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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Erydel
ClinicalTrials.gov Identifier:
NCT01255358
First received: December 3, 2010
Last updated: December 27, 2011
Last verified: December 2011
History of Changes
  Purpose

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 Intervention Phase
Nervous System Disorder
Genetic Syndrome
 Drug: Dexamethasone
 Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Evaluations of Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Ataxia Teleangiectasia Patients

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Erydel:

Primary Outcome Measures:
  • Changes in Neurological symptoms assessed by using ICARS score [ Time Frame: At the end of 6 months of treatment ] [ Designated as safety issue: No ]
    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 [ Time Frame: During the whole treatment period ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
    To evaluate the effect of Ery-Dexon adaptive behavior measured by VABS


Enrollment: 22
Study Start Date: February 2011
Study Completion Date: December 2011
Primary Completion Date: September 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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).

  Eligibility

Ages Eligible for Study:   3 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01255358

Locations
Italy
Spedali Civili
Brescia, Italy, 25123
University La Sapienza
Rome, Italy, 00185
Sponsors and Collaborators
Erydel
Investigators
Principal Investigator: Luciana Chessa, MD A.O. Sant'Andrea Rome Italy
  More Information

No publications provided

Responsible Party: Erydel
ClinicalTrials.gov Identifier: NCT01255358     History of Changes
Other Study ID Numbers: IEDAT 01
Study First Received: December 3, 2010
Last Updated: December 27, 2011
Health Authority: Italy: The Italian Medicines Agency

Keywords provided by Erydel:
Ataxia Teleangiectasia
AT
Ery-Dex
Dexamethasone
Dexamethasone sodium phosphate

Additional relevant MeSH terms:
Ataxia
Nervous System Diseases
Dyskinesias
Neurologic Manifestations
Signs and Symptoms
Dexamethasone acetate
Dexamethasone
Dexamethasone 21-phosphate
Sodium phosphate
BB 1101
Anti-Inflammatory Agents
Therapeutic Uses
Pharmacologic Actions
Antiemetics
 Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Central Nervous System Agents
Gastrointestinal Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Cathartics

ClinicalTrials.gov processed this record on May 22, 2013