Data collection for each patient during the in-patient portion of the study will be required for up to a maximum of 14 days while under critical care, with additional collection of safety-related findings at 7 days and 14 days after the last infusion of SANGUINATE. Each patient will thus have up to 28 days participation in the trial. A minimum of 100 subjects are to be enrolled and to receive at least one infusion of SANGUINATE, with 14-day follow-up after the last infusion. Because of the life-threatening effects of severe anemia, it is expected that all patients in this trial will receive care within a critical care facility (may be called intensive care or some similar name), wherein all patients will have real-time monitoring of vital signs and ECG (e.g., by telemetry) before, during, and after all study drug infusions.

Official Title
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An Open-label Phase 1 Safety Study of SANGUINATE™ Infusion in Patients With Acute Severe Anemia Who Are Unable to Receive Red Blood Cell Transfusion

Conditions
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Acute Anemia

Intervention / Treatment
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* Drug: SANGUINATE™


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: The patient would otherwise receive RBC transfusion for treatment of severe anemia but cannot (hemolytic/allosensitized or no compatible blood) or will not (religious/personal objection) receive RBCs Hemoglobin ≤ 5 g/dL, or hemoglobin ≤ 7 g/dL following a decline of ≥ 5 g/dL over less than 7 days Age ≥ 18 years Receiving or willing to receive supplemental iron therapy (unless contraindicated) Receiving or willing to receive erythropoiesis-stimulating agent (EPO) therapy Patient or legally authorized representative provided consent to participate Investigator determination that the patient is an appropriate candidate for study enrollment Exclusion Criteria: Hemoglobin ≤ 2 g/dL Presence of severe trauma (e.g., Injury Severity Score (ISS)/New Injury Severity Score (NISS) Score ≥ 25) Unable to provide sufficient blood sample volume for screening assessments; or Pregnant; or Investigator determination that the patient is not an appropriate candidate for study enrollment

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: SANGUINATE™<br>As Needed Dosing of SANGUINATE | Drug: SANGUINATE™<br>* As needed (PRN) infusions of 500 mL of SANGUINATE<br>* Other names: pegylated carboxyhemoglobin bovine;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number and severity of adverse events within 24-hours of each SANGUINATE infusion | Composite endpoint defined by changes in vital signs, electrocardiographic, biochemical, hematological, and urinalysis, death and other reported adverse events | 24 Hours | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of patients who survive the acute episode of severe anemia |  | 14 Days | 
| Percentage of patients who recover from hypoxia-induced abnormal end-organ function |  | 14 Days | 
| Percentage of patients who improved with their hemodynamic status |  | 14 Days | 
| Percentage of patients who discontinue inotrope therapy |  | 14 Days | 
| Percentage of patients who discontinue respiratory support/therapy |  | 14 Days | 
| Number of serious adverse events following the start of SANGUINATE infusions through the final visit (up to 28 days) | Composite endpoint defined by changes in vital signs, electrocardiographic, biochemical, hematological, and urinalysis, death and other reported serious adverse events | 28 Days |