Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients (IEDAT-01)

Quince Therapeutics S.p.A.

Status and phase

Completed

Phase 2

Conditions

Genetic Syndrome

Nervous System Disorder

Treatments

Drug: Dexamethasone

Study type

Interventional

Funder types

Industry

Identifiers

NCT01255358

IEDAT-01

2010-022315-19 (EudraCT Number)

Details and patient eligibility

About

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 treatment with ERY-DEX (dexamethasone sodium phosphate ex vivo encapsulated into human autologous erythrocytes).

Full 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). The study consists of a screening period (max duration of 30 days) and a treatment period (duration 6 months).

Enrollment

22 patients

Sex

All

Ages

3+ years old

Volunteers

No Healthy Volunteers

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)
written IC to participate.

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
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