Non-transfusion Dependent ThalassemiaPhase 2THALASSANCT00873041
Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study
A Phase 2 study testing deferasirox in people with Non-transfusion Dependent Thalassemia, run by Novartis Pharmaceuticals at 21 sites in 9 countries.
Trial summary
CICL670A2209: This study will evaluate the safety and efficacy of deferasirox in non-transfusion dependent thalassemia patients with iron overload. Patients will be treated either with active treatment (deferasirox) or placebo for 12 months (core study phase). Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Non-transfusion Dependent Thalassemia
- What is being tested?
- deferasirox
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 166 participants
- Where
- 21 sitesin 9 countries
- Study dates
- Nov 2008to Jun 2012
- 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 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Core Inclusion Criteria:
- Male or female aged ≥ 10 years with non-transfusion dependent syndromes, not requiring transfusion within 6 months prior to study start. Note: there was a local country amendment for Greece only to change the age specific inclusion criteria to ≥ 18 years old
- Liver iron concentration ≥ 5 mg/g dry weight measured by Magnetic resonance imaging (MRI) before study start
- Serum ferritin >300 ng/mL at screening
Core Exclusion Criteria:
- Hemoglobin S (HbS)-variants of thalassemia syndromes
- Anticipated regular transfusion program during the study. Patients having a sporadic transfusion (e.g. in case of infection) throughout the study course will not be excluded
- Any blood transfusion 6 months prior to study start
- Creatinine clearance ≤ 60 mL/min at screening
- Serum creatinine above the upper limit of normal at both screening visits
- Significant proteinuria as indicated by a urine protein/urine creatinine ratio > 1.0 mg/mg
- Alanine aminotransferase (ALT) of > 5 x the upper limit of normal at both screening visits
- Concomitant therapy with hydroxyurea, erythropoietin, butyrate
- History of deferasirox treatment
- Pediatric patients: a patient's weight of below 20 kg
Extension Inclusion Criteria:
- Patients who completed the core CICL670A2209 clinical trial
- Written informed consent obtained prior entry to one year extension study CICL670A2209
Extension Exclusion Criteria:
- Patients with a continuous increase in serum creatinine ≥ 33% above the baseline value and > ULN who did not improve after drug interruption or dose reduction in the core study
- Patients with a continuous increase in ALT greater than 2 times the baseline value and > 5 times ULN who did not improve after drug interruption or dose reduction in the core study
- Patients with progressive proteinuria, as assessed by the investigator, who did not improve after drug interruption or dose reduction in the core study
- Significant medical condition interfering with the ability to partake in this study (e.g.systemic uncontrolled hypertension, unstable cardiac disease not controlled by standard medical therapy, systemic disease (cardiovascular, renal, hepatic, etc.)
Other protocol-defined inclusion/exclusion criteria may apply
Trial locations and contacts
21 sites in 9 countries.
21 sites in 9 countries
Italy5 sites
Novartis Investigative Site
Cagliari
Novartis Investigative Site
Genova
Novartis Investigative Site
Milan
Novartis Investigative Site
Naples
Novartis Investigative Site
Rome
Turkey (Türkiye)4 sites
Novartis Investigative Site
Adana
Novartis Investigative Site
Ankara
Novartis Investigative Site
Istanbul
Novartis Investigative Site
Izmir
United States3 sites
Children's Memorial Hospital/Division of Hematology/Oncology
Chicago, Illinois, 60614-3394
New York Presbyterian Hospital/Weill Medical College of Cornell University
New York, New York, 10021
Children's Hospital & Research Center Oakland
Oakland, California, 94609-1809
Greece3 sites
Novartis Investigative Site
Athens
Novartis Investigative Site
Pátrai
Novartis Investigative Site
Thessaloniki
Malaysia2 sites
Novartis Investigative Site
Ampang Selangor
Novartis Investigative Site
Kuala Lumpur
About this study
Official title: A Randomized, Double-blind, Placebo-controlled, Phase II Study to Evaluate Efficacy and Safety of Deferasirox in Non-transfusion-dependent Thalassemia Patients With Iron Overload
CICL670A2209: This study will evaluate the safety and efficacy of deferasirox in non-transfusion dependent thalassemia patients with iron overload. Patients will be treated either with active treatment (deferasirox) or placebo for 12 months (core study phase). Patients who complete the core study phase will be offered to continue their study with the active treatment (deferasirox) in a 12 months extension phase. During the core and extension, the effects of treatment on iron overload in the liver will be evaluated using magnetic resonance imaging (MRI) assessments.
CICL670A2209E1: A one-year open-label extension to a randomized, double-blind, placebo-controlled, phase II study to evaluate efficacy and safety of deferasirox in non-transfusion dependent thalassemia patients with iron overload (Thalassa).
- Conditions
- Non-transfusion Dependent Thalassemia
- Being tested
- deferasirox, placebo
- Keywords
- Thalassemia, thalassemia intermedia, alpha-thalassemia, beta-thalassemia, deferasirox, iron overload, non-transfusion dependent
- Medical subject headings
- Thalassemia, beta-Thalassemia, alpha-Thalassemia, Iron Overload
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
deferasiroxDrug
Supplied as 125 mg, 250 mg and 500 mg tablets.
placeboDrug
Supplied as matching 125 mg, 250 mg and 500 mg tablets.
| Study group | Role | What they receive |
|---|---|---|
| 5 mg/kg/day deferasirox | Experimental | Participants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks. Receives: Drug: deferasirox; Drug: placebo |
| 10 mg/kg/day deferasirox | Experimental | Participants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks. Receives: Drug: deferasirox; Drug: placebo |
| 5 mg/kg/day placebo | Placebo comparator | Placebo tablet matching 5 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks. Receives: Drug: placebo |
| 10 mg/kg/day placebo | Placebo comparator | Placebo tablet matching 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks. Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 52
LIC was measured by magnetic resonance imaging technique at baseline and Week 52. Estimates were obtained from an Analysis of Covariance (ANCOVA) model for change in LIC between baseline and Week 52 with treatment as factor and baseline LIC as covariate.
Measured: Baseline, Week 52
Extension Study: Percentage of Participants Reaching a Liver Iron Concentration (LIC) < 5 mg Fe/g dw From Core Baseline to End of Extension Study
Liver iron concentration was measured at Core Baseline and at the end of the Extension Study. Magnetic Resonance Imaging (MRI) scans were analyzed at a central laboratory to determine the LIC value. The percentage of participants with LIC < 5 mgFe/g dw (milligram iron/gram dry weight) change from Baseline at the end of the Extension Study is reported.
Measured: Core Baseline to End of Extension Study (up to 24 months)
Secondary outcomes (18)
Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 24
LIC was measured by magnetic resonance imaging technique at baseline and Week 24. Estimates were obtained from an Analysis of Covariance (ANCOVA) model for change in LIC between baseline and Week 24 with treatment as factor and baseline LIC as covariate.
Measured: Baseline, Week 24
Core Study: Change in Serum Ferritin Between Baseline and Fourth Quarter
Baseline serum ferritin average was the average of all available ferritin values from screening to last sample prior to the first intake of study drug.
Fourth quarter serum ferritin average was the average of all serum ferritin values obtained within days 286- End of Study.
Change from baseline: fourth quarter serum ferritin average - baseline serum ferritin average.
Measured: Baseline, (Day 286 to End of Study [Day 365])
Core Study: Change in Serum Ferritin Between Baseline and Second Quarter
Baseline serum ferritin average was the average of all available ferritin values from screening to last sample prior to the first intake of study drug.
Second quarter serum ferritin average was the average of all serum ferritin values obtained within days 106-195.
Change from baseline: second quarter serum ferritin average - baseline serum ferritin average.
Measured: Baseline, (Day 106 to Day 195)
Core Study: Percentage of Participants With Adverse Events Graded Mild, Moderate and Severe
Percentage of Participants with Mild, Moderate and Severe adverse events (AE) any primary system organ class regardless of study drug relationship. A patient with multiple occurrences of an AE is counted only once in the AE category for that treatment. A patient with multiple severity ratings for an AE while on a treatment is only counted once under the maximum rating.
Measured: 52 Weeks
Core Study: Change in Liver Iron Concentration (LIC) From Baseline At Week 24 and Week 52 in Patients With Dose Increases After Week 24
LIC was measured by magnetic resonance imaging technique at baseline, Week 24 and Week 52. Dose Doubling (Dose Increases) began at Week 24.
Measured: Baseline, Week 24, Week 52
Core Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration)
The correlation between serum ferritin and LIC was investigated using a scatter plot with a regression line for the following cases:
- Baseline serum ferritin versus baseline LIC
- Serum ferritin difference from baseline at fourth quarter versus difference from baseline in LIC at Week 52.
A value of 1.0 indicates a perfect correlation.
Measured: Baseline, 52 weeks
Core Study: Change From Baseline in Hemoglobin at Month 12
Blood was collected for Hemoglobin at baseline and Month 12. Change from baseline= Month 12 hemoglobin - baseline hemoglobin.
Measured: Baseline, Month 12
Core Study: Change From Baseline in Transferrin Saturation at Month 12
Blood was collected for transferrin saturation at Baseline and Month 12. Change from baseline= Month 12 transferrin saturation - baseline transferrin saturation.
Measured: Baseline, Month 12
Core Study: Change in Liver Iron Concentration (LIC) in Placebo Patients From Baseline to Week 52
LIC was measured by magnetic resonance imaging technique at baseline and Week 52. The change in liver iron concentration for participants in the placebo arm was used to assess the iron accumulation rate.
Measured: Baseline, Week 52
Core Study: Percentage of Participants With Notable Abnormal Post-baseline Laboratory Results
The percentage of participants with notable laboratory results:
Platelet count: (<100 x 10^9/L)
Absolute neutrophils: (<1.5 x 10^9/L)
Alanine aminotransferase (ALT): (>5 x Upper limit normal (ULN) and >2 x baseline).
Aspartate aminotransferase (AST): (>5 x ULN and >2 x baseline)
Serum creatinine: (>33% increase from baseline and >ULN at ≥2 consecutive post-baseline values) Creatinine clearance: (<60 mL/min at ≥2 consecutive post-baseline values)
Urinary protein/creatinine ratio: (≥ 1.0 mg/mg at ≥2 consecutive post-baseline values)
Measured: 52 Weeks
Core Study: Percentage of Participants With Notably Abnormal Post-baseline Systolic Blood Pressure
Systolic blood pressure was measured at each visit after the patient rested in the sitting position for at least 3 minutes.
A Notably Abnormal Systolic Blood Pressure was defined as a measurement in one of the following two categories:
High: ≥180 with an increase from baseline ≥20 mmHg
Low: ≤90 with a decrease from baseline ≥20 mmHg
Measured: Baseline, 52 Weeks
Core Study: Percentage of Participants With Notably Abnormal Post-baseline Diastolic Blood Pressure
Diastolic blood pressure was measured at each visit after the patient rested in the sitting position for at least 3 minutes.
A Notably Abnormal Diastolic Blood Pressure was defined as a measurement in one of the following two categories:
High: ≥105 with an increase from baseline ≥15 mmHg
Low: ≤50 with a decrease from baseline ≥15 mmHg
Measured: Baseline, 52 Weeks
Core Study: Percentage of Participants With Notably Abnormal Post-baseline Pulse Rate
Pulse Rate was measured at each visit.
A Notably Abnormal Pulse Rate was defined as a measurement in one of the following two categories:
High: ≥120 with an increase from baseline ≥15 beats per minute (bpm)
Low: ≤50 with a decrease from baseline ≥15 bpm
Measured: Baseline, 52 Weeks
Extension Study: Absolute Change in Serum Ferritin From Baseline to Eighth Quarter
Blood was collected for serum ferritin at Core Baseline and monthly during the Eighth quarter of the Extension Study. Absolute change from Baseline: quarterly average - baseline average. A negative change from baseline indicated improvement.
Measured: Core Baseline, Eighth Quarter (last 3 months of the study)
Extension Study: Change in Liver Iron Concentration (LIC) From Baseline at Month 24
LIC was measured by magnetic resonance imaging technique at Baseline and Month 24. A negative change from baseline indicated improvement.
Measured: Core Baseline, Month 24
Extension Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration)
The correlation between serum ferritin and LIC was investigated using a scatter plot with a regression line for serum ferritin difference from Baseline at Month 24 versus LIC difference from Baseline at Month 24.
A value of 1.0 indicates a perfect correlation.
Measured: Core Baseline, Month 24
Extension Study: Change From Baseline in Hemoglobin at Month 24
Blood was collected for Hemoglobin at Baseline and Month 24. Change from Baseline= Month 24 hemoglobin - Baseline hemoglobin.
Measured: Core Baseline, Month 24
Extension Study: Change From Baseline in Transferrin Saturation at Month 24
Blood was collected for transferrin saturation at Baseline and Month 24. Change from baseline= Month 24 transferrin saturation - baseline transferrin saturation.
Measured: Core Baseline, Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Apr 2009 (estimated)
- Study startNov 2008
- Primary completionJun 2011
- Study completionJun 2012
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.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Non-transfusion Dependent 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 166 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00873041
- Sponsor's protocol ID
- CICL670A2209
- Other IDs
- EudraCT 2007-007000-15
- Acronym
- THALASSA
- Status last verified
- May 2013
- First submitted
- 30 Mar 2009
- Last updated
- 9 Jul 2013
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Taher AT, Porter JB, Viprakasit V, Kattamis A, Chuncharunee S, Sutcharitchan P, Siritanaratkul N, Origa R, Karakas Z, Habr D, Zhu Z, Cappellini MD. Defining serum ferritin thresholds to predict clinically relevant liver iron concentrations for guiding deferasirox therapy when MRI is unavailable in patients with non-transfusion-dependent thalassaemia. Br J Haematol. 2015 Jan;168(2):284-90. doi: 10.1111/bjh.13119. Epub 2014 Sep 12.
PubMed 25212456Taher AT, Porter JB, Viprakasit V, Kattamis A, Chuncharunee S, Sutcharitchan P, Siritanaratkul N, Galanello R, Karakas Z, Lawniczek T, Habr D, Ros J, Zhang Y, Cappellini MD. Deferasirox demonstrates a dose-dependent reduction in liver iron concentration and consistent efficacy across subgroups of non-transfusion-dependent thalassemia patients. Am J Hematol. 2013 Jun;88(6):503-6. doi: 10.1002/ajh.23445. Epub 2013 May 13.
PubMed 23553596
Study at a glance
From the registry record, last updated by the sponsor on 9 Jul 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled, Phase II Study to Evaluate Efficacy and Safety of Deferasirox in Non-transfusion-dependent Thalassemia Patients With Iron Overload
- ClinicalTrials.gov ID
- NCT00873041
- Acronym / sponsor ID
- THALASSA / CICL670A2209
- Conditions
- Non-transfusion Dependent Thalassemia
- Keywords
- Thalassemia, thalassemia intermedia, alpha-thalassemia, beta-thalassemia, deferasirox, iron overload, non-transfusion dependent
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 166 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Nov 2008
- Primary completion
- Jun 2011 (actual)
- Study completion
- Jun 2012 (actual)
- First posted
- 1 Apr 2009 (estimated)
- Results
- Posted 25 Sep 2012
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
CICL670A2209: This study will evaluate the safety and efficacy of deferasirox in non-transfusion dependent thalassemia patients with iron overload. Patients will be treated either with active treatment (deferasirox) or placebo for 12 months (core study phase). Patients who complete the core study phase will be offered to continue their study with the active treatment (deferasirox) in a 12 months extension phase. During the core and extension, the effects of treatment on iron overload in the liver will be evaluated using magnetic resonance imaging (MRI) assessments.
CICL670A2209E1: A one-year open-label extension to a randomized, double-blind, placebo-controlled, phase II study to evaluate efficacy and safety of deferasirox in non-transfusion dependent thalassemia patients with iron overload (Thalassa).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 5 mg/kg/day deferasirox | Experimental | Drug: deferasirox, Drug: placeboParticipants received a starting dose of 5 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks. |
| 10 mg/kg/day deferasirox | Experimental | Drug: deferasirox, Drug: placeboParticipants received a starting dose of 10 mg/kg/day deferasirox tablets orally each day in the morning for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks. |
| 5 mg/kg/day placebo | Placebo comparator | Drug: placeboPlacebo tablet matching 5 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks. |
| 10 mg/kg/day placebo | Placebo comparator | Drug: placeboPlacebo tablet matching 10 mg/kg/day orally in the morning each day for 52 weeks. After 24 weeks of treatment Liver Iron Concentration (LIC) was assessed. Based on the LIC and change from baseline in LIC participants were eligible for dose escalation. In the Extension Study participants received deferasirox once daily (dose based on LIC) for 52 weeks. |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| deferasiroxSupplied as 125 mg, 250 mg and 500 mg tablets. | Drug | 10 mg/kg/day deferasirox, 5 mg/kg/day deferasirox |
| placeboSupplied as matching 125 mg, 250 mg and 500 mg tablets. | Drug | 10 mg/kg/day deferasirox, 10 mg/kg/day placebo, 5 mg/kg/day deferasirox, 5 mg/kg/day placebo |
Primary outcome
Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 52
Time frame: Baseline, Week 52
LIC was measured by magnetic resonance imaging technique at baseline and Week 52. Estimates were obtained from an Analysis of Covariance (ANCOVA) model for change in LIC between baseline and Week 52 with treatment as factor and baseline LIC as covariate.
Extension Study: Percentage of Participants Reaching a Liver Iron Concentration (LIC) < 5 mg Fe/g dw From Core Baseline to End of Extension Study
Time frame: Core Baseline to End of Extension Study (up to 24 months)
Liver iron concentration was measured at Core Baseline and at the end of the Extension Study. Magnetic Resonance Imaging (MRI) scans were analyzed at a central laboratory to determine the LIC value. The percentage of participants with LIC < 5 mgFe/g dw (milligram iron/gram dry weight) change from Baseline at the end of the Extension Study is reported.
18 secondary outcomes
Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 24
Time frame: Baseline, Week 24
Core Study: Change in Serum Ferritin Between Baseline and Fourth Quarter
Time frame: Baseline, (Day 286 to End of Study [Day 365])
Core Study: Change in Serum Ferritin Between Baseline and Second Quarter
Time frame: Baseline, (Day 106 to Day 195)
Core Study: Percentage of Participants With Adverse Events Graded Mild, Moderate and Severe
Time frame: 52 Weeks
Core Study: Change in Liver Iron Concentration (LIC) From Baseline At Week 24 and Week 52 in Patients With Dose Increases After Week 24
Time frame: Baseline, Week 24, Week 52
Core Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration)
Time frame: Baseline, 52 weeks
Core Study: Change From Baseline in Hemoglobin at Month 12
Time frame: Baseline, Month 12
Core Study: Change From Baseline in Transferrin Saturation at Month 12
Time frame: Baseline, Month 12
Core Study: Change in Liver Iron Concentration (LIC) in Placebo Patients From Baseline to Week 52
Time frame: Baseline, Week 52
Core Study: Percentage of Participants With Notable Abnormal Post-baseline Laboratory Results
Time frame: 52 Weeks
Core Study: Percentage of Participants With Notably Abnormal Post-baseline Systolic Blood Pressure
Time frame: Baseline, 52 Weeks
Core Study: Percentage of Participants With Notably Abnormal Post-baseline Diastolic Blood Pressure
Time frame: Baseline, 52 Weeks
Core Study: Percentage of Participants With Notably Abnormal Post-baseline Pulse Rate
Time frame: Baseline, 52 Weeks
Extension Study: Absolute Change in Serum Ferritin From Baseline to Eighth Quarter
Time frame: Core Baseline, Eighth Quarter (last 3 months of the study)
Extension Study: Change in Liver Iron Concentration (LIC) From Baseline at Month 24
Time frame: Core Baseline, Month 24
Extension Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration)
Time frame: Core Baseline, Month 24
Extension Study: Change From Baseline in Hemoglobin at Month 24
Time frame: Core Baseline, Month 24
Extension Study: Change From Baseline in Transferrin Saturation at Month 24
Time frame: Core Baseline, Month 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jul 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 25212456Taher AT, Porter JB, Viprakasit V, Kattamis A, Chuncharunee S, Sutcharitchan P, Siritanaratkul N, Origa R, Karakas Z, Habr D, Zhu Z, Cappellini MD. Defining serum ferritin thresholds to predict clinically relevant liver iron concentrations for guiding deferasirox therapy when MRI is unavailable in patients with non-transfusion-dependent thalassaemia. Br J Haematol. 2015 Jan;168(2):284-90. doi: 10.1111/bjh.13119. Epub 2014 Sep 12.
- Derived from this study: PubMed 23553596Taher AT, Porter JB, Viprakasit V, Kattamis A, Chuncharunee S, Sutcharitchan P, Siritanaratkul N, Galanello R, Karakas Z, Lawniczek T, Habr D, Ros J, Zhang Y, Cappellini MD. Deferasirox demonstrates a dose-dependent reduction in liver iron concentration and consistent efficacy across subgroups of non-transfusion-dependent thalassemia patients. Am J Hematol. 2013 Jun;88(6):503-6. doi: 10.1002/ajh.23445. Epub 2013 May 13.
- Visit NovartisClinicalTrials.com: Pre-qualify for a trial, and view a list of trials and participating study centers.Link provided by the sponsor.
Related trials for Non-transfusion Dependent Thalassemia
Active, not recruitingPhase 3
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
RecruitingPhase 3
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
Active, not recruitingPhase 2
A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia
Anemia
RecruitingObservational study
A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East
β-thalassemia
Active, not recruitingPhase 2
A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body
Non-Transfusion Dependent Beta-Thalassemia (NTDT)
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
21 sites in 9 countries: 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 Non-transfusion Dependent Thalassemia, run by Novartis Pharmaceuticals at 21 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 9 Jul 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in Italy, Turkey (Türkiye), United States, Greece, Malaysia and 4 more countries. 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?
Yes. Results are posted in the registry (first posted 25 Sep 2012). 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?
Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 52 (time frame: Baseline, Week 52). The study lists 2 primary outcomes in all. 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 NCT00873041, which also lists 2 linked publications.
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 9 Jul 2013.