A Study of SANGUINATE for the Treatment of Vaso-occlusive Crisis (VOC) in Adult Sickle Cell Disease Patients

NCT02672540 | Completed Phase 2

• 1 other identifier: SGSC-002
• Study Type: interventional
• Target: 34
• Locations: 4 countries
• Sites: 4

Brief Summary

Safety and effect of SANGUINATE on Sickle Cell Disease patients experiencing a vaso-occlusive crisis who are admitted to the hospital for treatment.

Conditions

Anemia, Sickle Cell

Keywords

Sickle Cell; Anemia; VOC; Vaso occlusive crisis

Outcome Measures

Primary Outcomes (1)

• Time to readiness for discharge from in hospital stay following treatment with SANGUINATE and Normal Saline.

  Defined as the patient's response that their pain episode has improved enough for discharge from the hospital, or the Investigator's assessment that the patient is ready for discharge from the hospital.

  Up to 7 Days

Secondary Outcomes (7)

• Safety of treatment as defined by changes in vital signs, electrocardiographic, biochemical, hematological, and urinalysis measures, as well as reported increases in pain and other reported adverse events

  Up to 7 Days

• Proportion of patients who develop acute chest syndrome (ACS) during the study.

  Up to 14 Days

• Proportion of patients who are re-hospitalized for their vaso-occlusive crisis episode.

  Up to 7 Days

• Total length of stay (LOS) following treatment of SANGUINATE versus Normal Saline.

  Up to 7 Days

• Percent reduction in total pain medication required during in-hospital stay following treatment with SANGUINATE and Normal Saline.

  Up to 7 Days

Study Arms (2)

SANGUINATE 320 mg/kg

EXPERIMENTAL

Two-hour infusion of SANGUINATE on Day 1 and Day 2

Drug: SANGUINATE 320 mg/kg

Normal Saline

PLACEBO COMPARATOR

Two-hour infusion of Normal Saline and Day 1 and Day 2

Drug: Normal Saline

Interventions

SANGUINATE 320 mg/kg DRUG

Two-hour infusion of SANGUINATE on Day 1 and Day 2

Also known as: pegylated carboxyhemoglobin bovine

SANGUINATE 320 mg/kg

Normal Saline DRUG

Two-hour infusion of Normal Saline and Day 1 and Day 2

Normal Saline

Eligibility Criteria

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

You may qualify if:

• Age ≥ 18 and ≤ 65 years of age
• Diagnosis of Sickle Cell Disease (Hb-SS or any Genotype)
• Pain-score due to vaso-occlusive pain crisis (VOC) ≥ 8 on a 10 point scale
• VOC pain location ≥ 1 sites typical of vaso-occlusive crisis
• Patients with Priapism, acute chest syndrome, and/or with other Sickle Cell Disease comorbidities can be enrolled with good judgment of the Investigator.
• Signed and dated informed written consent by the subject
• Able to receive intravenous infusion of SANGUINATE or Normal Saline
• Women of childbearing potential with a negative serum pregnancy test and using a reliable method of contraception during the study period and for 30 days thereafter. Male study participants also agree to use contraception for 30 days after the study period

You may not qualify if:

• In the judgment of the investigator, the patient is not a good candidate for the study
• Females who are lactating and/or breastfeeding
• Fewer than 14 days since prior infusion pain medication treatment for VOC
• Medical history or evidence of moderate to severe renal insufficiency (estimated GFR \< 60 mL/min) or chronic kidney disease, or of moderate to severe hepatic disease (ALTs \> 5 x ULN)
• Concurrent or prior treatment within 30 days of Screening with an investigational medication.
• Symptoms or electrocardiogram (ECG)-based signs of acute myocardial infarction, unstable angina pectoris, decompensated heart failure, third degree heart block or cardiac arrhythmia associated with hemodynamic instability;
• Severe or unstable concomitant condition or disease (e.g., known significant neurologic deficit, cancer, hematologic, metabolic or coronary disease), or chronic condition (e.g., psychiatric disorder), that, in the opinion of the Investigator, may increase the risk associated with study participation or study drug administration, or may interfere with the interpretation of study results;
• Evidence or history of regular alcohol abuse
• Screening laboratory result indicating serologic positivity for hepatitis C antibodies or hepatitis B surface antigens, unless explained by a documented vaccination.
• Unable to comply with study attendance, protocol procedures or other study requirements;
• Abnormal Echocardiogram at Study Entry (defined as Tricuspid Regurgitant Jet Velocity \>3.1 m/sec).

Sponsors & Collaborators

• Prolong Pharmaceuticals: lead

Study Sites (4)

Unknown Facility

Bogotá, Colombia

Location

Unknown Facility

Santo Domingo, Dominican Republic

Location

Unknown Facility

San Pedro Sula, Honduras

Location

Unknown Facility

Panama City, Panama

Location

MeSH Terms

Conditions

Anemia, Sickle Cell; Anemia; Vaso-Occlusive Crises

Interventions

PEGylated carboxyhemoglobin bovine; Saline Solution

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Study Officials

• Hemant Misra, PhD

  Prolong Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 1, 2016
• First Posted: February 3, 2016
• Study Start: July 1, 2016
• Primary Completion: May 31, 2017
• Study Completion: May 31, 2017
• Last Updated: May 23, 2018

Record last verified: 2018-05

Data Sharing

• IPD Sharing: Will not share

Locations

DO (1); CO (1); HO (1); PA (1)