A Phase III, Multicenter, 52-Week, Randomized, Double-Blind, Placebo-Controlled Study of the Clinical Efficacy and Safety of ICA-17043 with or without Hydroxyurea Therapy in Patients with Sickle Cell Disease who have had = 2 Acute Sickle-Related Painful Crises within the Preceding 12 Months. - A Stratified Sickle Event Randomised Trial - ASSERT

Phase 1

• Conditions: Sickle Cell Disease

• Registration Number: EUCTR2004-004617-42-GB
• Lead Sponsor: Icagen Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2005-05-11
• Study Completion: 2007-06-01
• Last Update Posted: 27 July 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 328

Inclusion Criteria

Patients with sickle cell disease (SCD) meeting all of the following criteria will be considered for admission to the study:
1. 16 to 65 years of age (inclusive) with a minimum weight of 40 kilograms for those patients aged 16-17 (inclusive);
2. Male, or female not capable of becoming pregnant or using appropriate birth control method;
3. Negative serum pregnancy test on Screening Day and a negative urine pregnancy test (dipstick) prior to dosing on Day 1 (for females);
4. Confirmed medical history or diagnosis of SCD (e.g. HbSS, HbSC, HbSß0-thalassemia, HbSß+-thalassemia patients);
5. Have received HU for preceeding 12 months and be dose stabilised with HU for at least 90 days prior to day 1;
6. Have a history of at least two or more acute sickle-related painful crises requiring a visit to a medical facility within the preceding 12 months;
7. Acceptable study admission chest x-ray upon enrollment (i.e., no evidence of acute pulmonary infiltrates). Most recent chest x-ray must be no more than 90 days prior to Day 1;
8. Clinically acceptable 12-lead ECG on screening, with a normal QTc interval for this population (less than or equal to 475 msec) ;
9. Clinically acceptable medical history, physical examination, vital signs, clinical laboratory tests (see Exclusion Criteria #8, below, for specific laboratory exclusions); and
10. Have willingly given written informed consent (and/or assent for those patients under 18 years of age) to participate in this investigation.

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years)
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Patients presenting with any of the following will not be included in the study:
1. Any patient who has experienced any allergic reaction to any drugs which, in the opinion of the investigator, suggests an increased potential for a hypersensitivity to ICA-17043;
2. Any patient who has received ICA-17043 in a previous investigational study;
3. Any patient on a chronic transfusion program, has had a transfusion within 30 days prior to Day 1 or an exchange transfusion 60 days prior to day 1;
4. Any patient whose hemoglobin is < 4.0 g/dL or >11.0 g/dL
5. Any patient considering or scheduled to undergo a major surgical procedure during the duration of the study;
6. Patients with significant active and poorly controlled (unstable) cardiovascular (including atrial or ventricular cardiac arrhythmias or long QT syndrome), neurologic, endocrine, hepatic, or renal disorders. Laboratory abnormalities that will lead to exclusion are:

SystemLaboratory TestExcluded Abnormal Range
RenalCreatininemore than or equal to 1.5 mg/dL
LiverTotal bilirubinmore than or equal to 20.0 mg/dL
ALT (SGPT)more than or equal to 2x upper limit of normal range

7. Any patient diagnosed with cancer (except non-melanoma skin cancer) within the last 5 years;
8. Ingestion of any investigational medication within 30 days prior to Day 1, or plans for participating in another investigational drug trial for the duration of the study;
9. One or more of the following markers of hepatitis
a. a history of hepatitis B or C clinical infection;
b. a positive test for heptitis B surface antigen; OR
c. a positive test for hepatitis C antibody AND has evidence of active liver disease (e.g., elevated liver enzymes, signs and symptoms of portal hypertension including coagulopathy, ascites, encephalopathy or histopathological evidence of active liver inflammation from a liver biopsy);
10. Subjects with a history of HIV infection or demonstration of HIV antibodies;
11. A positive qualitative urine drug test at Screening (for cocaine, phencyclidine (PCP), or amphetamines); and
12. Any patient with a serious mental (including psychosis) or physical illness, which, in the opinion of the investigator would compromise participation in the study (e.g. impaired mental capacity, alcoholism);

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified