An open label, phase IIa study to evaluate the safety, tolerability, pharmacokinetics and the effects on liver iron concentration of repeated doses of 10 mg/Kg/day of ICL670 administered of pediatric patients with transfusion-dependent B-thalassemia major
DEC-NET Serial number IT426
Published online24/06/2005 15.27.00
Last updated24/06/2005 15.29.13
Other protocol ID number
Current trial statusComplete(closed to recruitment of participants: follow-up complete)
Major Disease
(ICD9 class)		Thalassemia
Experimental drug
CICL670A
GenderBoth
Age (range)2-18 years

Eligibility criteria
Inclusion criteria
- Thalassemia major - Transfusion-dependent - Treatment with deferoxamina for 4 weeks - Ferritin greater than 1000 mg/ml; LIC greater than 2.5 mg/g dw
Exclusion criteria
- Drepanocytosis - Hepatitis B and/or C - Serum creatine above average - ALT>250 U/L - Hypertension - Clinically significant cardiac problems - Infections in last 10 days: cataract or significant eye problems, vit. E deficiency - Systematic diseases - Psychiatric problems - Enuresis - Pregancy or breastfeeding - medical conditions that impede or hamper drug absorption, distribution, metabolism, or excretion - HIV - Non-compliance - Patient cannot undergo foreseen lab tests - Alcohol or drug abuse

Trial design/methodology
Phase2
Kind of studyEfficacy
Safety
Design
Purpose of study
Safety . Efficacy - Pharmacokinetics
Summary of study design, objectives, and ongoing research findings
An open label, phase IIa study to evaluate the safety, tolerability, pharmacokinetics and the effects on liver iron concentration of repeated doses of 10 mg/Kg/day of ICL670 administered of pediatric patients with transfusion-dependent B-thalassemia major
Principal investigator
NameDott. G.L. Forni
InstitutionE. O. Ospedali Galliera . Centro della Microcitemia
Postal addressVia Volta 6
CityGenova
CountryITALY
Phone+390105634557
Fax+390105634556
E-mailgianluca.forni@galliera.it


International lead principal investigator (for international trials)
NameGian Luca Forni
InstitutionE.O. Ospedali Galliera . Centro della Microcitemia
Postal addressVia Volta 6
CityGenova
CountryITALY
Phone010 5634557
Fax010 5634556
E-mail


Participating countries
ITALY
FRANCE


Participating centres
Centro della Microcitemia E.O. Ospedali Galliera (Genova)
DH talassemia dell.età volutiva Ospedale Regionale Microcitemie USL 21 (Cagliari)
Centro Microcitemia Dip. Scienze Pediatriche Univ. Osp. Regina Mergherita (Torino)
Centre Hospitalier Saint Vincent (Lille - Francia)

ISRCTN  EudraCT