A prospective single arm study to assess the efficacy and safety of deferasirox in transfusion dependent beta-thalassemia patients unresponsive to current treatment or with intolerance to single dose therapy

A prospective single arm study to assess the efficacy and safety of deferasirox 20 mg/kg BID in transfusion dependent beta-thalassemia patients unresponsive to current treatment with doses > 35 mg/kg QD or with intolerance to single dose therapy

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

IRCT

Registry ID

IRCT2014122920051N1

Enrollment

Registered

2014-12-29

Start date

2015-02-20

Completion date

Unknown

Last updated

2018-02-22

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

Conditions

Thalassemia. Beta thalassaemia

Interventions

Deferasirox 20 mg/kg BID ,During on year,all patients took defreasirox during the study.

Treatment - Drugs

Deferasirox 20 mg/kg BID ,During on year,all patients took defreasirox during the study

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Inclusion: Aged 2 years; Serum ferritin >2000 ng/ml and unresponsive to current treatment with doses > 35 mg/kg QD (decrease in ferritin level less than 10%); patients who did not tolerate single dose therapy due to adverse effects Exclusion: Patients will be excluded from this trial if they have one of the following criteria: positive serology for HBS Ag , HCV Ab, HIV Ab. Abnormal liver or kidney function tests;An alanine aminotranferase (ALT) level greater than 250 U/L;serum creatinine level above the upper limit of normal, and a urinary protein-creatinine ratio of greater than 0.5 mg/mg); a history of ocular toxicity related to iron chelation therapy;uncontrolled systemic hypertention, a prolonged corrected QT interval; systemic infection within 10days prior to enrollment; gastrointestinal absorption problem ;Patients who found complication of chelation therapy during trial as well as breast feeding and pregnant women.

Exclusion criteria

Exclusion criteria:

Design outcomes

Primary

Measure	Time frame
Efficacy as reduction in serum ferritin levels as well as change in T2MRI. Timepoint: Base line,three month,six month,nine month,end of study. Method of measurement: For better evaluation of serum ferritin changes during one year, we define an index: Mean of Relative Change of Serum Ferritin (MRC-SF) from the baseline levels that will be calculated as the percentage of difference between mean serum ferritin levels of each occasion from mean baseline serum ferritin levels.	—

Measure

Time frame

Efficacy as reduction in serum ferritin levels as well as change in T2MRI. Timepoint: Base line,three month,six month,nine month,end of study. Method of measurement: For better evaluation of serum ferritin changes during one year, we define an index: Mean of Relative Change of Serum Ferritin (MRC-SF) from the baseline levels that will be calculated as the percentage of difference between mean serum ferritin levels of each occasion from mean baseline serum ferritin levels.

—

Secondary

Measure	Time frame
Gastrointestinal symptoms and skin rash fatal, acute, irreversible renal failure. Timepoint: Baseline,three months,six months,nine months,End of study. Method of measurement: Exact follow up of the patients examination,laboratory data.;Safety. Timepoint: 12 years. Method of measurement: exact follow up of the patients examination and laboratory data.	—

Countries

Iran (Islamic Republic of)

Contacts

Public ContactSeazaneh Haghpanah

Hematology Research Center, Nemazee Hospital, Shiraz

haghpanah@sums.ac.ir+98 71 3612 5617

Outcome results

None listed

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