A Phase II, Multicenter, Twelve-Week, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Range-Finding Study of the Efficacy and Safety of ICA-17043 With or Without Hydroxyurea Therapy in Patients With Sickle Cell Anemia

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

Sponsors

Lead sponsor: Icagen

Source Icagen
Brief Summary

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.

Overall Status Completed
Start Date February 2002
Completion Date January 2004
Primary Completion Date November 2003
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
The primary efficacy endpoint was the change from Baseline in hemoglobin (Hb) 12 Weeks
Secondary Outcome
Measure Time Frame
Changes in other hematologic measurements 12 weeks
Changes in RBC indices, including: mean corpuscular volume (MCV), mean corpuscular Hb concentration (MCHC), and mean corpuscular Hb (MCH 12 weeks
Other laboratory measures associated with sickle cell crises activity including: direct and indirect bilirubin and lactic dehydrogenase (LDH) 12 weeks
Rate of painful crises 12 weeks
Time to first painful crisis 12 weeks
Morbidity of painful crises (maximum morbidity index, derived variable) 12 weeks
Pain intensity scores 12 weeks
Quality of Life (SF 36) 12 Weeks
Health economic data 12 weeks
Average plasma concentration 12 weeks
Correlation between the average plasma concentration and the change in Hb from Baseline to study endpoint 12 weeks
Enrollment 90
Condition
Intervention

Intervention type: Drug

Intervention name: Low Dose ICA-17043

Description: Low dose arm

Arm group label: ICA-17043 Low Dose 6 mg/day

Intervention type: Drug

Intervention name: High dose ICA-17043

Description: 150 mg Loading Dose; 10 mg daily dose

Arm group label: ICA-17043 High Dose 10 mg/day

Intervention type: Drug

Intervention name: Placebo

Description: Placebo Loading dose capsules and maintenance dose tablets matched 10 mg active treatment group

Arm group label: Placebo

Eligibility

Criteria:

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

Gender: All

Minimum age: 18 Years

Maximum age: 60 Years

Healthy volunteers: No

Overall Official
Last Name Role Affiliation
Kenneth I Ataga, MD Principal Investigator University of North Carolina, Chapel Hill
Location
facility
Study Site | Birmingham, Alabama, United States
Study Site | Oakland, California, United States
Study Site | San Francisco, California, United States
Study Site | Washington, District of Columbia, United States
Study Site | Augusta, Georgia, United States
Study Site | Chicago, Illinois, United States
Study Site | Baltimore, Maryland, United States
Study Site | Boston, Massachusetts, United States
Study Site | Detroit, Michigan, United States
Study Site | Jackson, Mississippi, United States
Study Site | Brooklyn, New York, United States
Study Site | New York, New York, United States
Study Site | Chapel Hill, North Carolina, United States
Study Site | Durham, North Carolina, United States
Study Site | Philadelphia, Pennsylvania, United States
Study Site | Pittsburgh, Pennsylvania, United States
Study Site | Nashville, Tennessee, United States
Study Site | Houston, Texas, United States
Study Site | Richmond, Virginia, United States
Location Countries

United States

Verification Date

July 2011

Responsible Party

Name title: Greg Rigdon, Vice President New Product Development

Organization: Icagen

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 3
Arm Group

Arm group label: ICA-17043 Low Dose 6 mg/day

Arm group type: Experimental

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

Arm group label: Placebo

Arm group type: Placebo Comparator

Arm group label: ICA-17043 High Dose 10 mg/day

Arm group type: Experimental

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

Study Design Info

Allocation: Randomized

Intervention model: Parallel Assignment

Primary purpose: Treatment

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Source: ClinicalTrials.gov