Clinical Trial: SANGUINATE™ in Sickle Cell Disease Associated Leg Ulcer

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: An Open-label, Repeated-dose, Dose Escalation Study of the Safety and Effectiveness of SANGUINATE™ for the Treatment of Leg Ulcers in Adult Patients With Sickle Cell

Brief Summary: SANGUINATE™ Sickle Cell Disease associated Leg Ulcers.

Detailed Summary:

This is an escalating, repeated-dose, open-label Phase 2 study to test SANGUINATE (pegylated carboxyhemoglobin bovine) in patients suffering from leg ulceration associated with Sickle Cell Disease (SCD leg ulcer).

Patients will undergo consent and screening procedures at the time of presentation for leg ulcer treatment. Eligible patients will begin a 3-week Run-In Period. A total of approximately fifteen (15) patients are expected to be screened in order to achieve an enrollment target of ten (10) evaluable patients for participation in the study. Evaluable is defined as a patient receiving a minimum of three doses of investigational product. Five (5) patients (Cohort 1) will receive four (4) once-weekly 2-hour IV infusions of SANGUINATE at a dose of 320 mg/kg (8 mL). An additional five (5) patients (Cohort 2) will receive six (6) once-weekly 2-hour IV infusions of SANGUINATE also at a dose of 320 mg/kg. Each patient will be contacted by telephone approximately 24 hours after dosing to verify the safety status of the patient. One week following the Treatment Phase, patients will return to the study center for a Final Visit where safety assessments will be performed. During the Final Visit, patients will receive standard of care treatment of ulcer. All patients will receive once-weekly Standard of Care (SOC) therapy for SCD leg ulcer for the duration of the study. SOC includes dressing the wound with multi-layer, short stretch, compression bandaging. Non-adherent, medical-grade foam will be used as the wound contact layer and primary dressing.


Sponsor: Prolong Pharmaceuticals

Current Primary Outcome:

  • Safety of treatment as determined by changes in vital signs, electrocardiographic, biochemical, hematological, and urinalysis measures, and reported adverse events [ Time Frame: 8 or 10 weeks ]
  • Change in Sickle Cell Disease leg ulcer wound pain scores as measured by a 10-point scale, relative to non-wound site pain scores [ Time Frame: 8 or 10 weeks ]
  • Rate and extent of leg ulcer wound healing as measured by change in wound surface area [ Time Frame: 8 or 10 weeks ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Changes in leg ulcer wound status as measured by need for debridement (type and quantity of non-viable tissue) [ Time Frame: 8 or 10 weeks ]
  • Changes in leg ulcer wound status as measured by extent of exudate production [ Time Frame: 8 or 10 weeks ]
    Change in quality of life as measured by SF-12 Health Survey (acute version) as well as other indicators of patient comfort and well-being
  • Changes in leg ulcer wound status as measured by type and amount of granulation tissue [ Time Frame: 8 or 10 weeks ]
  • Changes in leg ulcer wound status as measured by overall change in leg ulcer wound appearance [ Time Frame: 8 or 10 weeks ]
  • Change in local skin in the region of the leg ulcer as measured by skin appearance (coloration, inflammation, etc.) [ Time Frame: 8 or 10 weeks ]
  • Change in local skin in the region of the leg ulcer as measured by skin temperature (relative to body temperature) [ Time Frame: 8 or 10 weeks ]
  • Improvement in leg ulcer wound severity as measured by Venous Clinical Severity Score (VCSS) [ Time Frame: 8 or 10 weeks ]
  • Change in quality of life, as measured by an SF-12 Health Survey (acute version) as well as other indicators of patient comfort and well-being [ Time Frame: 8 or 10 weeks ]


Original Secondary Outcome: Same as current

Information By: Prolong Pharmaceuticals

Dates:
Date Received: November 2, 2015
Date Started: March 2016
Date Completion:
Last Updated: February 24, 2017
Last Verified: February 2017