Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients (IEDAT01)
Multi-centre, single arm, open label, 6 months, phase II study to evaluate the effect of Ery-Dex in improving Central Nervous System (CNS) symptoms in patients with Ataxia Teleangiectasia (AT). The study consists of a screening period (max duration of 30 days) and a treatment period (duration 6 months).
|Study Design:||Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Evaluations of Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Ataxia Teleangiectasia Patients|
- Changes in Neurological symptoms assessed by using ICARS score [ Time Frame: At the end of 6 months of treatment ] [ Designated as safety issue: No ]ICARS is a 100-point semiquantitative scale offering a compartimentalised quantification of 4 subscores: Posture and Gait disorders; Kinetic functions; Speech Disorders and Oculomotor Disorders, for a possible total of 100 (highest score corresponds to the worst status of the patient).
- Assessment of safety [ Time Frame: During the whole treatment period ] [ Designated as safety issue: Yes ]Evaluate the effect of Ery-Dex on treatment emergent adverse events including Serious Adverse Events (SAEs) and laboratory parameters
- Vineland adaptive behaviour scale (VABS) [ Time Frame: At the end of the study ] [ Designated as safety issue: No ]To evaluate the effect of Ery-Dexon adaptive behavior measured by VABS
|Study Start Date:||February 2011|
|Study Completion Date:||December 2011|
|Primary Completion Date:||September 2011 (Final data collection date for primary outcome measure)|
Patients treated with monthly treatment of Ery-Dex (dexamethasone sodium phosphate encapsulated in autologous erythrocytes)
dexamethasone encapsulated in human erythrocytes. One infusion per month of about 10-15 mg of dexamethasone. The treatment covers 1 month of therapy-
The study has the aim to evaluate the improvement in CNS symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with AT, during a period of treatment with Ery-Dex (dexamethasone sodium phosphate ex vivo encapsulated into human autologous erythrocytes).