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Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients

Information source: Erydel
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Nervous System Disorder; Genetic Syndrome

Intervention: Dexamethasone (Drug)

Phase: Phase 2

Status: Completed

Sponsored by: Erydel

Official(s) and/or principal investigator(s):
Luciana Chessa, MD, Principal Investigator, Affiliation: A.O. Sant'Andrea Rome Italy


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).

Clinical Details

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

Study design: Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: Changes in Neurological symptoms assessed by using ICARS score

Secondary outcome:

Assessment of safety

Vineland adaptive behaviour scale (VABS)

Detailed description: 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).


Minimum age: 3 Years. Maximum age: N/A. Gender(s): Both.


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

Locations and Contacts

Spedali Civili, Brescia 25123, Italy

University La Sapienza, Rome 00185, Italy

Additional Information

Starting date: February 2011
Last updated: December 27, 2011

Page last updated: August 23, 2015

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