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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01255358
First Posted: December 7, 2010
Last Update Posted: December 29, 2011
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.
Information provided by (Responsible Party):
Erydel
December 3, 2010
December 7, 2010
December 29, 2011
February 2011
September 2011   (Final data collection date for primary outcome measure)
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).
Same as current
Complete list of historical versions of study NCT01255358 on ClinicalTrials.gov Archive Site
  • 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
  • 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
Same as current
Not Provided
Not Provided
 
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients
Evaluations of Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Ataxia Teleangiectasia Patients
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).
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).
Interventional
Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • Nervous System Disorder
  • Genetic Syndrome
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
Experimental: Ery-Dex
Patients treated with monthly treatment of Ery-Dex (dexamethasone sodium phosphate encapsulated in autologous erythrocytes)
Intervention: Drug: Dexamethasone
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
22
December 2011
September 2011   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: All
3 Years and older   (Child, Adult, Senior)
No
Contact information is only displayed when the study is recruiting subjects
Italy
 
 
NCT01255358
IEDAT 01
No
Not Provided
Not Provided
Erydel
Erydel
Not Provided
Principal Investigator: Luciana Chessa, MD A.O. Sant'Andrea Rome Italy
Erydel
December 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP