Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients

Evaluations of Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Ataxia Teleangiectasia Patients

The study has the aim to evaluate the improvement in CNS symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with ataxia teleangectasia (AT), during a period of treatment with ERY-DEX (dexamethasone sodium phosphate ex vivo encapsulated into human autologous erythrocytes).

This is a 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 Teleangectasia (AT). The study consists of a screening period (max duration of 30 days) and a treatment period (duration 6 months).

Inclusion Criteria:

• neurological signs of AT
• patients in autonomous gait or helped by a support
• proven molecular diagnosis of AT
• Males and females aged > 3 years
• Body weight >15 kg
• Plasma levels of Lymphocytes CD4+/mm3 > 500 (for patients aged 3-6 years) or > 200 (older than 6 years)
• written IC to participate.

Exclusion Criteria:

• Current or previous neoplastic disease
• History of severe impairment of the immunological system
• Chronic conditions representing a contraindication to the use of steroid drugs
• Non compliance with the study request
• Any previous steroid assumption within 30 days before starting ERY-DEX
• Have any other significant disease that in the Investigator's opinion would exclude the patient from the trial
• Females of childbearing potential who were pregnant, breast-feeding or were not using adequate contraceptive methods