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

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00040677
• Other study ID #: ICA-17043-05
• Status: Completed
• Phase: Phase 2
• First received: July 8, 2002
• Last updated: July 13, 2011
• Start date: February 2002
• Est. completion date: January 2004

Study information

• Verified date: July 2011
• Source: Icagen
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 90
• Est. completion date: January 2004
• Est. primary completion date: November 2003
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 60 Years

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia
• Anemia, Sickle Cell
• Sickle Cell Anemia
• Sickle Cell Disease

Intervention

Drug:
• Low Dose ICA-17043
Low dose arm
• High dose ICA-17043
150 mg Loading Dose; 10 mg daily dose
• Placebo
Placebo Loading dose capsules and maintenance dose tablets matched 10 mg active treatment group

Locations

Country	Name	City	State
United States	Study Site	Augusta	Georgia
United States	Study Site	Baltimore	Maryland
United States	Study Site	Birmingham	Alabama
United States	Study Site	Boston	Massachusetts
United States	Study Site	Brooklyn	New York
United States	Study Site	Chapel Hill	North Carolina
United States	Study Site	Chicago	Illinois
United States	Study Site	Detroit	Michigan
United States	Study Site	Durham	North Carolina
United States	Study Site	Houston	Texas
United States	Study Site	Jackson	Mississippi
United States	Study Site	Nashville	Tennessee
United States	Study Site	New York	New York
United States	Study Site	Oakland	California
United States	Study Site	Philadelphia	Pennsylvania
United States	Study Site	Pittsburgh	Pennsylvania
United States	Study Site	Richmond	Virginia
United States	Study Site	San Francisco	California
United States	Study Site	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Icagen

Country where clinical trial is conducted

United States, 

References & Publications (1)

Ataga KI, Smith WR, De Castro LM, Swerdlow P, Saunthararajah Y, Castro O, Vichinsky E, Kutlar A, Orringer EP, Rigdon GC, Stocker JW; ICA-17043-05 Investigators. Efficacy and safety of the Gardos channel blocker, senicapoc (ICA-17043), in patients with sic — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The primary efficacy endpoint was the change from Baseline in hemoglobin (Hb)		12 Weeks	No
Secondary	Changes in other hematologic measurements		12 weeks	No
Secondary	Changes in RBC indices, including: mean corpuscular volume (MCV), mean corpuscular Hb concentration (MCHC), and mean corpuscular Hb (MCH		12 weeks	No
Secondary	Other laboratory measures associated with sickle cell crises activity including: direct and indirect bilirubin and lactic dehydrogenase (LDH)		12 weeks	No
Secondary	Rate of painful crises		12 weeks	No
Secondary	Time to first painful crisis		12 weeks	No
Secondary	Morbidity of painful crises (maximum morbidity index, derived variable)		12 weeks	No
Secondary	Pain intensity scores		12 weeks	No
Secondary	Quality of Life (SF 36)		12 Weeks	No
Secondary	Health economic data		12 weeks	No
Secondary	Average plasma concentration		12 weeks	No
Secondary	Correlation between the average plasma concentration and the change in Hb from Baseline to study endpoint		12 weeks	No

External Links

•   For more information about Icagen
•   For more information about Sickle Cell Disease