efficacy and safety of deferasirox in patients with non-transfusion dependent thalassemia

An open label, multi-center, efficacy and safety study of deferasirox in iron overloaded patients with non-transfusion dependent thalassemia (THETIS) - THETIS

Status

Active, not recruiting

Phases

Phase 4

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2012-000650-64-GB

Enrollment

120

Registered

2012-09-07

Start date

2012-11-05

Completion date

Unknown

Last updated

2020-07-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

iron overload in patients with non-transfusion dependent thalassemia MedDRA version: 20.0 Level: HLT Classification code 10022979 Term: Iron excess System Organ Class: 100000004861

Interventions

Trade Name: EXJADE Product Code: ICL670 Pharmaceutical Form: Dispersible tablet INN or Proposed INN: DEFERASIROX CAS Number: 201530-41-8 Concentration unit: mg milligram(s) Concentration type: equal C

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: • Male or female aged = 10 years with non-transfusion-dependent congenital or chronic anemias inclusive of beta-thalassemia intermedia, HbE beta-thalassemia or alpha-thalassemia intermedia (HbH disease) • LIC = 5 mg Fe/g dw measured by R2 MRI at screening • Serum ferritin = 300 ng/mL at screening (two consecutive values at least 14 days apart from each other) Additional inclusion criteria as per full protocol may apply Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: • HbS-beta Thalassemia • Anticipated regular transfusion program during the study • Any blood transfusion 6 months prior to study start • Significant proteinuria • creatinine clearance =40 ml/min • serum creatinine >ULN; ALT >5xULN Additional exclusion criteria as per full protocol may apply

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess the efficacy of deferasirox in patients with NTDT based on change in LIC from baseline after 52 weeks of treatment;Secondary Objective: • Assess response rates in patients with baseline LIC values >15mg Fe/g dw defined as the proportion of patients achieving an LIC <5mg Fe/g dw and time to achieving LIC <5mg Fe/g dw •Assess long-term efficacy and safety treatment to target LIC of 3mg Fe/g dw followed by one or more treatment holidays until the LIC is =5mg Fe/g dw •To evaluate the impact of deferasirox on Quality of Life • To evaluate the efficacy of deferasirox based on change in LIC from baseline over time • To assess the correlation of change in SF and LIC at baseline and end of study (EOS) • To assess the efficacy of deferasirox based on change in LIC from baseline after 52 weeks of treatment by NTDT syndrome • To assess the efficacy of deferasirox based on change in SF from baseline over 52 weeks of treatment • To evaluate the safety of deferasirox doses up to 30 mg/kg/day • To assess the efficacy of deferasirox on endocrine function • To conduct pharmacokinetic (pK) analysis in a subset of patients;Primary end point(s): Change in LIC from baseline ;Timepoint(s) of evaluation of this end point: 52 weeks	—

Measure

Time frame

Main Objective: To assess the efficacy of deferasirox in patients with NTDT based on change in LIC from baseline after 52 weeks of treatment;Secondary Objective: • Assess response rates in patients with baseline LIC values >15mg Fe/g dw defined as the proportion of patients achieving an LIC <5mg Fe/g dw and time to achieving LIC <5mg Fe/g dw •Assess long-term efficacy and safety treatment to target LIC of 3mg Fe/g dw followed by one or more treatment holidays until the LIC is =5mg Fe/g dw •To evaluate the impact of deferasirox on Quality of Life • To evaluate the efficacy of deferasirox based on change in LIC from baseline over time • To assess the correlation of change in SF and LIC at baseline and end of study (EOS) • To assess the efficacy of deferasirox based on change in LIC from baseline after 52 weeks of treatment by NTDT syndrome • To assess the efficacy of deferasirox based on change in SF from baseline over 52 weeks of treatment • To evaluate the safety of deferasirox doses up to 30 mg/kg/day • To assess the efficacy of deferasirox on endocrine function • To conduct pharmacokinetic (pK) analysis in a subset of patients;Primary end point(s): Change in LIC from baseline ;Timepoint(s) of evaluation of this end point: 52 weeks

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Secondary

Measure	Time frame
Secondary end point(s): - Proportion of patients with baseline LIC> 15 achieving LIC <5mg Fe/g dw and time to achieving LIC <5mg Fe/g dw - Time from target LIC 3mg Fe/g dw to the first LIC = 5mg Fe/g dw in the follow-up period - Change in serum ferritine (SF) from baseline - change in health related outcomes using Medical Outcomes Study From 36 - change in health related outcomes using Paediatric Quality of Life Questionnaire - change in liver iron concentraion (LIC) from baseline - SF versus LIC at baseline and end of study - number of participants with adverse events as a measure of safety and tolerability - endocrine laboratory parameters: change from baseline during treatment period in total and free testoserone (males), LH and FSH (females), TSH, total and free T4, total and free T3, fasting plasma glucose, insulin, insulin resistance, and cortisol - PK parameters (AUC, Cmax, tmax and trough levels);Timepoint(s) of evaluation of this end point: - every 4 weeks from week 52 to week 260 - every 4 weeks from week 52 to week 260 - week 260 - 156 weeks - 156 weeks - week 260 - week 260 - week 260 - week 260 - week 24 - week 24	—

Measure

Time frame

Secondary end point(s): - Proportion of patients with baseline LIC> 15 achieving LIC <5mg Fe/g dw and time to achieving LIC <5mg Fe/g dw - Time from target LIC 3mg Fe/g dw to the first LIC = 5mg Fe/g dw in the follow-up period - Change in serum ferritine (SF) from baseline - change in health related outcomes using Medical Outcomes Study From 36 - change in health related outcomes using Paediatric Quality of Life Questionnaire - change in liver iron concentraion (LIC) from baseline - SF versus LIC at baseline and end of study - number of participants with adverse events as a measure of safety and tolerability - endocrine laboratory parameters: change from baseline during treatment period in total and free testoserone (males), LH and FSH (females), TSH, total and free T4, total and free T3, fasting plasma glucose, insulin, insulin resistance, and cortisol - PK parameters (AUC, Cmax, tmax and trough levels);Timepoint(s) of evaluation of this end point: - every 4 weeks from week 52 to week 260 - every 4 weeks from week 52 to week 260 - week 260 - 156 weeks - 156 weeks - week 260 - week 260 - week 260 - week 260 - week 24 - week 24

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Countries

China, Greece, Italy, Lebanon, Russian Federation, Thailand, Tunisia, Turkey, United Kingdom

Contacts

Public ContactMedical Collaboration Centre

Novartis Pharmaceuticals Uk Ltd

medinfo.uk@novartis.com01276698370

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026