Clinical trial NCT02411708: Study of SANGUINATE™ In the Treatment of Sickle Cell Disease Patients With Vaso-Occlusive Crisis
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Anemia, Sickle Cell
Lead sponsor: Prolong Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2015-04-08
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Time to readiness for discharge from ambulatory site
timeFrame: 7 Days
Secondary outcomes:
measure: Safety of treatment
timeFrame: 1 Day
measure: Extent of reduction in pain score during ambulatory visit as assessed by the participant on a 10-point pain scale
timeFrame: 1 Day
measure: Total pain treatment received including the opioid dose (mg/kg) received during ambulatory visit
timeFrame: 1 Day
measure: 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)
timeFrame: 7 Days
measure: Reduction in the rate of recurrent ambulatory visit(s) for the VOC within 7 days post discharge
timeFrame: 7 Days
measure: Reduction in the rate of hospitalization(s) for VOC after treatment with SANGUINATE and within 7 days post-discharge
timeFrame: 7 Days
Other outcomes:
Locations:
city: Hollywood
country: United States
facility: FSCDR
state: Florida
city: Tampa
country: United States
facility: Florida Health Tampa General Hospital
state: Florida
city: Baltimore
country: United States
facility: University of Maryland School of Medicine
state: Maryland
city: Baltimore
country: United States
facility: Johns Hopkins Univeristy School of Medicine
state: Maryland
city: Newark
country: United States
facility: Newark Beth Israel Medical Center
state: New Jersey
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
city: Columbus
country: United States
facility: Ohio State University Medical Center
state: Ohio
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Milwaukee
country: United States
facility: Blood Center of Wisconsin
state: Wisconsin
Study officials:
References: