Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients

• Status: Completed
• Trial end date: 2011-12-01
• Target enrollment: 0
• Participant gender: All

Summary

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).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Erydel
• Treatments: BB 1101; Dexamethasone; Dexamethasone 21-phosphate; Dexamethasone acetate

Criteria

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)

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