Clinical trial NCT02754999: A Phase 1 Safety Study of SANGUINATE™ In Patients With Acute Severe Anemia.
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Acute Anemia
Lead sponsor: Prolong Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2016-04-28
Enrollment: 103
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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: 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.

Eligibility criteria: Inclusion Criteria:

1. 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
2. Hemoglobin ≤ 5 g/dL, or hemoglobin ≤ 7 g/dL following a decline of ≥ 5 g/dL over less than 7 days
3. Age ≥ 18 years
4. Receiving or willing to receive supplemental iron therapy (unless contraindicated)
5. Receiving or willing to receive erythropoiesis-stimulating agent (EPO) therapy
6. Patient or legally authorized representative provided consent to participate
7. Investigator determination that the patient is an appropriate candidate for study enrollment

Exclusion Criteria:

1. Hemoglobin ≤ 2 g/dL
2. Presence of severe trauma (e.g., Injury Severity Score (ISS)/New Injury Severity Score (NISS) Score ≥ 25)
3. Unable to provide sufficient blood sample volume for screening assessments; or
4. Pregnant; or
5. Investigator determination that the patient is not an appropriate candidate for study enrollment

Primary outcomes:
measure: Number and severity of adverse events within 24-hours of each SANGUINATE infusion
timeFrame: 24 Hours
Secondary outcomes:
measure: Percentage of patients who survive the acute episode of severe anemia
timeFrame: 14 Days
measure: Percentage of patients who recover from hypoxia-induced abnormal end-organ function
timeFrame: 14 Days
measure: Percentage of patients who improved with their hemodynamic status
timeFrame: 14 Days
measure: Percentage of patients who discontinue inotrope therapy
timeFrame: 14 Days
measure: Percentage of patients who discontinue respiratory support/therapy
timeFrame: 14 Days
measure: Number of serious adverse events following the start of SANGUINATE infusions through the final visit (up to 28 days)
timeFrame: 28 Days
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Brookwood Princeton Baptist
state: Alabama
city: Prescott
country: United States
facility: Yavapai Regional Medical Center
state: Arizona
city: Baldwin Park
country: United States
facility: Kaiser Permanente
state: California
city: Duarte
country: United States
facility: City of Hope
state: California
city: Lancaster
country: United States
facility: Antelope Valley Hospital
state: California
city: Loma Linda
country: United States
facility: Loma Linda Medical Center
state: California
city: Mission Hills
country: United States
facility: Providence Holy Cross Hospital
state: California
city: Washington, D.C.
country: United States
facility: Medstar Georgetown University Hosiptal
state: District of Columbia
city: Tampa
country: United States
facility: Tampa General Hospital
state: Florida
city: Augusta
country: United States
facility: Augusta University
state: Georgia
city: Marietta
country: United States
facility: Wellstar Atlanta Medical Centre
state: Georgia
city: Baltimore
country: United States
facility: Johns Hopkins University
state: Maryland
city: Detroit
country: United States
facility: Detroit Medical Center Harper Univesity Hospital
state: Michigan
city: Englewood
country: United States
facility: Englewood Medical Center
state: New Jersey
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: Livingston
country: United States
facility: St. Barnabas
state: New Jersey
city: Lake Success
country: United States
facility: Northwell - North Shore University Hospital
state: New York
city: New York
country: United States
facility: Mt. Sinai /Recanati/Miller Transplantation Institute, Icahn
state: New York
city: Charlotte
country: United States
facility: Presbyterian Hospital Novant Health
state: North Carolina
city: Abington
country: United States
facility: Abington University
state: Pennsylvania
city: Philadelphia
country: United States
facility: Hahnemann University Hospital (Rittenhouse)
state: Pennsylvania
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Philadelphia
country: United States
facility: Thomas Jefferson University
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Greenville
country: United States
facility: Bon Secour St Francis Cancer Center
state: South Carolina
city: El Paso
country: United States
facility: University of Texas Medical Center (El Paso)
state: Texas
city: Houston
country: United States
facility: Houston Methodist Hospital
state: Texas
city: Houston
country: United States
facility: St. Lukes
state: Texas
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Seattle
country: United States
facility: Swedish Medical Center
state: Washington
city: Milwaukee
country: United States
facility: Blood Center of Wisconsin
state: Wisconsin
Study officials:
affiliation: Prolong Pharamceuticals
name: Abe Abuchowski, PhD
role: STUDY_DIRECTOR
References: