Deferasirox BID (Twice a Day) in Transfusion Dependent Thalassemia Patients With Inadequate Response to High Doses

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT01948817
• Date of registration: 19/09/2013
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharmaceuticals
• Public title: Deferasirox BID (Twice a Day) in Transfusion Dependent Thalassemia Patients With Inadequate Response to High Doses
• Scientific title: A Prospective Single Arm Study to Assess the Efficacy and Safety of Deferasirox 20 mg/kg BID in Transfusion Dependent Thalassemia Patients Inadequately Responding to Current Treatment With Doses > 35mg/kg QD (Once a Day).
• Date of first enrolment: February 2014
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT01948817
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Brazil, Lebanon, Thailand

Contacts

• Name: Novartis Pharmaceuticals
• Affiliation: Novartis Pharmaceuticals

Key inclusion & exclusion criteria

Inclusion Criteria:

- Written informed consent must be obtained prior to any screening procedures

- Male or female aged = 2 years at screening

- Patients with transfusion dependent thalassemia

- Patients confirmed as inadequate- responders to deferasirox > 35 mg/kg QD and treated
with QD for at least 6 months

- Regular transfusion indicated by a blood requirement = 8 blood transfusions per year
at screening

- Serum ferritin level > 2,500 ng/mL at screening (two consecutive values at least 2
weeks apart from each other)

- One SF measurement > 2,500 ng/mL between 6 and 9 months prior to study enrollment

- Three SF measurements > 2,500 ng/mL, performed at least 3 weeks apart from each other,
during the 6 month treatment with QD dosing of deferasirox prior to study enrollment

- The average of the two screening SF values (collected 2 weeks apart from each other)
must not show a decrease from the 6 to 9 month SF value taken prior to study
enrollment

- The average of the two screening SF values (collected 2 weeks apart from each other)
must not show a decrease from each of the three SF values obtained during the 6 months
of deferasirox QD treatment prior to study enrollment

- LIC = 7 mg Fe/g dw measured at the screening visit, (this value will be used as a
baseline measurement

Exclusion Criteria:

- Patients who are intolerant to > 35 mg/kg/day QD of deferasirox in the 6 months prior
to study enrollment

- Patients with mean levels of ALT >5 x ULN

- Patients with serum creatinine above the upper limit of normal (ULN)

- Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5
(mg/mg)

- Creatinine clearance = 60 ml/min

- Chronic hepatitis B infection, active hepatitis C infection

- History of a positive HIV test

- Uncontrolled systemic hypertension

- Patients participating in another clinical trial or receiving a systemic
investigational drug within the past 4 weeks or topical investigational drug within
the past 7 days of screening

- History of non-compliance with medical regimens or patients who are considered
potentially unreliable and/or not cooperative, unwilling or unable to comply with the
protocol

- History of hypersensitivity to any of the study drug or excipients

- 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.)

- History of drug or alcohol abuse within the 12 months prior to enrollment.



Age minimum: 2 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Focus on Transfusion Dependent Thalassemia Patients Who Are Inadequate Responders to Deferasirox > 35mg/kgQD

Intervention(s)

Drug: Deferasirox

Primary Outcome(s)

Mean relative change in SF (Serum Ferritin) [Time Frame: Baseline, 24 Weeks]

Secondary Outcome(s)

Mean Absolute/relative change in LIC [Time Frame: Baseline, 24 Weeks]

Secondary ID(s)

2013-002624-16

CICL670A2420

Source(s) of Monetary Support

Please refer to primary and secondary sponsors