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Iron Chelator

Deferiprone for Iron Overload

Phase 4
Waitlist Available
Led By Janet Kwiatkowski, MD
Research Sponsored by ApoPharma
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up one year, two years, and three years after the start of deferiprone therapy
Awards & highlights

Summary

This trial is studying the safety and effectiveness of deferiprone, a medication that removes excess iron, in patients with sickle cell disease or other anemias who have too much iron from frequent blood transfusions. Deferiprone is an oral treatment that has been used to manage iron overload in patients with thalassemia and has shown positive results in various studies.

Eligible Conditions
  • Iron Overload
  • Anemia
  • Sickle Cell Disease

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~one year, two years, and three years after the start of deferiprone therapy
This trial's timeline: 3 weeks for screening, Varies for treatment, and one year, two years, and three years after the start of deferiprone therapy for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Patients With Adverse Events
Secondary outcome measures
Change From Baseline in Cardiac MRI T2*
Change From Baseline in Liver Iron Concentration (LIC)
Change From Baseline in Serum Ferritin

Side effects data

From 2013 Phase 4 trial • 32 Patients • NCT01770652
13%
Headache
100%
80%
60%
40%
20%
0%
Study treatment Arm
Severe Renal Impairment
Normal Hepatic Function (Healthy Volunteers)
Mild Renal Impairment
Moderate Renal Impairment

Trial Design

2Treatment groups
Experimental Treatment
Group I: Group 2: Deferiprone 2 yearsExperimental Treatment1 Intervention
Patients in this group are those who were randomized to the deferoxamine arm in study LA38-0411, and hence will receive deferiprone for 2 years (both of them in the extension study).
Group II: Group 1: Deferiprone 3 yearsExperimental Treatment1 Intervention
Patients in this group are those who were randomized to the deferiprone arm in study LA38-0411, and hence will receive deferiprone for a total of 3 years (1 year in the initial study plus 2 years in the extension study).
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Deferiprone
FDA approved

Find a Location

Who is running the clinical trial?

ApoPharmaLead Sponsor
25 Previous Clinical Trials
2,152 Total Patients Enrolled
6 Trials studying Iron Overload
677 Patients Enrolled for Iron Overload
Janet Kwiatkowski, MDPrincipal InvestigatorChildren's Hospital of Philadelphia
6 Previous Clinical Trials
969 Total Patients Enrolled
1 Trials studying Iron Overload
230 Patients Enrolled for Iron Overload
~13 spots leftby Sep 2025