A Study of the Efficacy and Safety of ICA-17043 (With or Without Hydroxyurea) in Patients With Sickle Cell Anemia.
This study has been completed.
Sponsor:
Icagen
Information provided by:
Icagen
ClinicalTrials.gov Identifier:
NCT00040677
First received: July 8, 2002
Last updated: July 13, 2011
Last verified: July 2011
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Sickle Cell Disease Sickle Cell Anemia |
Drug: Low Dose ICA-17043 Drug: High dose ICA-17043 Drug: Placebo |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
| 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 |
Resource links provided by NLM:
Genetics Home Reference related topics:
sickle cell disease
Drug Information available for:
Hydroxyurea
U.S. FDA Resources
Further study details as provided by Icagen:
Primary Outcome Measures:
- The primary efficacy endpoint was the change from Baseline in hemoglobin (Hb) [ Time Frame: 12 Weeks ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Changes in other hematologic measurements [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
- Changes in RBC indices, including: mean corpuscular volume (MCV), mean corpuscular Hb concentration (MCHC), and mean corpuscular Hb (MCH [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
- Other laboratory measures associated with sickle cell crises activity including: direct and indirect bilirubin and lactic dehydrogenase (LDH) [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
- Rate of painful crises [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
- Time to first painful crisis [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
- Morbidity of painful crises (maximum morbidity index, derived variable) [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
- Pain intensity scores [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
- Quality of Life (SF 36) [ Time Frame: 12 Weeks ] [ Designated as safety issue: No ]
- Health economic data [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
- Average plasma concentration [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
- Correlation between the average plasma concentration and the change in Hb from Baseline to study endpoint [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
| Enrollment: | 90 |
| Study Start Date: | February 2002 |
| Study Completion Date: | January 2004 |
| Primary Completion Date: | November 2003 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: ICA-17043 Low Dose 6 mg/day
Active study medication: 100 mg loading dose; 6 mg maintenance dose per day
|
Drug: Low Dose ICA-17043
Low dose arm
|
| Placebo Comparator: Placebo |
Drug: Placebo
Placebo Loading dose capsules and maintenance dose tablets matched 10 mg active treatment group
|
|
Experimental: ICA-17043 High Dose 10 mg/day
Active study medication: 150 mg loading dose; 10 mg maintenance dose per day
|
Drug: High dose ICA-17043
150 mg Loading Dose; 10 mg daily dose
|
Eligibility| Ages Eligible for Study: | 18 Years to 60 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00040677
Locations
| United States, Alabama | |
| Study Site | |
| Birmingham, Alabama, United States | |
| United States, California | |
| Study Site | |
| Oakland, California, United States | |
| Study Site | |
| San Francisco, California, United States | |
| United States, District of Columbia | |
| Study Site | |
| Washington, District of Columbia, United States | |
| United States, Georgia | |
| Study Site | |
| Augusta, Georgia, United States | |
| United States, Illinois | |
| Study Site | |
| Chicago, Illinois, United States | |
| United States, Maryland | |
| Study Site | |
| Baltimore, Maryland, United States | |
| United States, Massachusetts | |
| Study Site | |
| Boston, Massachusetts, United States | |
| United States, Michigan | |
| Study Site | |
| Detroit, Michigan, United States | |
| United States, Mississippi | |
| Study Site | |
| Jackson, Mississippi, United States | |
| United States, New York | |
| Study Site | |
| Brooklyn, New York, United States | |
| Study Site | |
| New York, New York, United States | |
| United States, North Carolina | |
| Study Site | |
| Chapel Hill, North Carolina, United States | |
| Study Site | |
| Durham, North Carolina, United States | |
| United States, Pennsylvania | |
| Study Site | |
| Philadelphia, Pennsylvania, United States | |
| Study Site | |
| Pittsburgh, Pennsylvania, United States | |
| United States, Tennessee | |
| Study Site | |
| Nashville, Tennessee, United States | |
| United States, Texas | |
| Study Site | |
| Houston, Texas, United States | |
| United States, Virginia | |
| Study Site | |
| Richmond, Virginia, United States | |
Sponsors and Collaborators
Icagen
Investigators
| Principal Investigator: | Kenneth I Ataga, MD | University of North Carolina, Chapel Hill |
More Information
Additional Information:
Publications:
| Responsible Party: | Greg Rigdon, Vice President New Product Development, Icagen |
| ClinicalTrials.gov Identifier: | NCT00040677 History of Changes |
| Other Study ID Numbers: | ICA-17043-05 |
| Study First Received: | July 8, 2002 |
| Last Updated: | July 13, 2011 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Icagen:
|
sickle cell anemia sickle cell disease anemia ICA-17043 senicapoc |
Additional relevant MeSH terms:
|
Anemia Anemia, Sickle Cell Hematologic Diseases Anemia, Hemolytic, Congenital Anemia, Hemolytic Hemoglobinopathies Genetic Diseases, Inborn Hydroxyurea |
Antineoplastic Agents Therapeutic Uses Pharmacologic Actions Antisickling Agents Hematologic Agents Enzyme Inhibitors Molecular Mechanisms of Pharmacological Action Nucleic Acid Synthesis Inhibitors |
ClinicalTrials.gov processed this record on May 23, 2013