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

Anemia, Sickle Cell Clinical Trial

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 With Vaso-Occlusive Crisis (VOC)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02672540
• Other study ID #: SGSC-002
• Status: Completed
• Phase: Phase 2
• Start date: July 2016
• Est. completion date: May 31, 2017

Study information

• Verified date: May 2018
• Source: Prolong Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 34
• Est. completion date: May 31, 2017
• Est. primary completion date: May 31, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

Inclusion Criteria:

1. Age = 18 and = 65 years of age

2. Diagnosis of Sickle Cell Disease (Hb-SS or any Genotype)

3. Pain-score due to vaso-occlusive pain crisis (VOC) = 8 on a 10 point scale

4. VOC pain location = 1 sites typical of vaso-occlusive crisis

5. Patients with Priapism, acute chest syndrome, and/or with other Sickle Cell Disease comorbidities can be enrolled with good judgment of the Investigator.

6. Signed and dated informed written consent by the subject

7. Able to receive intravenous infusion of SANGUINATE or Normal Saline

8. Women of childbearing potential with a negative serum pregnancy test and using a reliable method of contraception during the study period and for 30 days thereafter. Male study participants also agree to use contraception for 30 days after the study period

Exclusion Criteria:

1. In the judgment of the investigator, the patient is not a good candidate for the study

2. Females who are lactating and/or breastfeeding

3. Fewer than 14 days since prior infusion pain medication treatment for VOC

4. Medical history or evidence of moderate to severe renal insufficiency (estimated GFR < 60 mL/min) or chronic kidney disease, or of moderate to severe hepatic disease (ALTs > 5 x ULN)

5. Concurrent or prior treatment within 30 days of Screening with an investigational medication.

6. Symptoms or electrocardiogram (ECG)-based signs of acute myocardial infarction, unstable angina pectoris, decompensated heart failure, third degree heart block or cardiac arrhythmia associated with hemodynamic instability;

7. Severe or unstable concomitant condition or disease (e.g., known significant neurologic deficit, cancer, hematologic, metabolic or coronary disease), or chronic condition (e.g., psychiatric disorder), that, in the opinion of the Investigator, may increase the risk associated with study participation or study drug administration, or may interfere with the interpretation of study results;

8. Evidence or history of regular alcohol abuse

9. Screening laboratory result indicating serologic positivity for hepatitis C antibodies or hepatitis B surface antigens, unless explained by a documented vaccination.

10. Unable to comply with study attendance, protocol procedures or other study requirements;

11. Abnormal Echocardiogram at Study Entry (defined as Tricuspid Regurgitant Jet Velocity >3.1 m/sec).

Related Conditions & MeSH terms

• Anemia, Sickle Cell

Intervention

Drug:
• SANGUINATE 320 mg/kg
Two-hour infusion of SANGUINATE on Day 1 and Day 2
• Normal Saline
Two-hour infusion of Normal Saline and Day 1 and Day 2

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Prolong Pharmaceuticals

Countries where clinical trial is conducted

Colombia,  Dominican Republic,  Honduras,  Panama, 

Outcome

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