Status:
COMPLETED
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients
Lead Sponsor:
Quince Therapeutics S.p.A.
Conditions:
Nervous System Disorder
Genetic Syndrome
Eligibility:
All Genders
3+ years
Phase:
PHASE2
Brief Summary
The study has the aim to evaluate the improvement in CNS symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with ataxia teleangectasia (AT), during a period of tre...
Detailed Description
This is a 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 Teleangectasia (AT...
Eligibility Criteria
Inclusion
- 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)
- written IC to participate.
Exclusion
- 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
- Have any other significant disease that in the Investigator's opinion would exclude the patient from the trial
- Females of childbearing potential who were pregnant, breast-feeding or were not using adequate contraceptive methods
Key Trial Info
Start Date :
February 1 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2011
Estimated Enrollment :
22 Patients enrolled
Trial Details
Trial ID
NCT01255358
Start Date
February 1 2011
End Date
December 1 2011
Last Update
October 10 2024
Active Locations (2)
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1
Spedali Civili
Brescia, Italy, 25123
2
University La Sapienza
Rome, Italy, 00185