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

Anemia, Sickle Cell Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02411708
• Other study ID #: SGSC-005
• Status: Completed
• Phase: Phase 2
• Start date: November 2016
• Est. completion date: December 2017

Study information

• Verified date: May 2018
• Source: Prolong Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 24
• Est. completion date: December 2017
• Est. primary completion date: December 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Anemia, Sickle Cell

Intervention

Drug:
• SANGUINATE
Single two-hour infusion of SANGUINATE
• Placebo
Single two-hour infusion of placebo

Locations

Country	Name	City	State
United States	Johns Hopkins Univeristy School of Medicine	Baltimore	Maryland
United States	University of Maryland School of Medicine	Baltimore	Maryland
United States	Ohio State University Medical Center	Columbus	Ohio
United States	FSCDR	Hollywood	Florida
United States	Blood Center of Wisconsin	Milwaukee	Wisconsin
United States	Newark Beth Israel Medical Center	Newark	New Jersey
United States	Virginia Commonwealth University	Richmond	Virginia
United States	University of Rochester Medical Center	Rochester	New York
United States	Florida Health Tampa General Hospital	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Prolong Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Safety of treatment	as determined by changes in vital signs, EKG, biochemical, hematological, and urinalysis measures, and reported AEs	1 Day
Secondary	Extent of reduction in pain score during ambulatory visit as assessed by the participant on a 10-point pain scale		1 Day
Secondary	Total pain treatment received including the opioid dose (mg/kg) received during ambulatory visit		1 Day
Secondary	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
Secondary	Reduction in the rate of recurrent ambulatory visit(s) for the VOC within 7 days post discharge		7 Days
Secondary	Reduction in the rate of hospitalization(s) for VOC after treatment with SANGUINATE and within 7 days post-discharge		7 Days