Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients
Information source: Erydel
ClinicalTrials.gov processed this data on August 23, 2015
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Nervous System Disorder; Genetic Syndrome
Intervention: Dexamethasone (Drug)
Phase: Phase 2
Status: Completed
Sponsored by: Erydel Official(s) and/or principal investigator(s):
Luciana Chessa, MD, Principal Investigator, Affiliation: A.O. Sant'Andrea Rome Italy
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).
Clinical Details
Official title: Evaluations of Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Ataxia Teleangiectasia Patients
Study design: Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: Changes in Neurological symptoms assessed by using ICARS score
Secondary outcome: Assessment of safetyVineland adaptive behaviour scale (VABS)
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
Minimum age: 3 Years.
Maximum age: N/A.
Gender(s): Both.
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
Locations and Contacts
Spedali Civili, Brescia 25123, Italy
University La Sapienza, Rome 00185, Italy
Additional Information
Starting date: February 2011
Last updated: December 27, 2011
|
