Clinical trial NCT02069886: Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent Thalassemia
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
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE4
Conditions: Thalassemia (Transfusion Delendent)
Keywords: Endocrine complications
transfusion dependent thalassemia
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2014-02-24
Enrollment: 0
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

Brief 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. 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.


Eligibility criteria: Inclusion Criteria:

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%);

Exclusion Criteria:

1. Non transfusional hemosiderosis;
2. 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

Primary outcomes:
measure: Change from baseline of glucose blood level measured after 2 h after receiving a glucose-equivalent oral challenge
timeFrame: 36 months
Secondary outcomes:
measure: Glucose of OGTT ( AUC)
timeFrame: baseline and every 6 months measurement of 2hour Glocose of OGTT
measure: change on insulin secretion and sensitivity
timeFrame: baseline and every 6 months measurement of 2hr Glucose OGTT
measure: Measurement of thyroid hormones TSH and FT4
timeFrame: baseline and every 12 months
measure: Risk factors for the impairment of glucose homeostasis
timeFrame: baseline and monthly till End of Study
measure: Changes in endocrine funcionts parameters
timeFrame: baseline and monthly till EOS
measure: Changes in parameters of bone metabolism
timeFrame: baseline, monthly or every 6 months till end of study
measure: Iron overload status
timeFrame: baseline and regularly till end of study (monthly or yearly as specified)
measure: Safety of deferasirox therapy
timeFrame: baseline and at every scheduled visit (weekly for the first months or after dose escalation and monthly thereafter or yearly till EOS
Other outcomes:
Locations:
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: