Thalassemia (Transfusion Delendent)Phase 4CENTAurusNCT02069886
Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent Thalassemia
A Phase 4 study testing deferasirox in people with Thalassemia (Transfusion Delendent), run by Novartis Pharmaceuticals.
Trial summary
The CENTAurus trial is a prospective clinical study designed to address systematically some of the relevant endocrine complications in an iron overloaded thalassemic population, primary objective being the assessment of the effect of deferasirox therapy on glucose metabolism/homeostasis. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Thalassemia (Transfusion Delendent)
- What is being tested?
- deferasirox
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Dec 2014to Dec 2018
- Phase
- Phase 4
- 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 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 1. Beta thalassemia major and severe intermedia patients transfusion dependent and with transfusional iron overload 2. Patients with diagnosis of impaired fasting glucose or impaired glucose tolerance 4.Patients naïve to deferasirox or patients who already receive deferasirox at sub-optimal doses 5.Cardiac MRI T2 >10 msec; 7.normal cardiac function (LVEF > 56%);
Who cannot join
- Non transfusional hemosiderosis;
- Patients with diabetes mellitus (genetic or secondary) or history of diabetes mellitus in 1st degree relatives;
- 4.Patients who received organ transplant; 5.Patients with galactose intolerance, severe lactase deficiency or glucose-galactose malabsorption; 6.Patients unable to tolerate (or who have unacceptable toxicities to) prior treatment with deferasirox; 7.History of hypersensitivity to the study drug or any of its excipients; 8. Renal impairment 10. Liver impairment; 11.Patients with active chronic hepatitis B infection, active hepatitis C infection;
- Other protocol-defined inclusion/exclusion criteria may apply" at the end
About this study
Official title: A Multicenter, Open-label, Single Arm, Interventional Phase IV Study, to Evaluate the Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent Thalassemia
The CENTAurus trial is a prospective clinical study designed to address systematically some of the relevant endocrine complications in an iron overloaded thalassemic population, primary objective being the assessment of the effect of deferasirox therapy on glucose metabolism/homeostasis. Other endocrine parameters complementary or supportive to the primary objective will be assessed and analyzed during this study. A number of lab parameters related to other axes of the endocrine system will be collected and analyzed.
- Conditions
- Thalassemia (Transfusion Delendent)
- Being tested
- deferasirox
- Keywords
- Endocrine complications, transfusion dependent thalassemia
- Medical subject headings
- Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
deferasiroxDrug
125, 250, 500 mg dispersable tablets
| Study group | Role | What they receive |
|---|---|---|
| deferasirox | Experimental | single arm. all patients will receive deferasirox Receives: Drug: deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline of glucose blood level measured after 2 h after receiving a glucose-equivalent oral challenge
The primary efficacy variable is the change (mg/dl) from baseline to 36 months of glucose plasma levels measured 2 hr post glusose equivalent oral challange.
After a 12-hour overnight fasting, at zero time (baseline) blood sample will be drawn and afterwards patients will receive a glucose-equivalent oral challenge (75 grams). After glucose loading plasma samples will be drawn at 30, 60, 90, 120 minutes for determination of plasma glucose. This will be repeated every 6 month till end of study
Measured: 36 months
Secondary outcomes (8)
Glucose of OGTT ( AUC)
change in of glucose metabolism during deferasirox treatment measuring the change versus baseline of 2-hr and fasting glucose plasma level of OGTT.After a 12-hour overnight fasting, at zero time (baseline) blood sample will be drawn and afterwards patients will receive a glucose-equivalent oral challenge (75 grams). After glucose loading plasma samples will be drawn at 30, 60, 90, 120 minutes for determination of plasma glucose . This will be repeated every 6 months till end of study
Measured: baseline and every 6 months measurement of 2hour Glocose of OGTT
change on insulin secretion and sensitivity
Change in insulin secretion and insulin sensitivity at every measurement versus screening. Stumvol Formula will be applied to values of 2hr glucose OCTT to calucolate insulin secretion and insulin sensitivity. After a 12-hour overnight fasting, at zero time (baseline) blood sample will be drawn and afterwards patients will receive a glucose-equivalent oral challenge (75 grams). After glucose loading plasma samples will be drawn at 30, 60, 90, 120 minutes for determination of insulin concentration.
Measured: baseline and every 6 months measurement of 2hr Glucose OGTT
Measurement of thyroid hormones TSH and FT4
Changes in plasma levels of TSH and FT4 at every measurement versus screening. Blood samples will be drawn from the patient at baseline and every 12 month till end of study and plansa concentration of thyroid hormons TSH and FT4 will be assessed.
Measured: baseline and every 12 months
Risk factors for the impairment of glucose homeostasis
information on age, sex, ethnicity, BMI, metabolic syndrome (hypertension, dyslipidemia, hypertrygliceridemia), chronic use of steroids, use of immunosuppressive drugs, growth hormonal deficit will be collected on a monthly basis till end of study
Measured: baseline and monthly till End of Study
Changes in endocrine funcionts parameters
- Female patients will be assessed for the presence or absence of menses every month versus baseline .Use of current hormonal replacing therapy, if any (e.g. thyroxin therapy for patients with non-compensated hypothyroidism, hormonal replacement drugs for hypogonadal patients) will be checked on a monthly basis versus baseline
Measured: baseline and monthly till EOS
Changes in parameters of bone metabolism
- Blood samples wiil be drawn to measure levels of serum calcium, phosphorus, alkaline phosphatase, vitamin D levels (25-hydroxycholecalciferol), serum calcium, parathormone (intact 1-84 PTH) from baseline to the end of study. Blood samples will be drawn to assess also Vitamin D and parathormone at baseline and then every six months. The intact parathormone will be assessed by evaluating the 1-84 PTH form. Inorganic phosphorus, serum calcium will be assessed at baseline and then monthly; alkaline phosphatase will be evaluated at baseline, every two weeks during the first month of treatment and monthly thereafter till EOS.
Measured: baseline, monthly or every 6 months till end of study
Iron overload status
Blood samples will be drawn to assess Serum Ferriting. Liver and cardiac iron will be assessed by MRI (MRI R2 annual measurements for liver, MRI T2* annual measurements for cardiac); -Relationship between changes in SF, LIC and cardiac T2* and changes in primary (OGTT) and secondary endpoints (glucose metabolism trend, insulin secretion and insulin sensitivity);
Measured: baseline and regularly till end of study (monthly or yearly as specified)
Safety of deferasirox therapy
Clinical and laboratory monitoring of AEs and SAEs (in particular changes in hepatic, renal, audiometric and ophthalmology parameters). Blood samples will be drawn to assess liver functions, renal funtions. Urine test will be performed to assess renal functions. An audiometric and ophalmologic visit will be done to assess eyes and ears functions.
Measured: baseline and at every scheduled visit (weekly for the first months or after dose escalation and monthly thereafter or yearly till EOS
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2014 (estimated)
- Study startDec 2014
- Primary completionDec 2018 (estimated)
- Study completionDec 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thalassemia (Transfusion Delendent)Studies 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 4
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 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
- NCT02069886
- Sponsor's protocol ID
- CICL670AIT12
- Acronym
- CENTAurus
- Status last verified
- Mar 2015
- First submitted
- 20 Feb 2014
- Last updated
- 20 Apr 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Apr 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-label, Single Arm, Interventional Phase IV Study, to Evaluate the Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent Thalassemia
- ClinicalTrials.gov ID
- NCT02069886
- Acronym / sponsor ID
- CENTAurus / CICL670AIT12
- Conditions
- Thalassemia (Transfusion Delendent)
- Keywords
- Endocrine complications, transfusion dependent thalassemia
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Dec 2014
- Primary completion
- Dec 2018 (estimated)
- Study completion
- Dec 2018 (estimated)
- First posted
- 24 Feb 2014 (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 CENTAurus trial is a prospective clinical study designed to address systematically some of the relevant endocrine complications in an iron overloaded thalassemic population, primary objective being the assessment of the effect of deferasirox therapy on glucose metabolism/homeostasis. Other endocrine parameters complementary or supportive to the primary objective will be assessed and analyzed during this study. A number of lab parameters related to other axes of the endocrine system will be collected and analyzed.
Study design
- Primary purpose
- Supportive care
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| deferasirox | Experimental | Drug: deferasiroxsingle arm. all patients will receive deferasirox |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- 1. Beta thalassemia major and severe intermedia patients transfusion dependent and with transfusional iron overload 2. Patients with diagnosis of impaired fasting glucose or impaired glucose tolerance 4.Patients naïve to deferasirox or patients who already receive deferasirox at sub-optimal doses 5.Cardiac MRI T2 >10 msec; 7.normal cardiac function (LVEF > 56%);
Key exclusion criteria (4 listed)
- Non transfusional hemosiderosis;
- Patients with diabetes mellitus (genetic or secondary) or history of diabetes mellitus in 1st degree relatives;
- 4.Patients who received organ transplant; 5.Patients with galactose intolerance, severe lactase deficiency or glucose-galactose malabsorption; 6.Patients unable to tolerate (or who have unacceptable toxicities to) prior treatment with deferasirox; 7.History of hypersensitivity to the study drug or any of its excipients; 8. Renal impairment 10. Liver impairment; 11.Patients with active chronic hepatitis B infection, active hepatitis C infection;
- Other protocol-defined inclusion/exclusion criteria may apply" at the end
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 |
|---|---|---|
| deferasirox125, 250, 500 mg dispersable tablets | Drug | deferasirox |
Primary outcome
Change from baseline of glucose blood level measured after 2 h after receiving a glucose-equivalent oral challenge
Time frame: 36 months
The primary efficacy variable is the change (mg/dl) from baseline to 36 months of glucose plasma levels measured 2 hr post glusose equivalent oral challange. After a 12-hour overnight fasting, at zero time (baseline) blood sample will be drawn and afterwards patients will receive a glucose-equivalent oral challenge (75 grams). After glucose loading plasma samples will be drawn at 30, 60, 90, 120 minutes for determination of plasma glucose. This will be repeated every 6 month till end of study
8 secondary outcomes
Glucose of OGTT ( AUC)
Time frame: baseline and every 6 months measurement of 2hour Glocose of OGTT
change on insulin secretion and sensitivity
Time frame: baseline and every 6 months measurement of 2hr Glucose OGTT
Measurement of thyroid hormones TSH and FT4
Time frame: baseline and every 12 months
Risk factors for the impairment of glucose homeostasis
Time frame: baseline and monthly till End of Study
Changes in endocrine funcionts parameters
Time frame: baseline and monthly till EOS
Changes in parameters of bone metabolism
Time frame: baseline, monthly or every 6 months till end of study
Iron overload status
Time frame: baseline and regularly till end of study (monthly or yearly as specified)
Safety of deferasirox therapy
Time frame: baseline and at every scheduled visit (weekly for the first months or after dose escalation and monthly thereafter or yearly till EOS
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Apr 2017.
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
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing deferasirox in people with Thalassemia (Transfusion Delendent), run by Novartis Pharmaceuticals.
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 1 inclusion and 4 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: withdrawn, as of the sponsor's last update on 20 Apr 2017. Status can change between updates, so confirm with the study contact before making plans.
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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 was withdrawn before enrolling participants, so there are no results.
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?
Change from baseline of glucose blood level measured after 2 h after receiving a glucose-equivalent oral challenge (time frame: 36 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 20 Apr 2017.