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Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients (IEDAT01)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01255358
Recruitment Status : Completed
First Posted : December 7, 2010
Last Update Posted : December 29, 2011
Information provided by (Responsible Party):

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).

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Study Type : Interventional  (Clinical Trial)
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 : February 2011
Actual Primary Completion Date : September 2011
Actual Study Completion Date : December 2011

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

Primary Outcome Measures :
  1. Changes in Neurological symptoms assessed by using ICARS score [ Time Frame: 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 :
  1. Assessment of safety [ Time Frame: During the whole treatment period ]
    Evaluate the effect of Ery-Dex on treatment emergent adverse events including Serious Adverse Events (SAEs) and laboratory parameters

  2. Vineland adaptive behaviour scale (VABS) [ Time Frame: At the end of the study ]
    To evaluate the effect of Ery-Dexon adaptive behavior measured by VABS

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   3 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01255358

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Spedali Civili
Brescia, Italy, 25123
University La Sapienza
Rome, Italy, 00185
Sponsors and Collaborators
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Principal Investigator: Luciana Chessa, MD A.O. Sant'Andrea Rome Italy
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Responsible Party: Erydel Identifier: NCT01255358    
Other Study ID Numbers: IEDAT 01
First Posted: December 7, 2010    Key Record Dates
Last Update Posted: December 29, 2011
Last Verified: December 2011
Keywords provided by Erydel:
Ataxia Teleangiectasia
Dexamethasone sodium phosphate
Additional relevant MeSH terms:
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Nervous System Diseases
Neurologic Manifestations
Signs and Symptoms
Dexamethasone acetate
Dexamethasone 21-phosphate
BB 1101
Anti-Inflammatory Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action