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

Primary Purpose

Sickle Cell Disease, Sickle Cell Anemia

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Low Dose ICA-17043
High dose ICA-17043
Placebo
Sponsored by
Icagen
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Sickle Cell Disease focused on measuring sickle cell anemia, sickle cell disease, anemia, ICA-17043, senicapoc

Eligibility Criteria

18 Years - 60 Years (Adult)All SexesDoes not accept 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

Sites / Locations

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Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Placebo Comparator

Experimental

Arm Label

ICA-17043 Low Dose 6 mg/day

Placebo

ICA-17043 High Dose 10 mg/day

Arm Description

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

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

Outcomes

Primary Outcome Measures

The primary efficacy endpoint was the change from Baseline in hemoglobin (Hb)

Secondary Outcome Measures

Changes in other hematologic measurements
Changes in RBC indices, including: mean corpuscular volume (MCV), mean corpuscular Hb concentration (MCHC), and mean corpuscular Hb (MCH
Other laboratory measures associated with sickle cell crises activity including: direct and indirect bilirubin and lactic dehydrogenase (LDH)
Rate of painful crises
Time to first painful crisis
Morbidity of painful crises (maximum morbidity index, derived variable)
Pain intensity scores
Quality of Life (SF 36)
Health economic data
Average plasma concentration
Correlation between the average plasma concentration and the change in Hb from Baseline to study endpoint

Full Information

First Posted
July 8, 2002
Last Updated
July 13, 2011
Sponsor
Icagen
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1. Study Identification

Unique Protocol Identification Number
NCT00040677
Brief Title
A Study of the Efficacy and Safety of ICA-17043 (With or Without Hydroxyurea) in Patients With Sickle Cell Anemia.
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2011
Overall Recruitment Status
Completed
Study Start Date
February 2002 (undefined)
Primary Completion Date
November 2003 (Actual)
Study Completion Date
January 2004 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Icagen

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease, Sickle Cell Anemia
Keywords
sickle cell anemia, sickle cell disease, anemia, ICA-17043, senicapoc

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
90 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ICA-17043 Low Dose 6 mg/day
Arm Type
Experimental
Arm Description
Active study medication: 100 mg loading dose; 6 mg maintenance dose per day
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Title
ICA-17043 High Dose 10 mg/day
Arm Type
Experimental
Arm Description
Active study medication: 150 mg loading dose; 10 mg maintenance dose per day
Intervention Type
Drug
Intervention Name(s)
Low Dose ICA-17043
Intervention Description
Low dose arm
Intervention Type
Drug
Intervention Name(s)
High dose ICA-17043
Intervention Description
150 mg Loading Dose; 10 mg daily dose
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo Loading dose capsules and maintenance dose tablets matched 10 mg active treatment group
Primary Outcome Measure Information:
Title
The primary efficacy endpoint was the change from Baseline in hemoglobin (Hb)
Time Frame
12 Weeks
Secondary Outcome Measure Information:
Title
Changes in other hematologic measurements
Time Frame
12 weeks
Title
Changes in RBC indices, including: mean corpuscular volume (MCV), mean corpuscular Hb concentration (MCHC), and mean corpuscular Hb (MCH
Time Frame
12 weeks
Title
Other laboratory measures associated with sickle cell crises activity including: direct and indirect bilirubin and lactic dehydrogenase (LDH)
Time Frame
12 weeks
Title
Rate of painful crises
Time Frame
12 weeks
Title
Time to first painful crisis
Time Frame
12 weeks
Title
Morbidity of painful crises (maximum morbidity index, derived variable)
Time Frame
12 weeks
Title
Pain intensity scores
Time Frame
12 weeks
Title
Quality of Life (SF 36)
Time Frame
12 Weeks
Title
Health economic data
Time Frame
12 weeks
Title
Average plasma concentration
Time Frame
12 weeks
Title
Correlation between the average plasma concentration and the change in Hb from Baseline to study endpoint
Time Frame
12 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kenneth I Ataga, MD
Organizational Affiliation
University of North Carolina, Chapel Hill
Official's Role
Principal Investigator
Facility Information:
Facility Name
Study Site
City
Birmingham
State/Province
Alabama
Country
United States
Facility Name
Study Site
City
Oakland
State/Province
California
Country
United States
Facility Name
Study Site
City
San Francisco
State/Province
California
Country
United States
Facility Name
Study Site
City
Washington
State/Province
District of Columbia
Country
United States
Facility Name
Study Site
City
Augusta
State/Province
Georgia
Country
United States
Facility Name
Study Site
City
Chicago
State/Province
Illinois
Country
United States
Facility Name
Study Site
City
Baltimore
State/Province
Maryland
Country
United States
Facility Name
Study Site
City
Boston
State/Province
Massachusetts
Country
United States
Facility Name
Study Site
City
Detroit
State/Province
Michigan
Country
United States
Facility Name
Study Site
City
Jackson
State/Province
Mississippi
Country
United States
Facility Name
Study Site
City
Brooklyn
State/Province
New York
Country
United States
Facility Name
Study Site
City
New York
State/Province
New York
Country
United States
Facility Name
Study Site
City
Chapel Hill
State/Province
North Carolina
Country
United States
Facility Name
Study Site
City
Durham
State/Province
North Carolina
Country
United States
Facility Name
Study Site
City
Philadelphia
State/Province
Pennsylvania
Country
United States
Facility Name
Study Site
City
Pittsburgh
State/Province
Pennsylvania
Country
United States
Facility Name
Study Site
City
Nashville
State/Province
Tennessee
Country
United States
Facility Name
Study Site
City
Houston
State/Province
Texas
Country
United States
Facility Name
Study Site
City
Richmond
State/Province
Virginia
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
18192510
Citation
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 sickle cell anemia. Blood. 2008 Apr 15;111(8):3991-7. doi: 10.1182/blood-2007-08-110098. Epub 2008 Jan 11.
Results Reference
result
Links:
URL
http://www.icagen.com
Description
For more information about Icagen
URL
http://www.SCInfo.org
Description
For more information about Sickle Cell Disease

Learn more about this trial

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

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