A Study of the Efficacy and Safety of ICA-17043 (With or Without Hydroxyurea) in Patients With Sickle Cell Anemia.

Icagen

Status and phase: Completed; Phase 2

Conditions: Sickle Cell Anemia; Sickle Cell Disease

Treatments: Drug: Low Dose ICA-17043; Drug: Placebo; Drug: High dose ICA-17043

Study type: Interventional

Funder types: Industry

Identifiers

NCT00040677

ICA-17043-05

Details and patient eligibility

About

ICA-17043 is being developed for the chronic treatment of patients with sickle cell disease (SCD) in both adults and children. ICA-17043 is a potent and specific inhibitor of a channel in human red blood cells (RBCs) that blocks RBC dehydration. ICA-17043 is expected to inhibit RBC dehydration and thus should prevent or delay the sickling process. By reducing sickled cells, an improvement in anemia, a reduction in painful crises, and ultimately, less end-organ disease is anticipated.

Enrollment: 90 patients

Sex: All

Ages: 18 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Homozygous (HbSS) Sickle Cell Anemia
• Otherwise healthy (based on medical history, physical examination, 12-lead ECG, and clinical laboratory tests)
• Patients may be receiving hydroxyurea, but must have been dose stabilized for at least 3 months
• Patient has a history of at least one acute vaso-occlusive event requiring hospitalization

Exclusion criteria

• Patient participating in a chronic transfusion program
• Patient having a total hemoglobin of < 4.0 g/dL or > 10.0 g/dL
• Patient having a HbA > 10%
• Patient considering undergoing an elective surgery
• Patient taking prohibited medications such as Epoetin, Warfarin, etc.
• Patient who has had previous gastrointestinal surgery, except cholecystectomy or appendectomy
• Patient with significant active cardiovascular, neurologic, endocrine, hepatic, or renal disorders unrelated to sickle cell anemia

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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90 participants in 3 patient groups, including a placebo group

ICA-17043 Low Dose 6 mg/day

Experimental group

Description:

Active study medication: 100 mg loading dose; 6 mg maintenance dose per day

Treatment:

Drug: Low Dose ICA-17043

Placebo

Placebo Comparator group

Treatment:

Drug: Placebo

ICA-17043 High Dose 10 mg/day

Experimental group

Description:

Active study medication: 150 mg loading dose; 10 mg maintenance dose per day

Treatment:

Drug: High dose ICA-17043