A study of a drug (Ferriprox®) that removes extra iron from the body, to see how safe it is and how well it works in patients with sickle cell disease.

Long-term safety and efficacy study of Ferriprox® for the treatment of transfusional iron overload in patients with sickle cell disease or other anemias. - FIRST (Ferriprox in patients with iron overload in sickle cell disease trial)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2014-005685-30-GB

Enrollment

300

Registered

2015-07-20

Start date

2015-09-18

Completion date

Unknown

Last updated

2019-12-16

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

Conditions

Iron overload in sickle cell disease or other anemias MedDRA version: 20.0 Level: SOC Classification code 10005329 Term: Blood and lymphatic system disorders System Organ Class: 10005329 - Blood and lymphatic system disorders

Interventions

Trade Name: Ferriprox (deferiprone) 500 mg film-coated tablets Product Name: Ferriprox (deferiprone) 500 mg film-coated tablets Pharmaceutical Form: Film-coated tablet

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Completed study LA38-0411. Are the trial subjects under 18? yes Number of subjects for this age range: 5 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 5 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0

Exclusion criteria

Exclusion criteria: 1. Plan to participate in another clinical trial at any time from the day of enrollment until 30 days post-treatment in the current study. 2. For only those patients who were treated with deferoxamine in study LA38-0411 (Group 2): Presence of any medical condition (including clinically significant laboratory abnormalities, such as ALT = 5 x ULN or creatinine = 2 x ULN), psychological condition, or psychiatric condition which in the opinion of the investigator would cause participation in the study to be unwise. 3. Pregnant, breastfeeding, or planning to become pregnant during the study period. 4. Treatment failure after 1 year on deferiprone which in the investigator’s judgment indicates the need for the patient to be started on a different iron chelator.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the long-term safety and tolerability of deferiprone in iron-overloaded patients with sickle cell disease or other anemias;Secondary Objective: To evaluate the efficacy of deferiprone in the treatment of iron overload in patients with sickle cell disease or other anemias who have received deferiprone for up to 3 years; Primary end point(s): Safety: • Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product • Serious adverse events (SAEs): Frequency, severity, time to onset, duration, and relatedness to study product • Number of discontinuations due to AEs ; Timepoint(s) of evaluation of this end point: • Hematology: Group 1: Monthly up to Visit 9 (End of Study) or the Early Termination visit; Group 2: Weekly up to Visit 3 (Week 26), then biweekly up to Visit 5 (Week 52), then monthly up to Visit 9 (End of Study) or the Early Termination visit • Biochemistry: Visit 1 and semi-annually up to Visit 9 or the Early Termination visit • Serology: Visits 1 and 9	—

Measure

Time frame

Main Objective: To evaluate the long-term safety and tolerability of deferiprone in iron-overloaded patients with sickle cell disease or other anemias;Secondary Objective: To evaluate the efficacy of deferiprone in the treatment of iron overload in patients with sickle cell disease or other anemias who have received deferiprone for up to 3 years; Primary end point(s): Safety: • Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product • Serious adverse events (SAEs): Frequency, severity, time to onset, duration, and relatedness to study product • Number of discontinuations due to AEs ; Timepoint(s) of evaluation of this end point: • Hematology: Group 1: Monthly up to Visit 9 (End of Study) or the Early Termination visit; Group 2: Weekly up to Visit 3 (Week 26), then biweekly up to Visit 5 (Week 52), then monthly up to Visit 9 (End of Study) or the Early Termination visit • Biochemistry: Visit 1 and semi-annually up to Visit 9 or the Early Termination visit • Serology: Visits 1 and 9

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Secondary

Measure	Time frame
Secondary end point(s): Efficacy: For Group 1, the change from baseline to Year 1 is derived from study LA38-0411. For Group 2, there is no Year 3 of deferiprone treatment. • The change from baseline to Year 1 (both groups; Group 1 data are from LA38-0411), from baseline to Year 2 (both groups), and from baseline to Year 3 (Group 1 only) in liver iron concentration (LIC), as measured by magnetic resonance imaging (MRI) • The change from baseline to Year 1 (both groups; Group 1 data are from LA38-0411), from baseline to Year 2 (both groups), and (Group 1 only) from baseline to Year 3 in cardiac MRI T2* • The change from baseline to Year 1 (both groups; Group 1 data are from LA38-0411 data), from baseline to Year 2 (both groups), and from baseline to Year 3 (Group 1 only) in serum ferritin • Responder analysis, defined as the percentage of patients who show a =20% decline from baseline in LIC or serum ferritin or a =20% increase from baseline in cardiac MRI T2* at Year 1 (both groups; Group 1 data are from LA38-0411), at Year 2 (both groups), and at Year 3 (Group 1 only) ; Timepoint(s) of evaluation of this end point: • Serum ferritin: Visit 1 and quarterly up to Visit 9 or the Early Termination visit • Liver MRI scan: Annually, at Visits 1, 5, and 9 or Early Termination visit • Cardiac MRI T2* scan: Annually, at Visits 1, 5, and 9 or Early Termination visit	—

Measure

Time frame

Secondary end point(s): Efficacy: For Group 1, the change from baseline to Year 1 is derived from study LA38-0411. For Group 2, there is no Year 3 of deferiprone treatment. • The change from baseline to Year 1 (both groups; Group 1 data are from LA38-0411), from baseline to Year 2 (both groups), and from baseline to Year 3 (Group 1 only) in liver iron concentration (LIC), as measured by magnetic resonance imaging (MRI) • The change from baseline to Year 1 (both groups; Group 1 data are from LA38-0411), from baseline to Year 2 (both groups), and (Group 1 only) from baseline to Year 3 in cardiac MRI T2* • The change from baseline to Year 1 (both groups; Group 1 data are from LA38-0411 data), from baseline to Year 2 (both groups), and from baseline to Year 3 (Group 1 only) in serum ferritin • Responder analysis, defined as the percentage of patients who show a =20% decline from baseline in LIC or serum ferritin or a =20% increase from baseline in cardiac MRI T2* at Year 1 (both groups; Group 1 data are from LA38-0411), at Year 2 (both groups), and at Year 3 (Group 1 only) ; Timepoint(s) of evaluation of this end point: • Serum ferritin: Visit 1 and quarterly up to Visit 9 or the Early Termination visit • Liver MRI scan: Annually, at Visits 1, 5, and 9 or Early Termination visit • Cardiac MRI T2* scan: Annually, at Visits 1, 5, and 9 or Early Termination visit

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Countries

Canada, Egypt, Saudi Arabia, United Kingdom, United States

Contacts

Public ContactCaroline Fradette

ApoPharma Inc.

cfradett@apopharma.com16479639831

Outcome results

None listed

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