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

July 13, 2011 updated by: Icagen

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

90

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 60 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Drug: Placebo
Placebo Loading dose capsules and maintenance dose tablets matched 10 mg active treatment group
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
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
The primary efficacy endpoint was the change from Baseline in hemoglobin (Hb)
Time Frame: 12 Weeks
12 Weeks

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Icagen

Investigators

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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2002

Primary Completion (Actual)

November 1, 2003

Study Completion (Actual)

January 1, 2004

Study Registration Dates

First Submitted

July 8, 2002

First Submitted That Met QC Criteria

July 9, 2002

First Posted (Estimate)

July 10, 2002

Study Record Updates

Last Update Posted (Estimate)

July 18, 2011

Last Update Submitted That Met QC Criteria

July 13, 2011

Last Verified

July 1, 2011

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ICA-17043-05

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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