A study looking at the safety and tolerability of a new formulation of deferiprone inpatients with excess iron due to frequent blood transfusions

Safety and acceptability of deferiprone delayed release tablets in patients with systemic iron overload - LA61-0218

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-004016-22-GR

Enrollment

Registered

2018-12-05

Start date

2018-12-27

Completion date

Unknown

Last updated

2025-01-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Systemic iron overload MedDRA version: 20.0 Level: PT Classification code 10065973 Term: Iron overload System Organ Class: 10027433 - Metabolism and nutrition disorders

Interventions

Product Name: Deferiprone delayed release 1000 mg tablets Pharmaceutical Form: Modified-release tablet INN or Proposed INN: DEFERIPRONE CAS Number: 30652-11-0 Current Sponsor code: APO-066 Concentrati

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: •Male or female aged = 18 years •Diagnosis of thalassemia syndrome, sickle cell disease, or other disorder requiring a regular regimen of red blood cell transfusions •On a stable regimen (=3 months) of Ferriprox tablets for the treatment of systemic iron overload •A record of at least the last 12 measured ALT and AST levels prior to baseline Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 30 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0

Exclusion criteria

Exclusion criteria: •ALT and/or AST value > 5 x ULN at screening •Active case of hepatitis B or C at screening

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the safety of deferiprone delayed release (DR) tablets in two different dosage groups in patients with systemic iron overload.;Secondary Objective: •To evaluate the tolerability of deferiprone DR tablets in two different dosage groups in patients with systemic iron overload •To evaluate the acceptability to patients of deferiprone DR ;Primary end point(s): The primary endpoint is the incidence of any post-dose occurrence of increases in ALT and/or ALT levels that meet one of the criteria for being considered a safety concern. The safety criteria are: •For a patient whose baseline level is within the normal range, the criterion will be reaching a value of 5 times the ULN during the study. •For a patient whose baseline level is above the ULN, the criterion will be reaching a value of 5 times the baseline value during the study or >10 x ULN. ;Timepoint(s) of evaluation of this end point: Adverse events will be collected throughout the study; laboratory safety assessments will be performed at screening, baseline, and Days 3, 7, 14, 21, and 28	—

Measure

Time frame

Main Objective: To evaluate the safety of deferiprone delayed release (DR) tablets in two different dosage groups in patients with systemic iron overload.;Secondary Objective: •To evaluate the tolerability of deferiprone DR tablets in two different dosage groups in patients with systemic iron overload •To evaluate the acceptability to patients of deferiprone DR ;Primary end point(s): The primary endpoint is the incidence of any post-dose occurrence of increases in ALT and/or ALT levels that meet one of the criteria for being considered a safety concern. The safety criteria are: •For a patient whose baseline level is within the normal range, the criterion will be reaching a value of 5 times the ULN during the study. •For a patient whose baseline level is above the ULN, the criterion will be reaching a value of 5 times the baseline value during the study or >10 x ULN. ;Timepoint(s) of evaluation of this end point: Adverse events will be collected throughout the study; laboratory safety assessments will be performed at screening, baseline, and Days 3, 7, 14, 21, and 28

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Secondary

Measure	Time frame
Secondary end point(s): Secondary endpoints are as follows: •The assessment of tolerability will be based on the incidence of gastrointestinal (GI) distress reported by patients during the study. •The assessment of acceptability will be based on the number of patients who indicate that they prefer the DR formulation over Ferriprox IR after considering their preference with respect to scheduling, ease of administration, and tolerability. ;Timepoint(s) of evaluation of this end point: •A questionnaire asking patients for their views on the acceptability of the delayed release formulation vs. that of Ferriprox will be administered on Day28. •Adverse events related to gastrointestinal tolerability will be collected throughout the study as per the timepoints.	—

Measure

Time frame

Secondary end point(s): Secondary endpoints are as follows: •The assessment of tolerability will be based on the incidence of gastrointestinal (GI) distress reported by patients during the study. •The assessment of acceptability will be based on the number of patients who indicate that they prefer the DR formulation over Ferriprox IR after considering their preference with respect to scheduling, ease of administration, and tolerability. ;Timepoint(s) of evaluation of this end point: •A questionnaire asking patients for their views on the acceptability of the delayed release formulation vs. that of Ferriprox will be administered on Day28. •Adverse events related to gastrointestinal tolerability will be collected throughout the study as per the timepoints.

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Countries

Canada, Greece, United States

Contacts

Public ContactJohn Connelly

ApoPharma Inc.

jconnell@apopharma.com1416401-7296

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026