Sickle Cell DiseasePhase 2NCT01090323
Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO).
A Phase 2 study testing ICL670 in people with Sickle Cell Disease, run by Novartis Pharmaceuticals at 39 sites in 5 countries.
Trial summary
The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- ICL670
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 185 participants
- Where
- 39 sitesin 5 countries
- Study dates
- Jul 2004to Jul 2009
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients were included who met the following criteria:
- Completion of the core [Study 0109]
- Serum ferritin greater than or equal to 500 µg/L
- Ability to comply with all study-related procedures, medications, and evaluations
- Sexually active post-menarche female patients must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.
- Written informed consent and assent by the patient and or their parents or legal guardian.
- Additional inclusion criteria for pediatric patients The definition of the term 'pediatric' for enrollment and study conduct was in accordance with local law. Parents or the legal guardians were fully informed by the investigator as to the requirements of the study. The pediatric patients themselves were informed according to their capabilities in a language and terms that they were able to understand. Written informed consent was obtained from their legal guardian on the patient's behalf in accordance with national legislation. If capable, all patients had to also personally sign their written informed consent.
Who cannot join
- Patients who met the following criteria were to be excluded:
- History of non-compliance to medical regimens and patients who are considered potentially unreliable and/or not cooperative
- Serum creatinine above the age-appropriate upper limit of normal within one week prior to entry
- Patients with ALT ≥ 500 U/L within one week prior to entry
- Evidence of chelation-related cataracts or hearing loss within 4 weeks prior to baseline
- Pregnancy (as indicated by serum β-HCG pregnancy test for all female patients with the potential to become pregnant) and patients who are breastfeeding
- Patients treated with systemic investigational drug within 4 weeks prior to or with topical investigational drug within 7 days prior to the baseline visit
- Other protocol-defined inclusion/exclusion criteria may apply
Trial locations and contacts
39 sites in 5 countries.
39 sites in 5 countries
United States31 sites
Grady Hospital, Georgia Comprehensive Sickle Cell Center
Atlanta, Georgia, 30303
Medical College of Georgia
Augusta, Georgia, 30912
Children's Hospital Boston
Boston, Massachusetts, 02118
New York Methodist Hospital
Brooklyn, New York, 11215
University of Illinois at Chicago
Chicago, Illinois, 60612
Children's Memorial Hospital
Chicago, Illinois, 60614
Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University of Cincinnati
Cincinnati, Ohio, 45267
Palmetto Health Richland
Columbia, South Carolina, 29203
University of Colorado Health Science Center
Denver, Colorado, 80262
Karmanos Cancer Institute
Detroit, Michigan, 48201
Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
Texas Children's Hospital
Houston, Texas, 77030
The Methodist Hospital
Houston, Texas, 77030
Loma Linda University Medical Center
Loma Linda, California, 92354
Children's Hospital Los Angeles
Los Angeles, California, 90027
St. Jude's Children Research Hospital
Memphis, Tennessee, 38105
University of South Alabama College of Medicine
Mobile, Alabama, 36604
Tulane University Medical Center
New Orleans, Louisiana, 70112
New York Presbyterian Hospital
New York, New York, 10021
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507
Children's Hospital & Research Center at Oakland
Oakland, California, 90609
Yasin
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213
LSUHSC Dept of Pediatrics
Shreveport, Louisiana, 71130
Santee Hematology/Oncology
Sumter, South Carolina, 29150
St Joseph Children's Hospital of Tampa
Tampa, Florida, 33607
Scott & White Memorial Hospital
Temple, Texas, 76508
Montefiore Medical Center
The Bronx, New York, 10467
Howard University Hospital
Washington D.C., District of Columbia, 20059
Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157
Italy4 sites
Novartis Investigative Site
Catania
Novartis Investigative Site
Genova
Novartis Investigative Site
Milan
Novartis Investigative Site
Roma
France2 sites
Novartis Investigative Site
Créteil
Novartis Investigative Site
Paris
About this study
Official title: A One Year Open Label, Non-comparative Extension to a Randomized, Multicenter, Phase II Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration (LIC) of Repeated Doses of 5-30mg/kg/Day ICL670 Relative to Deferoxamine (DFO) in Sickle Cell Disease (SCD) Patients With Transfusional Hemosideresis (THS) [Amendment 3: Extension Prolonged to 4-years]
The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis.
Read the detailed description
The treatment period started once the patient completed the core study and signed informed consent. It is continued for up to 4 years. Safety parameters were assessed every 4 weeks. Eye and Ear examinations were performed on a yearly basis. To further investigate the extent of iron overload, serum ferritin, iron, and transferrin were monitored every four weeks. The Program Safety Board monitored the safety of ICL670 during the study to evaluate and categorize any serious case reported in association with ICL670.
- Conditions
- Sickle Cell Disease
- Being tested
- ICL670
- Keywords
- Deferasirox, ICL670A, Iron chelators, Sickle Cell Disease, Transfusional Hemosiderosis
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ICL670Drug
Daily doses of ICL670 were taken orally 30 minutes before breakfast. The doses range from 5-40 mg/kg and were determined based on the patient's trend in serum ferritin over time during the core study (0109) and on the frequency of blood transfusions the patient received. The treatment duration was up to 4 years.
| Study group | Role | What they receive |
|---|---|---|
| ICL670 | Experimental | Not described Receives: Drug: ICL670 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events After Start of ICL670
Safety as assessed by the number of participants with adverse event or death after the start of ICL670.
Measured: 0 - 60 months
Secondary outcomes (1)
Change in Serum Ferritin From Start of ICL670 to End of Study
The main efficacy variable was change in serum ferritin in response to therapy with ICL670. Due to variability of serum ferritin, end of study was considered as the mean of at most the last 3 available observations after the start of ICL670.
Measured: 0 - 60 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2010 (estimated)
- Study startJul 2004
- Primary completionJul 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 185 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01090323
- Sponsor's protocol ID
- CICL670A0109E1
- Other IDs
- EudraCT no. 2004-000597-31
- Status last verified
- May 2011
- First submitted
- 15 Mar 2010
- Last updated
- 6 Jun 2011
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 Jun 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A One Year Open Label, Non-comparative Extension to a Randomized, Multicenter, Phase II Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration (LIC) of Repeated Doses of 5-30mg/kg/Day ICL670 Relative to Deferoxamine (DFO) in Sickle Cell Disease (SCD) Patients With Transfusional Hemosideresis (THS) [Amendment 3: Extension Prolonged to 4-years]
- ClinicalTrials.gov ID
- NCT01090323
- Acronym / sponsor ID
- CICL670A0109E1
- Conditions
- Sickle Cell Disease
- Keywords
- Deferasirox, ICL670A, Iron chelators, Sickle Cell Disease, Transfusional Hemosiderosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 185 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Jul 2004
- Primary completion
- Jul 2009 (actual)
- First posted
- 19 Mar 2010 (estimated)
- Results
- Posted 20 Jan 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The safety, tolerability, effects on liver iron concentration and pharmacokinetics of ICL670 is studied in sickle cell disease patients with transfusional hemosiderosis.
Detailed description
The treatment period started once the patient completed the core study and signed informed consent. It is continued for up to 4 years. Safety parameters were assessed every 4 weeks. Eye and Ear examinations were performed on a yearly basis. To further investigate the extent of iron overload, serum ferritin, iron, and transferrin were monitored every four weeks. The Program Safety Board monitored the safety of ICL670 during the study to evaluate and categorize any serious case reported in association with ICL670.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ICL670 | Experimental | Drug: ICL670 |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Completion of the core [Study 0109]
- Serum ferritin greater than or equal to 500 µg/L
- Ability to comply with all study-related procedures, medications, and evaluations
- Sexually active post-menarche female patients must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.
- Written informed consent and assent by the patient and or their parents or legal guardian.
Key exclusion criteria (8 listed)
- History of non-compliance to medical regimens and patients who are considered potentially unreliable and/or not cooperative
- Serum creatinine above the age-appropriate upper limit of normal within one week prior to entry
- Patients with ALT ≥ 500 U/L within one week prior to entry
- Evidence of chelation-related cataracts or hearing loss within 4 weeks prior to baseline
- Pregnancy (as indicated by serum β-HCG pregnancy test for all female patients with the potential to become pregnant) and patients who are breastfeeding
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 |
|---|---|---|
| ICL670Daily doses of ICL670 were taken orally 30 minutes before breakfast. The doses range from 5-40 mg/kg and were determined based on the patient's trend in serum ferritin over time during the core study (0109) and on the frequency of blood transfusions the patient received. The treatment duration was up to 4 years. | Drug | ICL670 |
Primary outcome
Number of Participants With Adverse Events After Start of ICL670
Time frame: 0 - 60 months
Safety as assessed by the number of participants with adverse event or death after the start of ICL670.
1 secondary outcome
Change in Serum Ferritin From Start of ICL670 to End of Study
Time frame: 0 - 60 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jun 2011.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
39 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ICL670 in people with Sickle Cell Disease, run by Novartis Pharmaceuticals at 39 sites in 5 countries.
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 7 inclusion and 8 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 6 Jun 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 39 sites in United States, Italy, France, Canada, United Kingdom. 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. 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?
Yes. Results are posted in the registry (first posted 20 Jan 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Adverse Events After Start of ICL670 (time frame: 0 - 60 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 6 Jun 2011.