Pharmacokinetic study of deferiprone in paediatric patients

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 30

Inclusion Criteria

a) 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.
b) 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.
c) 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:
d) Patients affected by any hereditary haemoglobinopathies requiring
chronic transfusion therapy including but not limited to thalassaemia
and sickle cell disease
e) 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
Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

a) Patient with known intolerance or contraindication to the trial
treatment
b) Patient with Hb levels less than 8g/dl (entry may be delayed until
values return to normal)
c) Patient with platelet count <100.000/mm3 or absolute neutrophil
count <1.500/mm3 (entry may be delayed until values return to normal)
d) 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)
e) iron overload from causes other than trasfusional haemosiderosis
f) 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,
g) Patient with serum creatinine level above the upper normal limit at
screening; entry may be delayed until values return to normal.
h) Serological evidence of chronic hepatitis B (presence of HBe Ag,
HBsAg, HBcAb-IgM, in the absence of HBsAb).
i) History of significant medical or psychiatric disorder that may impair
compliance with the requirements of the protocol.
j) The patient has received another investigational drug within 30 days
prior to this study.
k) 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.
l) Patient with a known history of HIV seropositivity.
m) Fever and other signs/symptoms of infection in the 10 days before
drug administration(treatment day)
n) Concomitant use of other iron chelators or trivalent cation-dependent
medicinal products such as aluminium-based antacids.
o) Patient with a chronic condition that does not allow suspension of
related treatment from starting of washout until drug is administered.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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