Sickle Cell DiseasePhase 2NCT00040677
A Study of the Efficacy and Safety of ICA-17043 (With or Without Hydroxyurea) in Patients With Sickle Cell Anemia.
A Phase 2 study testing Low Dose ICA-17043 and High dose ICA-17043 in people with Sickle Cell Disease and Sickle Cell Anemia, run by Icagen at 19 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Sickle Cell Disease, Sickle Cell Anemia
- What is being tested?
- Low Dose ICA-17043 and High dose ICA-17043
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 90 participants
- Where
- 19 sitesUnited States
- Study dates
- Feb 2002to Jan 2004
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
19 sites in 1 country.
19 sites in 1 country
United States19 sites
Study Site
Augusta, Georgia
Study Site
Baltimore, Maryland
Study Site
Birmingham, Alabama
Study Site
Boston, Massachusetts
Study Site
Brooklyn, New York
Study Site
Chapel Hill, North Carolina
Study Site
Chicago, Illinois
Study Site
Detroit, Michigan
Study Site
Durham, North Carolina
Study Site
Houston, Texas
Study Site
Jackson, Mississippi
Study Site
Nashville, Tennessee
Study Site
New York, New York
Study Site
Oakland, California
Study Site
Philadelphia, Pennsylvania
Study Site
Pittsburgh, Pennsylvania
Study Site
Richmond, Virginia
Study Site
San Francisco, California
Study Site
Washington D.C., District of Columbia
About this study
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
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.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia
- Being tested
- Low Dose ICA-17043, High dose ICA-17043, Placebo
- Keywords
- sickle cell anemia, sickle cell disease, anemia, ICA-17043, senicapoc
- Medical subject headings
- Anemia, Sickle Cell, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Low Dose ICA-17043Drug
Low dose arm
High dose ICA-17043Drug
150 mg Loading Dose; 10 mg daily dose
PlaceboDrug
Placebo Loading dose capsules and maintenance dose tablets matched 10 mg active treatment group
| Study group | Role | What they receive |
|---|---|---|
| ICA-17043 Low Dose 6 mg/day | Experimental | Active study medication: 100 mg loading dose; 6 mg maintenance dose per day Receives: Drug: Low Dose ICA-17043 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
| ICA-17043 High Dose 10 mg/day | Experimental | Active study medication: 150 mg loading dose; 10 mg maintenance dose per day Receives: Drug: High dose ICA-17043 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The primary efficacy endpoint was the change from Baseline in hemoglobin (Hb)
Measured: 12 Weeks
Secondary outcomes (11)
Changes in other hematologic measurements
Measured: 12 weeks
Changes in RBC indices, including: mean corpuscular volume (MCV), mean corpuscular Hb concentration (MCHC), and mean corpuscular Hb (MCH
Measured: 12 weeks
Other laboratory measures associated with sickle cell crises activity including: direct and indirect bilirubin and lactic dehydrogenase (LDH)
Measured: 12 weeks
Rate of painful crises
Measured: 12 weeks
Time to first painful crisis
Measured: 12 weeks
Morbidity of painful crises (maximum morbidity index, derived variable)
Measured: 12 weeks
Pain intensity scores
Measured: 12 weeks
Quality of Life (SF 36)
Measured: 12 Weeks
Health economic data
Measured: 12 weeks
Average plasma concentration
Measured: 12 weeks
Correlation between the average plasma concentration and the change in Hb from Baseline to study endpoint
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2002 (estimated)
- Study startFeb 2002
- Primary completionNov 2003
- Study completionJan 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 90 participants (actual)
Sponsor and investigators
- Lead sponsor
- Icagen
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Kenneth I Ataga, MD, Principal investigator, University of North Carolina, Chapel Hill
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00040677
- Sponsor's protocol ID
- ICA-17043-05
- Status last verified
- Jul 2011
- First submitted
- 8 Jul 2002
- Last updated
- 18 Jul 2011
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 18192510
Study at a glance
From the registry record, last updated by the sponsor on 18 Jul 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00040677
- Acronym / sponsor ID
- ICA-17043-05
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia
- Keywords
- sickle cell anemia, sickle cell disease, anemia, ICA-17043, senicapoc
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 90 participants (actual)
- Lead sponsor
- IcagenIndustry (pharma, biotech, devices)
- Study start
- Feb 2002
- Primary completion
- Nov 2003 (actual)
- Study completion
- Jan 2004 (actual)
- First posted
- 10 Jul 2002 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| ICA-17043 Low Dose 6 mg/day | Experimental | Drug: Low Dose ICA-17043Active study medication: 100 mg loading dose; 6 mg maintenance dose per day |
| Placebo | Placebo comparator | Drug: Placebo |
| ICA-17043 High Dose 10 mg/day | Experimental | Drug: High dose ICA-17043Active study medication: 150 mg loading dose; 10 mg maintenance dose per day |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Low Dose ICA-17043Low dose arm | Drug | ICA-17043 Low Dose 6 mg/day |
| High dose ICA-17043150 mg Loading Dose; 10 mg daily dose | Drug | ICA-17043 High Dose 10 mg/day |
| PlaceboPlacebo Loading dose capsules and maintenance dose tablets matched 10 mg active treatment group | Drug | Placebo |
Primary outcome
The primary efficacy endpoint was the change from Baseline in hemoglobin (Hb)
Time frame: 12 Weeks
11 secondary outcomes
Changes in other hematologic measurements
Time frame: 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
Other laboratory measures associated with sickle cell crises activity including: direct and indirect bilirubin and lactic dehydrogenase (LDH)
Time frame: 12 weeks
Rate of painful crises
Time frame: 12 weeks
Time to first painful crisis
Time frame: 12 weeks
Morbidity of painful crises (maximum morbidity index, derived variable)
Time frame: 12 weeks
Pain intensity scores
Time frame: 12 weeks
Quality of Life (SF 36)
Time frame: 12 Weeks
Health economic data
Time frame: 12 weeks
Average plasma concentration
Time frame: 12 weeks
Correlation between the average plasma concentration and the change in Hb from Baseline to study endpoint
Time frame: 12 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jul 2011.
- Results publication: PubMed 18192510Ataga 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.
- For more information about IcagenLink provided by the sponsor.
- For more information about Sickle Cell DiseaseLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kenneth I Ataga, MD principal investigator
University of North Carolina, Chapel Hill
19 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Low Dose ICA-17043 and High dose ICA-17043 in people with Sickle Cell Disease and Sickle Cell Anemia, run by Icagen at 19 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Jul 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The primary efficacy endpoint was the change from Baseline in hemoglobin (Hb) (time frame: 12 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00040677, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jul 2011.