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

NCT01948817 | Withdrawn Phase 2

• 2 other identifiers: CICL670A24202013-002624-16
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is an open label, single arm, and multicenter study. The study will include the following phases. A screening phase which lasts for 4 weeks to determine patient eligibility. This phase will be followed by a 24 week Open label treatment phase. The study treatment is defined as deferasirox 20mg/kg BID 9Twice a day). Serum Ferritin Levels and MRI (Magmetic Resonance Imaging) LIC (Liver Iron Concentration) will be measured to evaluate the response to BID.

Conditions

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

Keywords

Thalassemia

Outcome Measures

Primary Outcomes (1)

• Mean relative change in SF (Serum Ferritin)

  To evaluate the efficacy of a twice-daily dose regimen of deferasirox in inadequate-responders to a once daily dose regimen as measured by relative change in serum ferritin from baseline to 24 weeks of treatment

  Baseline, 24 Weeks

Secondary Outcomes (1)

• Mean Absolute/relative change in LIC

  Baseline, 24 Weeks

Study Arms (1)

Deferasirox

EXPERIMENTAL

Deferasirox 20mg/kg taken BID

Drug: Deferasirox

Interventions

Deferasirox DRUG

Deferasirox 20mg/kg taken BID

Deferasirox

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

MeSH Terms

Conditions

Thalassemia

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Benzoates; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Triazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 19, 2013
• First Posted: September 24, 2013
• Study Start: February 1, 2014
• Primary Completion: February 1, 2016
• Study Completion: February 1, 2016
• Last Updated: April 20, 2017

Record last verified: 2014-01