NCT01255358

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 22 (actual)
• Sponsor: Quince Therapeutics S.p.A. · Industry
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

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

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

Conditions

• Nervous System Disorder
• Genetic Syndrome

Interventions

Type	Name	Description
DRUG	Dexamethasone	Dexamethasone sodium phosphate (2 vials, 250 mg/10 ml each) to be administered via encapsulation into autologous erythrocytes (ERY-DEX system). Final amount administered to the patient: about 10-15 mg of dexamethasone sodium phosphate, encapsulated into autologous blood (2 vials of 250 mg each of dexamethasone sodium phosphate will be used in the ex vivo process together with 50 ml of blood previously taken from the same patient). The treatment has to be repeated at intervals of 30 days (±10 days)

Timeline

• Start date: 2011-02-01
• Primary completion: 2011-09-01
• Completion: 2011-12-01
• First posted: 2010-12-07
• Last updated: 2024-10-10
• Results posted: 2024-10-10

Locations

2 sites across 1 country: Italy