Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients

Genetic Syndrome Clinical Trial

— IEDAT01  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01255358
• Other study ID #: IEDAT 01
• Status: Completed
• Phase: Phase 2
• First received: December 3, 2010
• Last updated: December 27, 2011
• Start date: February 2011
• Est. completion date: December 2011

Study information

• Verified date: December 2011
• Source: Erydel
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 22
• Est. completion date: December 2011
• Est. primary completion date: September 2011
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Ataxia
• Genetic Syndrome
• Nervous System Diseases
• Nervous System Disorder

Intervention

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-

Locations

Country	Name	City	State
Italy	Spedali Civili	Brescia
Italy	University La Sapienza	Rome

Sponsors (1)

Lead Sponsor	Collaborator
Erydel

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Changes in Neurological symptoms assessed by using ICARS score	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).	At the end of 6 months of treatment
Secondary	Assessment of safety	Evaluate the effect of Ery-Dex on treatment emergent adverse events including Serious Adverse Events (SAEs) and laboratory parameters	During the whole treatment period
Secondary	Vineland adaptive behaviour scale (VABS)	To evaluate the effect of Ery-Dexon adaptive behavior measured by VABS	At the end of the study