Clinical Trial: A Study of SANGUINATE for the Treatment of Vaso-occlusive Crisis (VOC) in Adult Sickle Cell Disease Patients

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

Official Title: A Phase II, Multicenter, Single-Blind, Randomized Study of the Safety and Effectiveness of SANGUINATE™ Versus Normal Saline in Adult Sickle Cell Disease Patients Wit

Brief Summary: Safety and effect of SANGUINATE on Sickle Cell Disease patients experiencing a vaso-occlusive crisis who are admitted to the hospital for treatment.

Detailed Summary: A single-blind, multicenter, randomized, placebo-control study in which 30 Sickle Cell disease patients having a vaso-occlusive crisis will either receive SANGUINATE 320 mg/kg/patient (8 mL/kg/patient) or Normal Saline on Day 1 (Visit 1) and Day 2 (Visit 2) infused over 2 hours each day. Patients are to remain in the hospital for up to 7 days but can be discharged at any time after receiving the second dose of SANGUINATE, provided their vaso-occlusive crisis has resolved and the patient has completed discharge procedures. Patients will have a follow-up phone call 7 days after discharge from the hospital to obtain safety, concomitant medication and pain assessments.
Sponsor: Prolong Pharmaceuticals

Current Primary Outcome: Time to readiness for discharge from in hospital stay following treatment with SANGUINATE and Normal Saline. [ Time Frame: Up to 7 Days ]

Defined as the patient's response that their pain episode has improved enough for discharge from the hospital, or the Investigator's assessment that the patient is ready for discharge from the hospital.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Safety of treatment as defined 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: Up to 7 Days ]
    Composite endpoint with multiple vital signs, ECGs, echocardiograms, clinical assessments and bio-analytical lab measurements over the 7 day time frame
  • Proportion of patients who develop acute chest syndrome (ACS) during the study. [ Time Frame: Up to 14 Days ]
  • Proportion of patients who are re-hospitalized for their vaso-occlusive crisis episode. [ Time Frame: Up to 7 Days ]
  • Total length of stay (LOS) following treatment of SANGUINATE versus Normal Saline. [ Time Frame: Up to 7 Days ]
  • Percent reduction in total pain medication required during in-hospital stay following treatment with SANGUINATE and Normal Saline. [ Time Frame: Up to 7 Days ]
  • Percent reduction in pain score utilizing a visual analog scale following treatment with SANGUINATE and Normal Saline. [ Time Frame: Up to 7 Days ]
  • Reduction in the level of C-Reactive Protein following treatment of SANGUINATE versus Normal Saline. [ Time Frame: Up to 7 Days ]


Original Secondary Outcome: Same as current

Information By: Prolong Pharmaceuticals

Dates:
Date Received: February 1, 2016
Date Started: August 2016
Date Completion: September 2017
Last Updated: March 30, 2017
Last Verified: March 2017