Phase 4 N=134 Treatment

Efficacy and Safety Study of Deferasirox in Patients With Non-transfusion Dependent Thalassemia

Non-transfusion Dependent Thalassemia

Bottom Line

View on ClinicalTrials.gov: NCT01709838 ↗

Enrolled (actual)

134

Serious AEs

33.6%

Results posted

Aug 2019

Primary outcome: Primary: Absolute Change in Liver Iron Content (LIC) at 52 Weeks From Baseline — -6.68 mg Fe/g dw

Study Design & Population

Study type: Interventional
Phase: Phase 4
Interventions: deferasirox (Drug)
Age: Pediatric, Adult, Older Adult · 10+ yrs
Sex: All
Sponsor: Novartis Pharmaceuticals
Primary completion: Jan 2015

Outcome Measures

Outcome	Result	p-value
PRIMARY Absolute Change in Liver Iron Content (LIC) at 52 Weeks From Baseline	-6.68	—
SECONDARY Percentage of Participants With Baseline LIC>15 Achieving LIC<5 mg Fe/g dw	51.0	—
SECONDARY Time to Achieving LIC <5 mg Fe/g dw	36.3	—
SECONDARY Time From Target LIC of 3 mg Fe/g dw to the First LIC ≥5 mg Fe/g dw in the Follow up Period	27.4	—
SECONDARY Absolute Change in Health-related Outcomes Using Medical Outcomes Study Form 36 (SF-36v2)	-0.5; -0.7; 0.6; 0.9; -1.0; 1.0	—
SECONDARY Absolute Change in Health-related Outcomes Using the Pediatric Quality of Life Questionnaire (PedsQL™)	2.3; 1.0; -0.9; 0.7; 3.1; 10.9	—
SECONDARY Absolute Change in LIC From Baseline Over Time	-3.67; -7.02; -8.93; -9.63; -10.03; -10.20	—
SECONDARY Serum Ferritin (SF) vs LIC at Baseline and EOS (Week 260 + 30 Days Follow-up)	0.730; 0.531	—
SECONDARY Correlation Analysis for Absolute Change in LIC and Serum Ferritin at Week 24 and EOS (Week 260 + 30 Days Follow-up)	0.299; 0.740	—
SECONDARY Absolute Change in LIC From Baseline After 52 Weeks of Treatment by Underlying Non-transfusion Dependent Thalassemia (NTDT) Syndrome	-6.11; -6.18; -7.97; -6.00	—
SECONDARY Absolute Change in Serum Ferritin From Baseline After 52 Weeks	-494.64	—
SECONDARY PK Parameters: AUCtau	678.2	—
SECONDARY PK Parameters: Cmax	53.367	—
SECONDARY PK Parameters: Tmax	2.5131	—
SECONDARY Plasma Pharmacokinetics (PK) Deferasirox Concentrations	6.513; 48.556; 44.652	—

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 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01709838). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.