Pilot Study for Patients With Poor Response to Deferasirox

PHASE4CompletedINTERVENTIONAL
Enrollment

15

Participants

Timeline

Start Date

March 31, 2008

Primary Completion Date

October 31, 2008

Study Completion Date

November 30, 2008

Conditions
Transfusion-dependent HemachromatosisThalassemia MajorSickle Cell Disease
Interventions
DRUG

Deferoxamine

After a 3-day washout period from all chelation, all patients have a 12 hour infusion of 50mg/kg of deferoxamine with urine collection and pre and post blood sampling to assess iron and Total Iron Binding Capacity (TIBC) by atomic absorption.

DRUG

Deferasirox

After a 3-day washout period from all chelation, patients had a desferal challenge which was followed by a single dose of deferasirox, 35mg/kg orally with blood sampling taken pre-deferasirox and at intervals for 24 hours after the dose.

RADIATION

HIDA

All patients had a HIDA scan to assess physiologic liver clearance capacity.

Trial Locations (1)

02115

Children's Hospital Boston, Boston

Sponsors

Collaborators (1)

All Listed Sponsors
collaborator

Novartis

INDUSTRY

lead

Boston Children's Hospital

OTHER