Clinical trial NCT00040677: A Study of the Efficacy and Safety of ICA-17043 (With or Without Hydroxyurea) in Patients With Sickle Cell Anemia.
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Sickle Cell Disease
Sickle Cell Anemia
Keywords: sickle cell anemia
sickle cell disease
anemia
ICA-17043
senicapoc
Lead sponsor: Icagen
Sponsor class: INDUSTRY
First posted: 2002-07-10
Enrollment: 90
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: The primary efficacy endpoint was the change from Baseline in hemoglobin (Hb)
timeFrame: 12 Weeks
Secondary outcomes:
measure: Changes in other hematologic measurements
timeFrame: 12 weeks
measure: Changes in RBC indices, including: mean corpuscular volume (MCV), mean corpuscular Hb concentration (MCHC), and mean corpuscular Hb (MCH
timeFrame: 12 weeks
measure: Other laboratory measures associated with sickle cell crises activity including: direct and indirect bilirubin and lactic dehydrogenase (LDH)
timeFrame: 12 weeks
measure: Rate of painful crises
timeFrame: 12 weeks
measure: Time to first painful crisis
timeFrame: 12 weeks
measure: Morbidity of painful crises (maximum morbidity index, derived variable)
timeFrame: 12 weeks
measure: Pain intensity scores
timeFrame: 12 weeks
measure: Quality of Life (SF 36)
timeFrame: 12 Weeks
measure: Health economic data
timeFrame: 12 weeks
measure: Average plasma concentration
timeFrame: 12 weeks
measure: Correlation between the average plasma concentration and the change in Hb from Baseline to study endpoint
timeFrame: 12 weeks
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Study Site
state: Alabama
city: Oakland
country: United States
facility: Study Site
state: California
city: San Francisco
country: United States
facility: Study Site
state: California
city: Washington
country: United States
facility: Study Site
state: District of Columbia
city: Augusta
country: United States
facility: Study Site
state: Georgia
city: Chicago
country: United States
facility: Study Site
state: Illinois
city: Baltimore
country: United States
facility: Study Site
state: Maryland
city: Boston
country: United States
facility: Study Site
state: Massachusetts
city: Detroit
country: United States
facility: Study Site
state: Michigan
city: Jackson
country: United States
facility: Study Site
state: Mississippi
city: Brooklyn
country: United States
facility: Study Site
state: New York
city: New York
country: United States
facility: Study Site
state: New York
city: Chapel Hill
country: United States
facility: Study Site
state: North Carolina
city: Durham
country: United States
facility: Study Site
state: North Carolina
city: Philadelphia
country: United States
facility: Study Site
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Study Site
state: Pennsylvania
city: Nashville
country: United States
facility: Study Site
state: Tennessee
city: Houston
country: United States
facility: Study Site
state: Texas
city: Richmond
country: United States
facility: Study Site
state: Virginia
Study officials:
affiliation: University of North Carolina, Chapel Hill
name: Kenneth I Ataga, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Ataga KI, Smith WR, De Castro LM, Swerdlow P, Saunthararajah Y, Castro O, Vichinsky E, Kutlar A, Orringer EP, Rigdon GC, Stocker JW; ICA-17043-05 Investigators. Efficacy and safety of the Gardos channel blocker, senicapoc (ICA-17043), in patients with sickle cell anemia. Blood. 2008 Apr 15;111(8):3991-7. doi: 10.1182/blood-2007-08-110098. Epub 2008 Jan 11.