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Related Experiment Video

Updated: Jul 13, 2026

Quantifiable and Inexpensive Cell-Free Fluorescent Method to Confirm the Ability of Novel Compounds to Chelate Iron

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Drug evaluation: Deferitrin for iron overload disorders.

James C Barton¹

¹Southern Iron Disorders Center, Birmingham, AL 35209, USA. ironmd@dnamail.com

Idrugs : the Investigational Drugs Journal

|July 21, 2007

Summary

Deferitrin, a novel oral iron chelator, effectively promotes iron excretion in patients with beta-thalassemia major. Early trials show good tolerability and safety, suggesting potential for iron overload treatment.

Area of Science:

Pharmacology and Therapeutics
Hematology
Drug Development

Background:

Severe iron overload is a significant complication in patients requiring chronic erythrocyte transfusions for anemias like beta-thalassemia major.
Existing iron chelation therapies have limitations, necessitating the development of novel, effective treatments.

Purpose of the Study:

To evaluate the safety, tolerability, and pharmacokinetic profile of Deferitrin (GT-56-252), a new oral hexadentate iron chelator.
To assess Deferitrin's efficacy in promoting iron excretion in adults with beta-thalassemia.

Main Methods:

Phase I clinical trials were conducted in adult patients with beta-thalassemia.
Deferitrin was administered in both liquid and capsule forms under fed and fasted conditions.

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Iron excretion, safety, and tolerability were monitored.

Main Results:

Deferitrin demonstrated dose-related promotion of iron excretion.
The drug was well tolerated in both liquid and capsule formulations.
No serious adverse events or significant laboratory abnormalities were reported.

Conclusions:

Deferitrin shows promise as a potential monotherapy or combination therapy for severe iron overload in beta-thalassemia major.
Further extensive clinical trials are required to confirm its pharmacokinetic profile, efficacy, safety, and tolerability.
A Phase I/II trial has reportedly completed recruitment, advancing its development.