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

A Phase II, Multicenter, Single-Blind, Randomized Study of the Safety and Effectiveness of SANGUINATE™ Versus Normal Saline in Adult Sickle Cell Disease Patients With Vaso-Occlusive Crisis (VOC)

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

Study Overview

• Status: Completed
• Conditions: Anemia, Sickle Cell
• Intervention / Treatment: Drug: SANGUINATE 320 mg/kg; Drug: Normal Saline

Detailed Description

A single-blind, multicenter, randomized, placebo-control study in which 30 Sickle Cell disease patients having a vaso-occlusive crisis will either receive SANGUINATE 320 mg/kg/patient (8 mL/kg/patient) or Normal Saline on Day 1 (Visit 1) and Day 2 (Visit 2) infused over 2 hours each day. Patients are to remain in the hospital for up to 7 days but can be discharged at any time after receiving the second dose of SANGUINATE, provided their vaso-occlusive crisis has resolved and the patient has completed discharge procedures. Patients will have a follow-up phone call 7 days after discharge from the hospital to obtain safety, concomitant medication and pain assessments.

• Study Type: Interventional
• Enrollment (Actual): 34
• Phase: Phase 2

Contacts and Locations

Study Locations

• Colombia
  • Bogota, Colombia
• Dominican Republic
  • Santo Domingo, Dominican Republic
• Honduras
  • San Pedro Sula, Honduras
• Panama
  • Panama City, Panama

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Age ≥ 18 and ≤ 65 years of age
2. Diagnosis of Sickle Cell Disease (Hb-SS or any Genotype)
3. Pain-score due to vaso-occlusive pain crisis (VOC) ≥ 8 on a 10 point scale
4. VOC pain location ≥ 1 sites typical of vaso-occlusive crisis
5. Patients with Priapism, acute chest syndrome, and/or with other Sickle Cell Disease comorbidities can be enrolled with good judgment of the Investigator.
6. Signed and dated informed written consent by the subject
7. Able to receive intravenous infusion of SANGUINATE or Normal Saline
8. 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

Exclusion Criteria:

1. In the judgment of the investigator, the patient is not a good candidate for the study
2. Females who are lactating and/or breastfeeding
3. Fewer than 14 days since prior infusion pain medication treatment for VOC
4. 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)
5. Concurrent or prior treatment within 30 days of Screening with an investigational medication.
6. 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;
7. 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;
8. Evidence or history of regular alcohol abuse
9. Screening laboratory result indicating serologic positivity for hepatitis C antibodies or hepatitis B surface antigens, unless explained by a documented vaccination.
10. Unable to comply with study attendance, protocol procedures or other study requirements;
11. Abnormal Echocardiogram at Study Entry (defined as Tricuspid Regurgitant Jet Velocity >3.1 m/sec).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: SANGUINATE 320 mg/kg; Two-hour infusion of SANGUINATE on Day 1 and Day 2	Drug: SANGUINATE 320 mg/kg; Two-hour infusion of SANGUINATE on Day 1 and Day 2; Other Names: pegylated carboxyhemoglobin bovine
Placebo Comparator: Normal Saline; Two-hour infusion of Normal Saline and Day 1 and Day 2	Drug: Normal Saline; Two-hour infusion of Normal Saline and Day 1 and Day 2

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	Composite endpoint with multiple vital signs, ECGs, echocardiograms, clinical assessments and bio-analytical lab measurements over the 7 day time frame	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
Percent reduction in pain score utilizing a visual analog scale following treatment with SANGUINATE and Normal Saline.		Up to 7 Days
Reduction in the level of C-Reactive Protein following treatment of SANGUINATE versus Normal Saline.		Up to 7 Days

Collaborators and Investigators

• Sponsor: Prolong Pharmaceuticals
• Investigators: Study Director: Hemant Misra, PhD, Prolong Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): July 1, 2016
• Primary Completion (Actual): May 31, 2017
• Study Completion (Actual): May 31, 2017

Study Registration Dates

• First Submitted: February 1, 2016
• First Submitted That Met QC Criteria: February 1, 2016
• First Posted (Estimate): February 3, 2016

Study Record Updates

• Last Update Posted (Actual): May 23, 2018
• Last Update Submitted That Met QC Criteria: May 22, 2018
• Last Verified: May 1, 2018

More Information

Terms related to this study

• Keywords: Anemia; VOC; Sickle Cell; Vaso occlusive crisis
• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: SGSC-002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes