Sickle Cell Anemia, Sickle Cell Disease
Conditions
Keywords
sickle cell anemia, sickle cell disease, anemia, ICA-17043, senicapoc
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.
Interventions
DRUGLow Dose ICA-17043
Low dose arm
DRUGHigh dose ICA-17043
150 mg Loading Dose; 10 mg daily dose
DRUGPlacebo
Placebo Loading dose capsules and maintenance dose tablets matched 10 mg active treatment group
Sponsors
Icagen
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 60 Years
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary efficacy endpoint was the change from Baseline in hemoglobin (Hb) | 12 Weeks |
Secondary
| Measure | Time frame |
|---|---|
| Changes in RBC indices, including: mean corpuscular volume (MCV), mean corpuscular Hb concentration (MCHC), and mean corpuscular Hb (MCH | 12 weeks |
| Other laboratory measures associated with sickle cell crises activity including: direct and indirect bilirubin and lactic dehydrogenase (LDH) | 12 weeks |
| Rate of painful crises | 12 weeks |
| Time to first painful crisis | 12 weeks |
| Morbidity of painful crises (maximum morbidity index, derived variable) | 12 weeks |
| Changes in other hematologic measurements | 12 weeks |
| Quality of Life (SF 36) | 12 Weeks |
| Health economic data | 12 weeks |
| Average plasma concentration | 12 weeks |
| Correlation between the average plasma concentration and the change in Hb from Baseline to study endpoint | 12 weeks |
| Pain intensity scores | 12 weeks |
Countries
United States
Outcome results
None listed