Sickle Cell DiseasePhase 2NCT00110617
Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients
A Phase 2 study testing Deferasirox (ICL670) and Deferoxamine (DFO) in people with Sickle Cell Disease and Iron Overload, run by Novartis Pharmaceuticals at 59 sites in 2 countries.
Trial summary
This study will examine the long-term safety and efficacy of Deferasirox in patients with sickle cell disease and iron overload from repeated blood transfusions. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Sickle Cell Disease, Iron Overload, Hemolytic Anemia
- What is being tested?
- Deferasirox (ICL670) and Deferoxamine (DFO)
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 212 participants
- Where
- 59 sitesin 2 countries
- Study dates
- May 2005to Apr 2008
- 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
- Age greater than or equal to 2 years
- Male or female patients with sickle cell disease (SS, SC, SD, Sβo or Sβ+ thalassemia)
- Iron overload from repeated blood transfusion, as defined by one of the following:
- For patients > 16 years old receiving simple transfusions: lifetime history of receipt of at least 120 ml/kg or 30 adult units of packed red blood cells, OR
- For patients ≤ 16 years old receiving simple transfusions: lifetime history of receipt of at least 120 ml/kg of packed red blood cells, OR
- For all patients receiving exchange transfusions in the absence of a previous attempt to achieve negative iron balance: lifetime performance of at least 20 procedures, OR
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- For all patients: liver iron content ≥ 7 mg Fe/g dry weight as measured by biopsy, Magnetic Resonance Imaging (MRI), or magnetic susceptibility performed within 3 months prior to entry into screening
- For entry into the screening period: serum ferritin of ≥ 1000 µg/mL on at least two occasions during the prior year obtained in the absence of concomitant infection.
- Body weight > 10 kg
- No known allergy or contraindication to the administration of deferoxamine
- Ability to comply with all study-related procedures, medications, and evaluations
- Sexually active pre-menopausal 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 by the patient or for pediatric patient's consent of the patient's legal guardian. The definition of the term 'pediatric' for enrollment and study conduct will be in accordance with the local legislation.
Who cannot join
- Serum creatinine above the upper limit of normal
- Significant proteinuria
- History of nephrotic syndrome
- Alanine aminotransferase (ALT) ≥ 250 U/L at screening
- Clinical evidence of active hepatitis B or hepatitis C
- History of HIV
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- Fever or other signs/symptoms of infection within 10 days prior to the screening visit
- Uncontrolled systemic hypertension
- History of Myocardial Infarction, Congestive Heart Failure or unstable cardiac disease not controlled by standard medical therapy
- Clinically relevant cataract or a previous history of clinically relevant ocular toxicity related to iron chelation
- Presence of a surgical or medical condition that might significantly alter the absorption, distribution, metabolism or excretion of any study drug
- History of drug or alcohol abuse within the 12 months prior to enrollment
- Pregnant or breast feeding patients
- Patients treated with systemic investigational drug within 4 weeks prior or with topical investigational drug 7 days prior to the screening visit
- Randomization in a previous clinical trial involving ICL670
- Other protocol-related inclusion / exclusion criteria may apply.
Trial locations and contacts
59 sites in 2 countries.
59 sites in 2 countries
United States56 sites
University of Michigan
Ann Arbor, Michigan, 48109-0238
Emory University School of Medicine
Atlanta, Georgia, 30303
Children's Healthcare of Atlanta at Scottish Rite
Atlanta, Georgia, 30342
Adult Sickle Cell Clinic
Augusta, Georgia, 30912-3128
St. Jude Children's Hospital Affiliate
Baton Rouge, Louisiana, 70808
University of Alabama Pediatric Hematology/Oncology
Birmingham, Alabama, 35233
University of Alabama Medical center
Birmingham, Alabama, 35294
Brigham and Woman's Hospital/Harvard Medical School
Boston, Massachusetts, 02115
Children's Hospital Boston
Boston, Massachusetts, 02115
Children's Hospital
Boston, Massachusetts, 02115
SUNY Downstate Medical Center
Brooklyn, New York, 11203
New York Methodist Hospital
Brooklyn, New York, 11215
Carolinas Medical Transplant Center
Charlotte, North Carolina, 28232
University of Illinois at Chicago
Chicago, Illinois, 60612
Children's Memorial Hospital
Chicago, Illinois, 60614-3394
University of Cincinnati
Cincinnati, Ohio, 45219
Children's Hospital Medical Center
Cincinnati, Ohio, 72764
Liberty Hematology Oncology Center
Columbia, South Carolina, 29203
Palmetto Health Clinical Trials
Columbia, South Carolina, 29203
Karmanos Cancer Institute
Detroit, Michigan, 48201
Cooks Children's Hospital
Fort Worth, Texas, 76104-2724
Texas Children's Hospital/Baylor College of Medicine
Houston, Texas, 77030-2399
Pediatric Sickle Cell Program/James Whitcomb Riley Hospital for Children
Indianapolis, Indiana, 46202
Loma Linda University Medical Center
Loma Linda, California, 92354
St Jude's Children's Research Hospital
Memphis, Tennessee, 38105-2794
St. Jude's Children Research Hospital
Memphis, Tennessee, 38105
Miami Children's Hospital
Miami, Florida, 33155
University of South Alabama Medical Center
Mobile, Alabama, 36604
University of South Alabama
Mobile, Alabama, 36617
Tulane University Sickle Cell Center
New Orleans, Louisiana, 70112
Children's Hospital
New Orleans, Louisiana, 70118
Weill Medical College of Cornell University
New York, New York, 10021
Columbia University
New York, New York, 10032
Children's Hospital of the King's Daughter
Norfolk, Virginia, 23507
Children's Hospital Oakland
Oakland, California, 94609
The University of Oklahoma
Oklahoma City, Oklahoma, 73104
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4399
Pennsylvania Oncology/Hematology
Philadelphia, Pennsylvania, 19106
Jefferson University
Philadelphia, Pennsylvania, 19107
Drexel University College of Medicine
Philadelphia, Pennsylvania, 19134-1095
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Hillman Cancer Center
Pittsburgh, Pennsylvania, 15232
Medical College of Virginia
Richmond, Virginia, 23298-0306
Virginia Commonwealth University
Richmond, Virginia, 23298-0646
Backus Children's Hospital
Savannah, Georgia, 31403
LSU Health Sciences Center/Carroll W. Feist Professor of Cancer Research
Shreveport, Louisiana, 71130
Washington University School of Medicine
St Louis, Missouri, 63110
Santee Hematology/Oncology
Sumter, South Carolina, 29150
Tampa Children's Hospital at St Joseph's
Tampa, Florida, 33607-6387
Tampa Children's Hospital at St. Joseph's
Tampa, Florida, 33607-6387
H. Lee Muffit Cancer Center and Research Institute/James A. Haley Veterans Hospital
Tampa, Florida, 33612
Scott and White Memorial Hospital & Clinics
Temple, Texas, 76508
Sickle Cell Center, Montefiore Hospital
The Bronx, New York, 10467-2490
Center for Cancer and Blood Disorders
Washington D.C., District of Columbia, 20010-2970
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Howard University Hospital
Washington D.C., District of Columbia, 20059
Canada3 sites
University of Alberta
Edmonton, Alberta, T6G 2H7
Hopital Ste-Justine
Montreal, Quebec, H3T 1C5
The Ottawa Hospital
Ottawa, Ontario, K1H 8L6
About this study
Official title: A Randomized, Open-label, Multi-center, Phase II Study to Evaluate the Safety and Efficacy of Deferasirox (ICL670) 20 mg/kg/Day Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients With Iron Overload From Repeated Blood Transfusions
This study will examine the long-term safety and efficacy of Deferasirox in patients with sickle cell disease and iron overload from repeated blood transfusions.
- Conditions
- Sickle Cell Disease, Iron Overload, Hemolytic Anemia
- Being tested
- Deferasirox (ICL670), Deferoxamine (DFO)
- Keywords
- Sickle Cell Disease, Iron Overload from Repeated Blood Transfusions, Iron Overload, Blood Transfusions
- Medical subject headings
- Anemia, Sickle Cell, Iron Overload, Anemia, Hemolytic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Deferasirox (ICL670)Drug
Deferasirox was provided in 125 mg, 250 mg, and 500 mg dispersible tablets and was administered orally at an initial dose of 20 mg/kg/day.
Deferoxamine (DFO)Drug
Deferoxamine was supplied in vials of 500 mg and 2000 mg administered subcutaneously for a weekly dose of 175 mg/kg.
| Study group | Role | What they receive |
|---|---|---|
| Deferasirox (ICL670) | Experimental | Deferasirox (ICL670) 20 mg/kg orally once daily for 104 weeks. Receives: Drug: Deferasirox (ICL670) |
| Deferoxamine (DFO) then ICL670 | Experimental | Deferoxamine (DFO) subcutaneously for a weekly dose of 175 mg/kg for 24 weeks then crossed over to receive Deferasirox (ICL670) orally 20 mg/kg for a total of 104 weeks on therapy. Receives: Drug: Deferasirox (ICL670); Drug: Deferoxamine (DFO) |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
The number of participants with Adverse Events (AEs) overall and according to Medical Dictionary for Regulatory Activities (MedDRA) preferred term greater than or equal to 5% participants in any group by treatment in the first 24 weeks.
Measured: 24 Weeks
Secondary outcomes (3)
Absolute Change in Serum Ferritin From Baseline to Week 24
Absolute change from baseline serum ferritin after 24 weeks of treatment with Deferasirox (ICL670) and absolute change from baseline serum ferritin after 24 weeks of treatment with Deferoxamine. Means were adjusted for the amount of transfused blood.
Measured: Baseline, 24 Weeks
Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 24 and to Week 52
Absolute change in serum ferritin after start of treatment with Deferasirox (ICL670) to week 24 and the absolute change in serum ferritin after start of treatment with Deferasirox (ICL670) to week 52 for the Deferasirox treatment group and the Deferoxamine then Deferasirox treatment group. Means were adjusted for the amount of transfused blood.
Measured: Start of Deferasirox (ICL670) treatment, 24 Weeks, 52 Weeks
Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 104
Absolute change in serum ferritin after start of treatment with Deferasirox (ICL670) to week 104 for the Deferasirox treatment group. Means were adjusted for the amount of transfused blood.
Measured: Start of Deferasirox (ICL670) treatment, 104 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 May 2005 (estimated)
- Study startMay 2005
- Primary completionApr 2008
- Study completionApr 2008
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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 212 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
- NCT00110617
- Sponsor's protocol ID
- CICL670A2201
- Status last verified
- May 2011
- First submitted
- 10 May 2005
- Last updated
- 26 May 2011
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 26 May 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Open-label, Multi-center, Phase II Study to Evaluate the Safety and Efficacy of Deferasirox (ICL670) 20 mg/kg/Day Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients With Iron Overload From Repeated Blood Transfusions
- ClinicalTrials.gov ID
- NCT00110617
- Acronym / sponsor ID
- CICL670A2201
- Conditions
- Sickle Cell Disease, Iron Overload, Hemolytic Anemia
- Keywords
- Sickle Cell Disease, Iron Overload from Repeated Blood Transfusions, Iron Overload, Blood Transfusions
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 212 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- May 2005
- Primary completion
- Apr 2008 (actual)
- Study completion
- Apr 2008 (actual)
- First posted
- 11 May 2005 (estimated)
- Results
- Posted 2 May 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will examine the long-term safety and efficacy of Deferasirox in patients with sickle cell disease and iron overload from repeated blood transfusions.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Deferasirox (ICL670) | Experimental | Drug: Deferasirox (ICL670)Deferasirox (ICL670) 20 mg/kg orally once daily for 104 weeks. |
| Deferoxamine (DFO) then ICL670 | Experimental | Drug: Deferasirox (ICL670), Drug: Deferoxamine (DFO)Deferoxamine (DFO) subcutaneously for a weekly dose of 175 mg/kg for 24 weeks then crossed over to receive Deferasirox (ICL670) orally 20 mg/kg for a total of 104 weeks on therapy. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (13 listed)
- Age greater than or equal to 2 years
- Male or female patients with sickle cell disease (SS, SC, SD, Sβo or Sβ+ thalassemia)
- Iron overload from repeated blood transfusion, as defined by one of the following:
- For entry into the screening period: serum ferritin of ≥ 1000 µg/mL on at least two occasions during the prior year obtained in the absence of concomitant infection.
- Body weight > 10 kg
Key exclusion criteria (16 listed)
- Serum creatinine above the upper limit of normal
- Significant proteinuria
- History of nephrotic syndrome
- Alanine aminotransferase (ALT) ≥ 250 U/L at screening
- Clinical evidence of active hepatitis B or hepatitis C
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 |
|---|---|---|
| Deferasirox (ICL670)Deferasirox was provided in 125 mg, 250 mg, and 500 mg dispersible tablets and was administered orally at an initial dose of 20 mg/kg/day. | Drug | Deferasirox (ICL670), Deferoxamine (DFO) then ICL670 |
| Deferoxamine (DFO)Deferoxamine was supplied in vials of 500 mg and 2000 mg administered subcutaneously for a weekly dose of 175 mg/kg. | Drug | Deferoxamine (DFO) then ICL670 |
Primary outcome
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
Time frame: 24 Weeks
The number of participants with Adverse Events (AEs) overall and according to Medical Dictionary for Regulatory Activities (MedDRA) preferred term greater than or equal to 5% participants in any group by treatment in the first 24 weeks.
3 secondary outcomes
Absolute Change in Serum Ferritin From Baseline to Week 24
Time frame: Baseline, 24 Weeks
Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 24 and to Week 52
Time frame: Start of Deferasirox (ICL670) treatment, 24 Weeks, 52 Weeks
Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 104
Time frame: Start of Deferasirox (ICL670) treatment, 104 Weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 May 2011.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Visit NovartisClinicalTrials.com: Pre-qualify for a trial, and view a list of trials and participating study centers.Link 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.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
59 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Deferasirox (ICL670) and Deferoxamine (DFO) in people with Sickle Cell Disease and Iron Overload, run by Novartis Pharmaceuticals at 59 sites in 2 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 13 inclusion and 16 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 26 May 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 59 sites in United States, Canada. 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 2 May 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?
The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment (time frame: 24 Weeks). 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 26 May 2011.