Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients

NCT ID: NCT01255358

Last Updated: 2024-10-10

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 22 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-02-28
• Study Completion Date: 2011-12-31

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 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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

ERY-DEX

Patients treated with monthly treatment of ERY-DEX (dexamethasone sodium phosphate encapsulated in autologous erythrocytes)

Group Type: EXPERIMENTAL

Dexamethasone

Intervention Type: DRUG

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)

Interventions

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)

Intervention Type: DRUG

Other Intervention Names

Dexamethasone sodium phosphate; Dex 21P

Eligibility 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)
• 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

• Minimum Eligible Age: 3 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Quince Therapeutics S.p.A.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Luciana Chessa, MD

Role: PRINCIPAL_INVESTIGATOR

A.O. Sant'Andrea Rome Italy

Locations

Spedali Civili

Brescia, , Italy

University La Sapienza

Rome, , Italy

Countries

Italy

Other Identifiers

2010-022315-19

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

IEDAT-01

Identifier Type: -

Identifier Source: org_study_id