Treatment of Leukemia and Lymphoma in Children With Ataxia Telangiectasia- A Retrospective Study

Trial Information
Status: Recruiting
Location: See location...
Study Type: Observational

Ataxia telangiectasia (A-T) is a multisystem disease with diverse manifestations, including progressive neurodegeneration, immunodeficiency, respiratory disease, and genomic instability. One of the most important features of A-T is the increased predisposition to cancer, especially to lymphoid malignancies. Patients with A-T are generally excluded from collaborative clinical trials, their treatment outcomes and toxicity profiles have rarely been reported, and little is currently known concerning the treatment intensity required to provide a reasonable balance between efficacy and toxicity. The aims of this study are to build a large international de-identified database of children with A-T treated for leukemia and lymphoma, to investigate epidemiology and outcome of treatment, toxicity profiles and risk factors which impact outcome, in order to eventually enable the generation of data-based treatment recommendations for this population.

Am I eligible for this trial?
Participation Requirements
Maximum Age:
Healthy Volunteers:

• Individuals diagnosed with ataxia telangiectasia and leukemia or lymphoma

• Age 0-21

Where is this trial taking place?
Other Locations
Schneider Children's Medical Center
Petah Tikva
Who do I contact about this trial?
Sarah Elitzur, MD
Naomi Litichever, PhD
When is this trial taking place?
Start Date: July 28, 2019
Estimated Completion Date: December 30, 2021
How many participants will be in this trial?
Target number of participants: 250
Who are the authors of this trial?

This content was sourced from

Effects of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T): A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study
Who is this study for:Child to adult patients with Ataxia Telangiectasia
Start Date:January 8, 2020
Study Drug:N-Acetyl-L-Cysteine
Study Type:Drug
Phase: Phase 2
Neurofilament Light- Chain as Biomarker for Neurodegeneration in Ataxia Telangiectasia
Status:Enrolling by invitation
Start Date:February 1, 2020
Study Type:Procedure