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

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

Detailed Description
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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.

Official Title
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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

Conditions
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Anemia, Sickle Cell

Intervention / Treatment
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* Drug: SANGUINATE
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Age ≥ 18 years, Sickle Cell Disease (all genotypes), Diagnosis of a severe vaso-occlusive crisis (VOC), based on the clinical judgement of the Investigator, Participant needs to be admitted to the ambulatory site for treatment of VOC requiring IV pain medication, Able to provide written consent, Able to receive IV infusion of study drug. Exclusion Criteria: In the judgment of the Investigator, the participant is not a good candidate for the study, An acute severe complication of SCD beyond VOC, Pregnant or actively trying to become pregnant, or breastfeeding, Participant had > 6 urgent visits for SCD complications in the prior 3 months, Fewer than 30 days since any prior treatment with IV pain medication for VOC, Onset of current acute painful crisis > 3 days prior to dosing, 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, Concurrent or prior treatment within 90 days with an investigational medication, Abnormal ECG due to cardiac ischemia and/or atrial fibrillation of acute onset, in the opinion of the Investigator.

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
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: SANGUINATE<br>320 mg/kg | Drug: SANGUINATE<br>* Single two-hour infusion of SANGUINATE<br>* Other names: pegylated carboxyhemoglobin bovine;|
| Placebo Comparator: Placebo<br>Normal saline IV infusion | Drug: Placebo<br>* Single two-hour infusion of placebo<br>* Other names: Normal Saline;|

What is the study measuring?
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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 |