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Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Teleangiectasia Patients

NCT01255358 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 22

Last updated 2024-10-10

Study results available
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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).

Conditions

  • Nervous System Disorder
  • Genetic Syndrome

Interventions

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)

Sponsors & Collaborators

  • Quince Therapeutics S.p.A.

    lead INDUSTRY

Principal Investigators

  • Luciana Chessa, MD · A.O. Sant'Andrea Rome Italy

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-02-28
Primary Completion
2011-09-30
Completion
2011-12-31

Countries

  • Italy

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01255358 on ClinicalTrials.gov