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:
Information provided by:
Icagen
ClinicalTrials.gov Identifier:
NCT00040677
First received: July 8, 2002
Last updated: July 13, 2011
Last verified: July 2011
  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:


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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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 October 02, 2014