Pharmacokinetic Study of Deferiprone in Paediatric Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01740713
• Date of registration: 28/11/2012
• Prospective Registration: Yes
• Primary sponsor: Consorzio per Valutazioni Biologiche e Farmacologiche
• Public title: Pharmacokinetic Study of Deferiprone in Paediatric Patients DEEP-1
• Scientific title: Multi-centre, Oral Single Dose Experimental and Modelling Study to Evaluate the Pharmacokinetics of Deferiprone in Patients Aged From 1 Month to Less Than 6 Years of Age Affected by Transfusion-dependent Haemoglobinopathies.
• Date of first enrolment: December 2012
• Target sample size: 23
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01740713
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

Cyprus, Egypt, Italy

Contacts

• Name: Giovanni Carlo Del Vecchio
• Affiliation: Azienda Ospedaliero-Universitaria Consorziale Policlinico di Bari, Italy

• Name: Oscar Della Pasqua
• Affiliation: Universiteit Leiden, The Netherlands

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients in a chronic transfusional program who have received at least 150 ml/kg/year
of packed red blood cells (corresponding approximately to 12 transfusions) and on
current treatment with DFO, DFX, DFP; aged from 1 month to less than 6 years; or

- Patients naïve to any chelation treatment who have received not less than 150 ml/kg of
packed red blood cells (corresponding to approximately 12 transfusions) and have
ferritin levels > 800 ng/mL, aged from 1 month to less than 6 years; or

- Patients who meet the transfusion criteria (150 ml/kg/year corresponding approximately
to 12 transfusions) and have known intolerance or contraindication to DFO

And if all of the following criteria apply:

- Patients affected by any hereditary haemoglobinopathies requiring chronic transfusion
therapy including but not limited to thalassaemia and sickle cell disease

- Written informed consent obtained from their legal guardian on the patient's behalf in
accordance with the national legislations. According to his/her capability, patient's
informed assent will be collected

Exclusion Criteria:

- Patient with known intolerance or contraindication to the trial treatment

- Patient with Hb levels less than 8g/dl (entry may be delayed until values return to
normal)

- Patient with platelet count <100.000/mm3 or absolute neutrophil count <1.500/mm3
(entry may be delayed until values return to normal)

- Patient with evidence of abnormal liver function (ALT level >5 times the upper normal
limit during six months preceding enrolment; entry may be delayed until values return
to normal)

- iron overload from causes other than transfusional haemosiderosis

- severe heart dysfunction secondary to iron overload defined as the occurrence of heart
failure or severe arrhythmia or as indicated by cardiac T2* lower than 10 ms, if
recent MRI data is available,

- Patient with serum creatinine level above the upper normal limit at screening; entry
may be delayed until values return to normal.

- Serological evidence of chronic hepatitis B (presence of HBe Ag, HBsAg, HBcAb-IgM, in
the absence of HBsAb).

- History of significant medical or psychiatric disorder that may impair compliance with
the requirements of the protocol.

- The patient has received another investigational drug within 30 days prior to this
study.

- Patient with a pre-existing condition or any other surgical or medical condition which
might significantly interfere with normal gastrointestinal and hepatic function that
could alter the absorption, metabolism, and/or excretion of the study drug.

- Patient with a known history of HIV seropositivity.

- Fever and other signs/symptoms of infection in the 10 days before drug
administration(treatment day)

- Concomitant use of other iron chelators or trivalent cation-dependent medicinal
products such as aluminium-based antacids.

- Patient with a chronic condition that does not allow suspension of related treatment
from starting of washout until drug is administered.



Age minimum: 1 Month
Age maximum: 6 Years
Gender: All

Health Condition(s) or Problem(s) studied

Chronic Iron Overload

Intervention(s)

Drug: Deferiprone, dose level 1

Drug: Deferiprone, dose level 2

Drug: Deferiprone, dose level 3

Primary Outcome(s)

Css [Time Frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)]

CL/F [Time Frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)]

Tmax [Time Frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)]

V/F [Time Frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)]

AUC (0-8h) [Time Frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)]

Cmax [Time Frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)]

Ka [Time Frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)]

Cmin [Time Frame: Day 1 of single dose treatment (6 sampling time range: from predose up to 8h post first administration)]

Secondary Outcome(s)

Adverse Events [Time Frame: from drug administration up to 8 days post treatment]

Secondary ID(s)

DEEP-1

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

European Commission

Results

• Results available: Yes
• Date Posted: 11/01/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01740713