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

• ClinicalTrials.gov Identifier: NCT02411708
• Recruitment Status: Completed
• First Posted: April 8, 2015
• Last Update Posted: May 23, 2018
• Sponsor: Prolong Pharmaceuticals
• Information provided by (Responsible Party): Prolong Pharmaceuticals

Tracking Information

• First Submitted Date: April 2, 2015
• First Posted Date: April 8, 2015
• Last Update Posted Date: May 23, 2018
• Actual Study Start Date: November 2016
• Actual Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: November 29, 2016)

Time to readiness for discharge from ambulatory site [ Time Frame: 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

Original Primary Outcome Measures (submitted: April 7, 2015)

• Safety of treatment as determined by changes in vital signs, electrocardiographic, biochemical, hematological, and urinalysis measures, as well as reported increases in pain and other reported adverse events [ Time Frame: 7 Days ]
• Time to readiness to discharge: Patient's response that their pain episode has improved enough for discharge from the clinic; Investigator's assessment of patient's readiness for discharge from the clinic; Time to last IV opioid administration. [ Time Frame: 1 Day ]

Current Secondary Outcome Measures (submitted: November 29, 2016)

• Safety of treatment [ Time Frame: 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 [ Time Frame: 1 Day ]
• Total pain treatment received including the opioid dose (mg/kg) received during ambulatory visit [ Time Frame: 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) [ Time Frame: 7 Days ]
• Reduction in the rate of recurrent ambulatory visit(s) for the VOC within 7 days post discharge [ Time Frame: 7 Days ]
• Reduction in the rate of hospitalization(s) for VOC after treatment with SANGUINATE and within 7 days post-discharge [ Time Frame: 7 Days ]

Original Secondary Outcome Measures (submitted: April 7, 2015)

• Number of patients on treatment with pain score at discharge ≤ 5 on a 10-point scale [ Time Frame: 1 Day ]
• Total pain treatment received during clinic stay [ Time Frame: 1 Day ]
• Total opioid dose (mg/kg) administered during clinic stay [ Time Frame: 1 Day ]
• Patient overall improvement using the 10-point scale Numeric Pain Rating Scale [ Time Frame: 1 Day ]
• Global assessment of the patient's improvement by the investigator (at the time of clinic discharge, at the 72-hour follow-up visit, and during the Day 7 telephone contact) using the Physician Global Assessment - Improvement Scale (PGA-I). [ Time Frame: 7 Days ]
• Reduction in the repeat clinic visit rate for VOC (clinic discharge to 7 days post-discharge) [ Time Frame: 7 Days ]
• Reduction in the hospitalization rate for VOC (clinic discharge to 7 days post-discharge) [ Time Frame: 7 days ]

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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
• 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 Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Single (Participant); Primary Purpose: Treatment
• Condition: Anemia, Sickle Cell

Intervention

• Drug: SANGUINATE
  Single two-hour infusion of SANGUINATE
  Other Name: pegylated carboxyhemoglobin bovine
• Drug: Placebo
  Single two-hour infusion of placebo
  Other Name: Normal Saline

Study Arms

• Experimental: SANGUINATE
  320 mg/kg
  Intervention: Drug: SANGUINATE
• Placebo Comparator: Placebo
  Normal saline IV infusion
  Intervention: Drug: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: April 7, 2015): 24
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: December 2017
• Actual Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT02411708
• Other Study ID Numbers: SGSC-005
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Prolong Pharmaceuticals
• Original Responsible Party: Same as current
• Current Study Sponsor: Prolong Pharmaceuticals
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Prolong Pharmaceuticals
• Verification Date: May 2018