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Pharmacokinetic Study of Deferiprone in Paediatric Patients

NCT01740713 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 23

Last updated 2017-01-11

Study results available
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Summary

Deferiprone (DFP) is the most extensively studied oral iron chelator to date. It has been authorised in Europe in 1999 for the treatment of iron overload in patients with beta-thalassaemia major when DFO is contraindicated or inadequate. Despite a wide experience of DFP there are limited experimental data available on DFP in children and no pharmacokinetic data in children under 6 years of age. On the basis of the existing data in adults and adolescent, in the DEEP-1 trial a pharmacokinetic bridging model was developed to support the dose selection in children aged less than 6 years affected by transfusion dependent haemoglobinopathies. The study consisted of two phases, namely an experimental phase, during which patients received a single dose and a modeling phase, during which PK data obtained after single dose in patients \< 6 years of age were analysed in conjunction with historical PK data in adults and older children and adolescents. The model-based analysis of the data obtained after single dose enabled the assessment of the dosing regimen required for the purpose of accurate pharmacokinetic bridging. The ratio between the predicted systemic exposure parameters (AUC and Cmax) in the target population and reference group were used as basis for recommendation of the dose in the target population.

Conditions

  • Chronic Iron Overload

Interventions

DRUG

Deferiprone, dose level 1

a solution at 80 mg/mL will be administered orally

DRUG

Deferiprone, dose level 2

a solution at 80 mg/mL will be administered orally

DRUG

Deferiprone, dose level 3

a solution at 80 mg/mL will be administered orally

Sponsors & Collaborators

  • European Commission

    collaborator OTHER

  • Consorzio per Valutazioni Biologiche e Farmacologiche

    lead OTHER

Principal Investigators

  • Oscar Della Pasqua · Universiteit Leiden, The Netherlands

  • Giovanni Carlo Del Vecchio · Azienda Ospedaliero-Universitaria Consorziale Policlinico di Bari, Italy

Study Design

Allocation
RANDOMIZED
Masking
SINGLE
Model
PARALLEL

Eligibility

Min Age
1 Month
Max Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-12-31
Primary Completion
2013-12-31
Completion
2014-02-28

Countries

  • Cyprus
  • Egypt
  • Italy

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01740713 on ClinicalTrials.gov