IEDAT01: Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients

Sponsor

Erydel (Industry)

Overall Status

Completed

CT.gov ID

NCT01255358

Collaborator

(none)

Enrollment

Locations

Arm

Duration (Months)

Patients Per Site

1.1

Patients Per Site Per Month

Study Details

Study Description

Brief 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).

Condition or Disease	Intervention/Treatment	Phase
Nervous System Disorder Genetic Syndrome	Drug: Dexamethasone	Phase 2

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).

Study Design

Study Type:

Interventional

Actual Enrollment :

22 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Study Start Date :

Feb 1, 2011

Actual Primary Completion Date :

Sep 1, 2011

Actual Study Completion Date :

Dec 1, 2011

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Ery-Dex Patients treated with monthly treatment of Ery-Dex (dexamethasone sodium phosphate encapsulated in autologous erythrocytes)	Drug: Dexamethasone dexamethasone encapsulated in human erythrocytes. One infusion per month of about 10-15 mg of dexamethasone. The treatment covers 1 month of therapy- Other Names: Dexamethasone sodium phosphate Dex 21P

Arm

Intervention/Treatment

Experimental: Ery-Dex

Patients treated with monthly treatment of Ery-Dex (dexamethasone sodium phosphate encapsulated in autologous erythrocytes)

Drug: Dexamethasone

dexamethasone encapsulated in human erythrocytes. One infusion per month of about 10-15 mg of dexamethasone. The treatment covers 1 month of therapy-

Other Names:

Dexamethasone sodium phosphate

Dex 21P

Outcome Measures

Primary Outcome Measures

Changes in Neurological symptoms assessed by using ICARS score [At the end of 6 months of treatment]
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).

Secondary Outcome Measures

Assessment of safety [During the whole treatment period]
Evaluate the effect of Ery-Dex on treatment emergent adverse events including Serious Adverse Events (SAEs) and laboratory parameters
Vineland adaptive behaviour scale (VABS) [At the end of the study]
To evaluate the effect of Ery-Dexon adaptive behavior measured by VABS

Eligibility Criteria

Criteria

Ages Eligible for Study:

3 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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)