Efficacy and Safety Study of Deferasirox in Patients With Non-transfusion Dependent Thalassemia
THETIS
An Open Label, Multi-center, Efficacy and Safety Study of Deferasirox in Iron Overloaded Patients With Non-transfusion Dependent Thalassemia
2 other identifiers
interventional
134
7 countries
8
Brief Summary
Assessed the efficacy of deferasirox in patients with non-transfusion dependent thalassemia based on change in liver iron concentration from baseline after 52 weeks of treatment. Provided further assessment of the long-term efficacy and safety of deferasirox in NTDT patients with iron overload (LIC ≥ 5 mg Fe/g liver dw and SF ≥ 300 ng/mL) for up to 260 weeks.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_4
Started Dec 2012
Longer than P75 for phase_4
8 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 16, 2012
CompletedFirst Posted
Study publicly available on registry
October 18, 2012
CompletedStudy Start
First participant enrolled
December 6, 2012
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 3, 2015
CompletedStudy Completion
Last participant's last visit for all outcomes
January 17, 2019
CompletedResults Posted
Study results publicly available
August 28, 2019
CompletedOctober 2, 2019
September 1, 2019
2.1 years
October 16, 2012
July 17, 2019
September 19, 2019
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Absolute Change in Liver Iron Content (LIC) at 52 Weeks From Baseline
Absolute change in liver iron concentration measured by MRI from baseline after 52 weeks of treatment
Baseline, 52 weeks
Secondary Outcomes (14)
Percentage of Participants With Baseline LIC>15 Achieving LIC<5 mg Fe/g dw
5 years
Time to Achieving LIC <5 mg Fe/g dw
5 years
Time From Target LIC of 3 mg Fe/g dw to the First LIC ≥5 mg Fe/g dw in the Follow up Period
post-baseline, up to 260 weeks
Absolute Change in Health-related Outcomes Using Medical Outcomes Study Form 36 (SF-36v2)
Baseline, 52, 104 & 156 Weeks
Absolute Change in Health-related Outcomes Using the Pediatric Quality of Life Questionnaire (PedsQL™)
Baseline, 52, 104 & 156 Weeks
- +9 more secondary outcomes
Study Arms (1)
Deferasirox
OTHERAll patients were treated with 10mg/kg/day deferasirox with dose adjustments after 4 weeks of treatment according to baseline Liver Iron Concentration (LIC).
Interventions
Deferasirox dispersible tablets at strengths of 125 mg, 250 mg, and 500 mg were administered by oral daily dosing.
Eligibility Criteria
You may qualify if:
- Non-transfusion dependent congenital or chronic anemia inclusive of beta-thalassemia intermedia, HbE beta-thalassemia or alpha-thalassemia intermedia (HbH disease)/ Liver iron concentration \>/= 5 mg Fe/g dw Serum Ferritin \>/= 300 ng/mL
You may not qualify if:
- HbS-beta Thalassemia, anticipated regular transfusion program during the study, blood transfusion 6 months prior to study start, significant proteinuria, creatinine clearance \</= 40 ml/min, serum creatinine \> ULN, ALT \>5 x ULN, active hepatitis B or C, cirrhosis
- Pediatrics Only:
- A patient's weight of at least 20 kg is required to allow dosing of 5 mg/kg with one tablet of 125 mg
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (11)
Novartis Investigative Site
Nanning, Guangxi, 530021, China
Novartis Investigative Site
Goudi-Athens, GR, 115 27, Greece
Novartis Investigative Site
Cagliari, CA, 09121, Italy
Novartis Investigative Site
Milan, MI, 20122, Italy
Novartis Investigative Site
Hazmiyeh, Beyrouth, PO BOX 213, Lebanon
Novartis Investigative Site
Bangkok, 10700, Thailand
Novartis Investigative Site
Tunis, 1006, Tunisia
Novartis Investigative Site
Adana, 01330, Turkey (Türkiye)
Novartis Investigative Site
Istanbul, 34093, Turkey (Türkiye)
Novartis Investigative Site
Izmir, 35040, Turkey (Türkiye)
Novartis Investigative Site
London, NW1 2PJ, United Kingdom
MeSH Terms
Interventions
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Novartis Pharmaceuticals
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 16, 2012
First Posted
October 18, 2012
Study Start
December 6, 2012
Primary Completion
January 3, 2015
Study Completion
January 17, 2019
Last Updated
October 2, 2019
Results First Posted
August 28, 2019
Record last verified: 2019-09
Data Sharing
- IPD Sharing
- Will share
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.