SANGUINATE™ in Sickle Cell Disease Associated Leg Ulcer

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT02600390
• Date of registration: 02/11/2015
• Prospective Registration: Yes
• Primary sponsor: Prolong Pharmaceuticals
• Public title: SANGUINATE™ in Sickle Cell Disease Associated Leg Ulcer LU
• Scientific 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 Disease
• Date of first enrolment: March 2016
• Target sample size: 10
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02600390
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Dominican Republic, Panama

Contacts

• Name: Hemant Misra, PhD
• Affiliation: Prolong Pharma

Key inclusion & exclusion criteria

Inclusion Criteria:

- Properly consented patients suffering from Sickle Cell Disease (Hb-SS or Hb- Sß°
genotype) associated Leg ulcer.

- Presence of leg ulcer for at least 4 weeks

- Venous Clinical Severity Score of moderate to severe (defined as =12)

- Leg ulcer below the knee

Exclusion Criteria:

- Patient presenting with a clinically & laboratory confirmed bacterial, fungal or acid
fast organisms

- Patient has a recent acute complication of Sickle Cell Disease (e.g., pain crisis
within 7 days, or acute chest syndrome within 21 days)

- Patient is planning to be pregnant, is pregnant, or is breast-feeding

- Presence of moderate to severe renal insufficiency (CrCl < 30 mL/min) or chronic
kidney disease, or of moderate to severe hepatic insufficiency (Child-Pugh class B or
C)

- Patients with more than 3 times the upper limit of normal laboratory reference range

- Concurrent or recent prior treatment (within 90 days) with an investigational
medication

- Patients currently receiving treatment with hydroxyurea must be on stable dose for at
least 30 days

- Receipt of a blood transfusion within 21 days



Age minimum: 18 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Leg Ulcer

Intervention(s)

Biological: SANGUINATE

Primary Outcome(s)

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]

Rate and extent of leg ulcer wound healing as measured by change in wound surface area [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]

Secondary Outcome(s)

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 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 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]

Changes in leg ulcer wound status as measured by overall change in leg ulcer wound appearance [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]

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 type and amount of granulation tissue [Time Frame: 8 or 10 weeks]

Secondary ID(s)

SGSC-009

Source(s) of Monetary Support

Please refer to primary and secondary sponsors