Clinical trial NCT01709838: Efficacy and Safety Study of Deferasirox in Patients With Non-transfusion Dependent Thalassemia
Official title: An Open Label, Multi-center, Efficacy and Safety Study of Deferasirox in Iron Overloaded Patients With Non-transfusion Dependent Thalassemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Non-transfusion Dependent Thalassemia
Keywords: Non-transfusion dependent thalassemia
NTDT
deferasirox
ICL670
LIC
Liver Iron Concentration
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2012-10-18
Enrollment: 134
Sex: ALL
Minimum age (years): 10
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

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

Exclusion Criteria:

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

Primary outcomes:
measure: Absolute Change in Liver Iron Content (LIC) at 52 Weeks From Baseline
timeFrame: Baseline, 52 weeks
Secondary outcomes:
measure: Percentage of Participants With Baseline LIC>15 Achieving LIC<5 mg Fe/g dw
timeFrame: 5 years
measure: Time to Achieving LIC <5 mg Fe/g dw
timeFrame: 5 years
measure: Time From Target LIC of 3 mg Fe/g dw to the First LIC ≥5 mg Fe/g dw in the Follow up Period
timeFrame: post-baseline, up to 260 weeks
measure: Absolute Change in Health-related Outcomes Using Medical Outcomes Study Form 36 (SF-36v2)
timeFrame: Baseline, 52, 104 & 156 Weeks
measure: Absolute Change in Health-related Outcomes Using the Pediatric Quality of Life Questionnaire (PedsQL™)
timeFrame: Baseline, 52, 104 & 156 Weeks
measure: Absolute Change in LIC From Baseline Over Time
timeFrame: 24, 52, 76, 104, 128, 156, 180, 208, 232, 260 Weeks
measure: Serum Ferritin (SF) vs LIC at Baseline and EOS (Week 260 + 30 Days Follow-up)
timeFrame: Baseline, End of Study (EOS): Week 260 + 30 days follow up
measure: Correlation Analysis for Absolute Change in LIC and Serum Ferritin at Week 24 and EOS (Week 260 + 30 Days Follow-up)
timeFrame: Week 24, End of Study (EOS): Week 260 + 30 days follow up
measure: Absolute Change in LIC From Baseline After 52 Weeks of Treatment by Underlying Non-transfusion Dependent Thalassemia (NTDT) Syndrome
timeFrame: Baseline, 52 Weeks
measure: Absolute Change in Serum Ferritin From Baseline After 52 Weeks
timeFrame: Baseline, 52 weeks
measure: PK Parameters: AUCtau
timeFrame: pre-dose (0 hour), and at 2, and 4 hours at Week 4
measure: PK Parameters: Cmax
timeFrame: pre-dose (0 hour), and at 2, and 4 hours at Week 4
measure: PK Parameters: Tmax
timeFrame: pre-dose (0 hour), and at 2, and 4 hours at Week 4
measure: Plasma Pharmacokinetics (PK) Deferasirox Concentrations
timeFrame: Weeks 12 & 24: pre-dose (0hr), 2hr & 4hr post-dose
Other outcomes:
Locations:
city: Nanning
country: China
facility: Novartis Investigative Site
state: Guangxi
city: Goudi-Athens
country: Greece
facility: Novartis Investigative Site
state: GR
city: Cagliari
country: Italy
facility: Novartis Investigative Site
state: CA
city: Milano
country: Italy
facility: Novartis Investigative Site
state: MI
city: Hazmiyeh
country: Lebanon
facility: Novartis Investigative Site
state: Beirut
city: Bangkok
country: Thailand
facility: Novartis Investigative Site
city: Tunis
country: Tunisia
facility: Novartis Investigative Site
city: Adana
country: Turkey
facility: Novartis Investigative Site
city: Istanbul
country: Turkey
facility: Novartis Investigative Site
city: Izmir
country: Turkey
facility: Novartis Investigative Site
city: London
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: