Dettagli per singolo studio

DEC-NET Serial number FR540

Published online	06/07/2006 16.01.00
Last updated	06/07/2006 16.00.38

Other protocol ID number	N/A
Current trial status	Planned (i.e. not yet recruiting participants)
Major Disease (ICD9 class)	SICKLE-CELL DISEASE UNSPECIFIED
Experimental drug
HYDROXYCARBAMIDE Treatment regimen (dosage and duration) 15-20mg/kg/day
Gender	Both
Age (range)	3 years and above
Eligibility criteria
Inclusion criteria
-Homozygous patients with sickle cell disease (SS)aged more than 3 years, from sub-Saharan Africa, in one of the folloing categories : 1.Patients treated for more than 3 months by hydroxyurea-HYDREA for a vasoocclusive episode, having required more than 3 hospitalisations/year, with criteria in clinical efficacy. 2. Patients non treated, with 3 or more veno-occlusive episodes having required more than 1 hospitalisations/year 3.Untreated patients, with an asymptomatic sickle cell disease (aged 5 years or more) 4.Control patients: Subjects AA or AS, siblings , parents AS; good condition, not taking any drug at the time of sampling, and after written assent. -Patients initially untreated could be included secondarily in the treated group if a hydroxyurea treatment proves to be necessary (and will be studied in the 2 groups). -Patients not taking other drugs only oracilline,folic acid, hydroxyurea or a martial supplementation. -Informed consent of the adult patient or two parents and the child in age to express it.
Exclusion criteria
-Patient in acute phase of the disease -Refusal of the adult patient, the parents or the child in age to express it. -Other drug taking that oracilline, folic acid, hydroxyurea or a martial supplementation. -Sick control patient. -Drug taking by the Control patient.
Trial design/methodology
Phase	2
Kind of study
Design
Purpose of study
-To determine the cellular origin and the number of blood MPs (microparticles) in sickle disease subjects, untreated (symptomatic or not for vasoocclusive crisis) or treated by hydroxyurea. -To measure the procoagulant and anticoagulant potential of these MPs. -To measure the impact of MPs from patients treated or not with HU on the in vitro activation of the endothelial cells, neutrophilic leukocytes and monocytes. -To study the relation between the cellular origin and the circulating number of MPs and the in vivo markers of activation : (i) coagulation, (ii) leukocytes (circulatingmetalloproteases), in order to validate our hypothesis. -To study the relation between the cellular origin and the circulating number of MPs and vasoocclusive symptomatology.
Primary outcomes
-Cellular origin and properties of the microparticles
Secondary outcomes
-Procoagulant and anticoagulant properties of the MPs
Summary of study design, objectives, and ongoing research findings
This is a prospective study in 60 children and 60 adults with sickle cell disease treated or not by hydroxyurea (HU). The untreated subjects are divided into two groups symptomatic and nonsymptomatic with regard to the vasoocclusive crisis. Objectives: -to determine the cellular origin and the number of circulating MPs -to measure their procoagulant and anticoagulant potential -to measure the impact of MPs from patients treated or not by the HU on endothelial cells, polynuclear neutrophiles and monocytes activation in vitro -to study the relation of the cellular origin , the circulating number of MPs with the markers of the in vivo activation of (i)coagulation, (ii) leucocytes (metalloproteases circulating) -to study the relation of the cellular origin and the circulating number of MPs with vasoocclusive symptomatology. The results include a better comprehension of the physiopathology of vasoocclusion in the sickle cell disease and the mode of action of hydroxyurea, only drug having shown its effectiveness, but the long-term effects are unknown.