OLE_NEATEryDex for Ataxia Telangiectasia Patients
Dexamethasone sodium phosphate
Primary Immunodeficiency Diseases+19
+ Ataxia
+ Ataxia Telangiectasia
Treatment Study
Summary
Study start date: December 11, 2024
Actual date on which the first participant was enrolled.This clinical study is designed to continue providing a treatment called EryDex to patients with a condition known as Ataxia Telangiectasia, who have already participated in an earlier study. Ataxia Telangiectasia is a rare genetic disorder affecting movement and immune system function. The main aim is to observe how patients respond to the treatment over a longer period. This study is important because it offers continuous treatment to those who have already been involved in previous research, potentially providing further insights into the effectiveness and safety of EryDex for managing symptoms of this challenging condition. Participants in this study will receive the EryDex treatment for 12 months. EryDex involves a special process where a medication called dexamethasone sodium phosphate is delivered inside the patient’s own red blood cells. This is done to help manage the symptoms of Ataxia Telangiectasia. Throughout the study, researchers will closely monitor the participants to ensure there are no safety issues and to gather more information on the treatment's impact on their health. By the end of the study, each participant will undergo a final safety check to complete their involvement.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.105 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.Over 6 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Criteria
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.One single intervention group is designated in this study
This study does not include a placebo group
Treatment Groups
Group I
ExperimentalStudy Objectives
Primary Objectives
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 22 locations
Great Ormond Street Hospital for Children, Zayed Centre for Research
London, United KingdomUniversity of California Los Angeles (UCLA), Ataxia Center and HD Center of excellence
Los Angeles, United StatesThe Johns Hopkins Hospital, Division of pediatric allergy and immunology
Baltimore, United States