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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00040677
Recruitment Status : Completed
First Posted : July 10, 2002
Last Update Posted : July 18, 2011
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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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Sickle Cell Anemia Drug: Low Dose ICA-17043 Drug: High dose ICA-17043 Drug: Placebo Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 90 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, 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
Study Start Date : February 2002
Actual Primary Completion Date : November 2003
Actual Study Completion Date : January 2004

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia
Drug Information available for: Hydroxyurea
U.S. FDA Resources

Arm Intervention/treatment
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

Primary Outcome Measures :
  1. The primary efficacy endpoint was the change from Baseline in hemoglobin (Hb) [ Time Frame: 12 Weeks ]

Secondary Outcome Measures :
  1. Changes in other hematologic measurements [ Time Frame: 12 weeks ]
  2. Changes in RBC indices, including: mean corpuscular volume (MCV), mean corpuscular Hb concentration (MCHC), and mean corpuscular Hb (MCH [ Time Frame: 12 weeks ]
  3. Other laboratory measures associated with sickle cell crises activity including: direct and indirect bilirubin and lactic dehydrogenase (LDH) [ Time Frame: 12 weeks ]
  4. Rate of painful crises [ Time Frame: 12 weeks ]
  5. Time to first painful crisis [ Time Frame: 12 weeks ]
  6. Morbidity of painful crises (maximum morbidity index, derived variable) [ Time Frame: 12 weeks ]
  7. Pain intensity scores [ Time Frame: 12 weeks ]
  8. Quality of Life (SF 36) [ Time Frame: 12 Weeks ]
  9. Health economic data [ Time Frame: 12 weeks ]
  10. Average plasma concentration [ Time Frame: 12 weeks ]
  11. Correlation between the average plasma concentration and the change in Hb from Baseline to study endpoint [ Time Frame: 12 weeks ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00040677

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
Principal Investigator: Kenneth I Ataga, MD University of North Carolina, Chapel Hill

Additional Information:
Publications of Results:
Responsible Party: Greg Rigdon, Vice President New Product Development, Icagen Identifier: NCT00040677     History of Changes
Other Study ID Numbers: ICA-17043-05
First Posted: July 10, 2002    Key Record Dates
Last Update Posted: July 18, 2011
Last Verified: July 2011

Keywords provided by Icagen:
sickle cell anemia
sickle cell disease

Additional relevant MeSH terms:
Anemia, Sickle Cell
Hematologic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Genetic Diseases, Inborn
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors