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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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 Adult

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

Detailed Summary: 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.
Sponsor: Prolong Pharmaceuticals

Current Primary Outcome: 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:

  • 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:

  • 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:

  • 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 ]


Information By: Prolong Pharmaceuticals

Dates:
Date Received: April 2, 2015
Date Started: November 2016
Date Completion: December 2017
Last Updated: April 26, 2017
Last Verified: November 2016