Study of SANGUINATE™ In the Treatment of Sickle Cell Disease Patients With Vaso-Occlusive Crisis

A Phase 2 Study of the Safety and Effectiveness of SANGUINATE™ in the Treatment of Vaso-Occlusive Crises in the Ambulatory Setting: A Placebo-Controlled, Single-Dose, Single-Blind Study in Adults With Sickle Cell Disease

Safety and effect of SANGUINATE on Sickle Cell Disease patients experiencing a vaso-occlusive crisis

Study Overview

• Status: Completed
• Conditions: Anemia, Sickle Cell
• Intervention / Treatment: Drug: SANGUINATE; Drug: Placebo

Detailed Description

Patients who are experiencing a vaso-occlusive crisis will report to the clinic for treatment and screening into the SGSC-005 study. Patients who meet all inclusion/exclusion criteria will be randomized to receive either SANGUINATE 320 mg/kg or placebo (saline) over a 2 hour infusion period. Assessments of vital signs, ECGs, safety labs, adverse events as well as patient and physician questionnaires will be completed up to 6 hours after the start of the infusion. Patients will then be assessed for discharge either to home or admitted to the hospital for further treatment depending on their disease severity. Follow-up phone calls will be completed 24 hours and 7 days after initiation of treatment . A follow-up visit will be completed in the clinic at 72 hours after initiation of treatment.

• Study Type: Interventional
• Enrollment (Actual): 24
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Florida
    • Hollywood, Florida, United States, 33021
      • FSCDR
    • Tampa, Florida, United States, 33606
      • Florida Health Tampa General Hospital
  • Maryland
    • Baltimore, Maryland, United States, 21201-1559
      • University of Maryland School of Medicine
    • Baltimore, Maryland, United States, 21205
      • Johns Hopkins Univeristy School of Medicine
  • New Jersey
    • Newark, New Jersey, United States, 07112
      • Newark Beth Israel Medical Center
  • New York
    • Rochester, New York, United States, 14627-0140
      • University of Rochester Medical Center
  • Ohio
    • Columbus, Ohio, United States, 43203
      • Ohio State University Medical Center
  • Virginia
    • Richmond, Virginia, United States, 23298
      • Virginia Commonwealth University
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Blood Center of Wisconsin

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

1. Age ≥ 18 years,
2. Sickle Cell Disease (all genotypes),
3. Diagnosis of a severe vaso-occlusive crisis (VOC), based on the clinical judgement of the Investigator,
4. Participant needs to be admitted to the ambulatory site for treatment of VOC requiring IV pain medication,
5. Able to provide written consent,
6. Able to receive IV infusion of study drug.

Exclusion Criteria:

1. In the judgment of the Investigator, the participant is not a good candidate for the study,
2. An acute severe complication of SCD beyond VOC,
3. Pregnant or actively trying to become pregnant, or breastfeeding,
4. Participant had > 6 urgent visits for SCD complications in the prior 3 months,
5. Fewer than 30 days since any prior treatment with IV pain medication for VOC,
6. Onset of current acute painful crisis > 3 days prior to dosing,
7. Evidence of moderate to severe renal insufficiency (CrCl < 50 mL/min) or chronic kidney disease, or of moderate to severe hepatic disease (LFTs > 2 x ULN) based on past medical history,
8. Concurrent or prior treatment within 90 days with an investigational medication,
9. Abnormal ECG due to cardiac ischemia and/or atrial fibrillation of acute onset, in the opinion of the Investigator.

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	Drug: SANGUINATE; Single two-hour infusion of SANGUINATE; Other Names: pegylated carboxyhemoglobin bovine
Placebo Comparator: Placebo; Normal saline IV infusion	Drug: Placebo; Single two-hour infusion of placebo; Other Names: Normal Saline

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to readiness for discharge from ambulatory site	Defined as the time from the start of study drug infusion until the time of participant's response that their pain episode has improved enough for discharge; investigator's assessment of participant's readiness for discharge; and participant no longer requires IV opioid administration	7 Days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety of treatment	as determined by changes in vital signs, EKG, biochemical, hematological, and urinalysis measures, and reported AEs	1 Day
Extent of reduction in pain score during ambulatory visit as assessed by the participant on a 10-point pain scale		1 Day
Total pain treatment received including the opioid dose (mg/kg) received during ambulatory visit		1 Day
Global assessment of the participant's improvement by the Investigator or study staff (at the time of readiness for discharge from the ambulatory visit, and at the 72-hour follow-up visit)		7 Days
Reduction in the rate of recurrent ambulatory visit(s) for the VOC within 7 days post discharge		7 Days
Reduction in the rate of hospitalization(s) for VOC after treatment with SANGUINATE and within 7 days post-discharge		7 Days

Collaborators and Investigators

• Sponsor: Prolong Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): November 1, 2016
• Primary Completion (Actual): December 1, 2017
• Study Completion (Actual): December 1, 2017

Study Registration Dates

• First Submitted: April 2, 2015
• First Submitted That Met QC Criteria: April 7, 2015
• First Posted (Estimate): April 8, 2015

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

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Anemia, Sickle Cell
• Other Study ID Numbers: SGSC-005

Plan for Individual participant data (IPD)

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