Sickle Cell Disease
Conditions
Sponsors
Icagen Inc
Eligibility
Sex/Gender
All
Inclusion criteria
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
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 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: System Laboratory Test Excluded Abnormal Range Renal Creatinine more than or equal to 1.5 mg/dL Liver Total bilirubin more 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);
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The objectives of this study are: 1. To establish the clinical efficacy of ICA-17043 when compared to placebo (with hydroxyurea (HU) as background therapy) in patients with sickle cell disease who have had = 2 acute sickle-related painful crises within the preceding 12 months; ;Secondary Objective: To obtain further safety information on the chronic dosing of ICA-17043 in patients with sickle cell disease; and To obtain ICA-17043 plasma concentrations following chronic dosing to assess steady-state ICA-17043 plasma concentrations in patients with sickle cell disease.;Primary end point(s): The primary efficacy end point will be the rate of acute sickle-related painful crises. The “painful crisis” rate for a patient is defined as the total number of painful crises divided by the number of months in which the patient was receiving treatment (i.e., the individual’s treatment phase). An acute sickle-related painful crisis, hereafter will be referred to as a “painful crisis”. A “painful crisis” must meet all of the criteria below: • an acute episode of pain; • no known cause for pain other than a vaso-occlusive event; • requiring a visit to a medical facility; and • requiring treatment with a parenteral, oral, or transdermal narcotics (including opiates), or parenteral NSAIDs. | — |
Countries
United Kingdom
Outcome results
None listed