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

Phase 2

Completed

• Conditions: Anemia, Sickle Cell
• Interventions: Drug: Placebo; Drug: SANGUINATE

• Registration Number: NCT02411708
• Lead Sponsor: Prolong Pharmaceuticals

Brief Summary

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

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 Timeline

• Study First Submitted: 2015-04-02
• Study First Submitted QC: 2015-04-07
• Study First Posted: 2015-04-08
• Study Start: 2016-11
• Primary Completion: 2017-12
• Study Completion: 2017-12
• Status Verified: 2018-05
• Last Update Submitted: 2018-05-22
• Last Update Posted: 2018-05-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 24

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Normal saline IV infusion
SANGUINATE	SANGUINATE	320 mg/kg

Primary Outcome Measures

Name	Time	Method
Time to readiness for discharge from ambulatory site	7 Days	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

Secondary Outcome Measures

Name	Time	Method
Safety of treatment	1 Day	as determined by changes in vital signs, EKG, biochemical, hematological, and urinalysis measures, and reported AEs
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

Trial Locations

Locations (9)

FSCDR; 🇺🇸 Hollywood, Florida, United States

University of Maryland School of Medicine; 🇺🇸 Baltimore, Maryland, United States

University of Rochester Medical Center; 🇺🇸 Rochester, New York, United States

Newark Beth Israel Medical Center; 🇺🇸 Newark, New Jersey, United States

Johns Hopkins Univeristy School of Medicine; 🇺🇸 Baltimore, Maryland, United States

Virginia Commonwealth University; 🇺🇸 Richmond, Virginia, United States

Ohio State University Medical Center; 🇺🇸 Columbus, Ohio, United States

Florida Health Tampa General Hospital; 🇺🇸 Tampa, Florida, United States

Blood Center of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States