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Completed

NCT Number: NCT00040677

A Study of the Efficacy and Safety of ICA-17043 (With or Without Hydroxyurea) 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.

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Key information

Conditions

Sickle Cell Disease Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Sickle Cell Anemia

Age range

18 year–60 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Study Site, Birmingham, Alabama, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Low Dose ICA-17043

Drug

Low dose arm

High dose ICA-17043

Drug

150 mg Loading Dose; 10 mg daily dose

Placebo

Drug

Placebo Loading dose capsules and maintenance dose tablets matched 10 mg active treatment group

Primary outcomes

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

    Time frame: 12 Weeks

Secondary outcomes

  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

Sponsors and collaborators

Lead sponsor

Icagen

Industry

Registry information

Official study 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

Important dates

Study start
2002
Primary completion
2003
Study completion
2004
First posted
Jul 10, 2002
Registry last updated
Jul 18, 2011

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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