Adults 6 Months to 9, any sex, with Beta Thalassemia Major Anemia or Iron Overload. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
The Percentage of Patients in Each Treatment Group Who Still Have a Serum Ferritin Level < 1000 Micrograms Per Liter (μg/L) at Month 12Primary· 12 months
Current treatment guidelines are that standard iron chelation therapy should begin after a serum ferritin level of 1000 μg/L has been reached; thus, patients who were at this level at two consecutive visits were removed from the study so that they could start on this therapy.
Group
Value
95% CI
Deferiprone
21
Placebo
12
Percentage of Patients With Serum Ferritin Still Below the Threshold at Different Time PointsSecondary· 4, 8, and 12 months
The outcome measure was the percentage of patients in each group still below the serum ferritin threshold at Months 4, 8, and 12. Without adequate chelation therapy, most patients receiving red blood cell transfusions are likely to exceed this level within a few months. However, a safety feature of the study design was that patients who reached the serum ferritin threshold were withdrawn (i.e., so that they could begin standard chelation therapy).
Number of patients still below threshold at Month 4
Group
Value
95% CI
Deferiprone
29
Placebo
28
Number of patients still below threshold at Month 8
Group
Value
95% CI
Deferiprone
22
Placebo
20
Number of patients still below threshold at Month 12
Group
Value
95% CI
Deferiprone
21
Placebo
12
Adverse events — posted to ClinicalTrials.gov
Time frame: 12 months.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on iron chelation therapy. Half the patients in the study will receive deferiprone, and the other half will receive placebo, for up to 12 months.
Publications & conference data
6 peer-reviewed publications reference this trial (live from Europe PMC):
Publications: Europe PMC API search by NCT ID, retrieved 9 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Chiesi Canada Corp
Last refreshed: 15 March 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03591575.