Beta-thalassemiaPhase 2NCT00061763
Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias
A Phase 2 study testing Deferasirox in people with Beta-thalassemia and Myelodysplastic Syndromes, run by Novartis Pharmaceuticals at 6 sites in United States.
Trial summary
The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with 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
- Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome
- What is being tested?
- Deferasirox
- How many people
- 175 participants
- Where
- 6 sitesUnited States
- Study dates
- May 2003to Nov 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Beta-thalassemia patients with documented non-compliance to deferoxamine, defined as taking less than 50% of prescribed doses in year prior to study, and having a liver iron content at least 14 mg iron/gm dry weight liver tissue
- Beta-thalassemia patients unable to take deferoxamine because of documented side effects or contra-indication, or documented poor response despite proper compliance, with liver iron content at least 2 mg iron/gm dry weight liver tissue
- Patients with chronic anemias with a liver iron content at least 2 mg/gm dry weight liver tissue.
- Beta-thalassemia or other chronic anemia patients having previously taken deferiprone, provided that they stop the deferiprone at least 28 days before the study and have a liver iron content at least 2 mg/gm dry weight liver tissue.
- All patients: Regular transfusions indicated by a requirement of at least 8 blood transfusions per year.
- Life expectancy of at least one year.
Who cannot join
- Beta-thalassemia able to be treated with deferoxamine, Sickle Cell Disease or non-transfusional iron overload
- Elevated liver enzymes in the year preceding enrollment
- Active Hepatitis B or Hepatitis C
- HIV seropositivity
- Elevated serum creatinine or significant proteinuria
- History of nephrotic syndrome
Show all 19Show fewer
- Uncontrolled systemic hypertension
- Fever and other signs/symptoms of infection within 10 days prior to start of the study.
- Presence of clinically relevant cataract or previous history of clinically relevant ocular toxicity related to iron chelation.
- Second or third degree AV block, clinically relevant Q-T interval prolongation, or patients requiring digoxin or other drugs that prolong the Q-T interval.
- Diseases (cardiovascular, renal, hepatic, etc.) that would prevent the patient from undergoing any of the treatment options.
- Psychiatric or additive disorders that would prevent the patient from giving informed consent.
- History of drug or alcohol abuse within the 12 months prior to the study.
- Pregnant or breast feeding patients.
- Patients treated with systemic investigational drugs within 4 weeks or topical investigational drugs within 7 days before the start of teh study.
- Any surgical or medical condition that might significantly alter the absorption, distribution, metabolism or excretion of any drug, such as gastrointestinal disease or major surgery, renal disease, difficulty voiding or urinary obstruction, or impaired pancreatic function.
- Non-compliant or unreliable patients
- Patients unable to undergo any study procedures such as the hearing or eye tests, or the liver echocardiography.
- Patients that would need a dose of Deferasirox less than 125 mg per day.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Northwest Medical Specialists
Arlington Heights, Illinois, 60004
Children's Hospital Boston
Boston, Massachusetts, 02115
Weill Medical College of Cornell University
New York, New York, 10021
Children's Hospital Oakland
Oakland, California, 94609
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4318
Stanford Hospital
Stanford, California, 94305-5208
About this study
Official title: Phase II Study of Safety & Efficacy of Deferasirox Given for 1 Year in Patients With Chronic Anemias and Transfusional Hemosiderosis Unable to be Treated With Deferoxamine
The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with iron overload from repeated blood transfusions. Beta-thalassemia patients unable to be treated with deferoxamine or patients with rare chronic anemias such as Myelodysplastic Syndrome, Fanconi's Syndrome, Blackfan-Diamond Syndrome, and Pure Red Blood Cell Anemia are eligible for this study. Liver iron content will be measured by liver biopsy at the beginning of the study and after one year of treatment. However, those patients living in the San Francisco/Oakland area may have a SQUID in place of the liver biopsy if the biopsy is not medically possible for them. The SQUID is a non-invasive magnetic means to measure liver iron content.
- Conditions
- Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan, Anemia, Aplastic
- Being tested
- Deferasirox
- Keywords
- beta-thalassemia, iron overload, deferoxamine, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan, Anemia, Aplastic
- Medical subject headings
- beta-Thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan, Anemia, Aplastic, Iron Overload
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DeferasiroxDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the effects of treatment on the liver iron content(LIC)
Secondary outcomes (4)
Evaluate tolerability profile
Estimate the absolute and relative change of LIC and total body iron excretion (TBIE) rate
Evaluate the relationship between LIC and potential surrogate markers
Evaluate the relationship between PD and safety variables
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jun 2003 (estimated)
- Study startMay 2003
- Primary completionNov 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta-thalassemiaStudies 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
- 175 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
- NCT00061763
- Sponsor's protocol ID
- CICL670A0108
- Status last verified
- Aug 2017
- First submitted
- 3 Jun 2003
- Last updated
- 22 Aug 2017
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of Safety & Efficacy of Deferasirox Given for 1 Year in Patients With Chronic Anemias and Transfusional Hemosiderosis Unable to be Treated With Deferoxamine
- ClinicalTrials.gov ID
- NCT00061763
- Acronym / sponsor ID
- CICL670A0108
- Conditions
- Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan, Anemia, Aplastic
- Keywords
- beta-thalassemia, iron overload, deferoxamine, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan, Anemia, Aplastic
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 175 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- May 2003
- Primary completion
- Nov 2004 (actual)
- First posted
- 4 Jun 2003 (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.
The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with iron overload from repeated blood transfusions. Beta-thalassemia patients unable to be treated with deferoxamine or patients with rare chronic anemias such as Myelodysplastic Syndrome, Fanconi's Syndrome, Blackfan-Diamond Syndrome, and Pure Red Blood Cell Anemia are eligible for this study. Liver iron content will be measured by liver biopsy at the beginning of the study and after one year of treatment. However, those patients living in the San Francisco/Oakland area may have a SQUID in place of the liver biopsy if the biopsy is not medically possible for them. The SQUID is a non-invasive magnetic means to measure liver iron content.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Beta-thalassemia patients with documented non-compliance to deferoxamine, defined as taking less than 50% of prescribed doses in year prior to study, and having a liver iron content at least 14 mg iron/gm dry weight liver tissue
- Beta-thalassemia patients unable to take deferoxamine because of documented side effects or contra-indication, or documented poor response despite proper compliance, with liver iron content at least 2 mg iron/gm dry weight liver tissue
- Patients with chronic anemias with a liver iron content at least 2 mg/gm dry weight liver tissue.
- Beta-thalassemia or other chronic anemia patients having previously taken deferiprone, provided that they stop the deferiprone at least 28 days before the study and have a liver iron content at least 2 mg/gm dry weight liver tissue.
- All patients: Regular transfusions indicated by a requirement of at least 8 blood transfusions per year.
Key exclusion criteria (19 listed)
- Beta-thalassemia able to be treated with deferoxamine, Sickle Cell Disease or non-transfusional iron overload
- Elevated liver enzymes in the year preceding enrollment
- Active Hepatitis B or Hepatitis C
- HIV seropositivity
- Elevated serum creatinine or significant proteinuria
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 | Drug | – |
Primary outcome
To evaluate the effects of treatment on the liver iron content(LIC)
4 secondary outcomes
Evaluate tolerability profile
Estimate the absolute and relative change of LIC and total body iron excretion (TBIE) rate
Evaluate the relationship between LIC and potential surrogate markers
Evaluate the relationship between PD and safety variables
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 22 Aug 2017.
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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
6 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 Deferasirox in people with Beta-thalassemia and Myelodysplastic Syndromes, run by Novartis Pharmaceuticals at 6 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 6 inclusion and 19 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 22 Aug 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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. 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?
To evaluate the effects of treatment on the liver iron content(LIC). 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 22 Aug 2017.