Global Directory A-T Global Directory

Clinical Trials

Clinical trials are research studies conducted in human beings with the goal of answering specific questions about new therapies, vaccines or diagnostic procedures, or new ways of using known treatments. They are used to determine whether new drugs, diagnostics or treatments are both safe and effective. Carefully conducted clinical trials are the fastest and safest way to find treatments that help people.

After researchers test investigational new therapies or procedures in the laboratory and in animal studies, those with the most promising possibilities are moved into human clinical trials. Clinical trials are broken down into different phases. During a trial, more and more information is gained about the potential treatment, its risks and how well it may or may not work, along with aspects related to quality of life.

The list below highlights some of the clinical trials which are are currently underway in various locations around the world. For a more comprehensive list of all clinical trials relating to Ataxia Telangiectasia, please visit or here for a useful lay summary of some of the current trials. (If you are aware of a trial which is not shown below or on the website, please contact us using the email address listed at the bottom of this page).

To Evaluate the Effects of EryDex in Patients With A-T (NEAT) This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate [DSP] in autologous erythrocytes), administered by intravenous (IV) infusion once every 28 days, on neurological symptoms of patients with Ataxia Telangectasia (A-T). USA Belgium Denmark Germany Italy Norway Poland Spain UK
A-T Treatment with the EryDex SysTem This large placebo controlled, cross-over trial is being performed to assess the safety and efficacy of the EryDex System (dexamethasone sodium phosphate loaded red blood cells) to treat neurological symptoms associated with A-T USA Belgium Germany Italy Norway Poland Spain Tunisia UK
Home-Based Exercise Intervention for Children with A-T This clinical trial aims to explore the effectiveness, feasibility, and acceptability of a co-produced home-based exercise intervention including yoga and breathing exercises, for children with A-T. To participate, please contact Munira at UK
Treating Mitochondrial Dysfunction With a Novel Form of Anaplerosis This combined phase 2a/b trial will attempt to determine the optimal dose, demonstrate tolerability, and examine the efficacy and safety of triheptanoin in participants with A-T Australia
N-Acetyl-L-Leucine for A-T The primary purpose of the study is to evaluate the safety and efficacy of N-Acetyl-L-Leucine (IB1001) in the treatment of A-T investigating the efficacy in terms of improving symptoms, functioning, and quality of life against the defined endpoints in patients with A-T USA Germany Spain UK
Neurofilament Light Chain in A-T This trial aims to measure and evaluate neurofilament light chain as serum biomarker of disease progression in A-T patients Germany