Trial intelligence / Profile preview

Deferitazole in Pediatric Transfusional Iron Overload

NCT01363908Phase 2

Key facts

Status: Terminated
Start: Aug 2011
Primary completion: May 2014
Countries: 5
Sponsor: Takeda
Modality: Small Molecules

Drugs: deferitazole
Conditions: Transfusional Iron Overload (TIO)

Overview

Phase 2, open-label trial of deferitazole in Transfusional Iron Overload (TIO) (3 arms), sponsored by Takeda. Primary endpoints: Maximum Observed Plasma Concentration at 24 hours, Time of Maximum Observed Plasma Concentration at 24 hours and 6 more. Started Aug 2011; status: terminated.

Design

3 arms

Study design: Non-randomizedOpen-label

PK Dose (16 mg/kg)
Single dose for PK assessment
Chronic Dose (26 mg/kg)
Daily dosing starting at 26 mg/kg
Chronic Dose (36 mg/kg)
Daily dosing starting at 36 mg/kg

Who it enrols

Pediatric patients (6-<18 years) with transfusional iron overload (ferritin >500 ng/mL, LIC >2 and <30 mg/g, cardiac T2* >10ms) requiring chronic chelation.

Endpoints

Primary

Maximum Observed Plasma Concentration24 hours
Time of Maximum Observed Plasma Concentration24 hours
Area Under The Plasma Concentration-Time Curve24 hours
Terminal Half-life24 hours
Renal Clearance24 hours

Secondary

Change From Baseline in Liver Iron Concentration Assessed by R2* MRI48 weeks
Change From Baseline in Cardiac Iron Load Assessed by T2* MRI48 weeks
Change From Baseline in Serum Ferritin48 weeks

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