Treatment of Leukemia and Lymphoma in Children With Ataxia Telangiectasia
1 other identifier
observational
250
1 country
1
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jul 2019
Typical duration for all trials
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 21, 2019
CompletedStudy Start
First participant enrolled
July 28, 2019
CompletedFirst Posted
Study publicly available on registry
July 30, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 28, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
December 30, 2021
CompletedOctober 1, 2021
August 1, 2021
2.3 years
July 21, 2019
September 30, 2021
Conditions
Outcome Measures
Primary Outcomes (5)
Event-free survival
Assess 5 and 3-year event-free survival
5 years
Overall survival
Assess 5 and 3-year overall survival
5 years
Cumulative incidence of relapse
Assess 5-year cumulative incidence of leukemia/lymphoma relapse
5 years
Cumulative incidence of treatment-related mortality
Assess 2-year cumulative incidence of treatment-related mortality
2 years
Cumulative incidence of second malignancies
Assess 5-year cumulative incidence of second malignancies
5 years
Secondary Outcomes (2)
Cause and timing of death
5 years
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
2 years
Eligibility Criteria
Children and young adults diagnosed with ataxia telangiectasia and leukemia or lymphoma
You may qualify if:
- Individuals diagnosed with ataxia telangiectasia and leukemia or lymphoma
- Age 0-21
You may not qualify if:
- Age greater than 21 years
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Rabin Medical Centerlead
- Israeli Society for Pediatric Hematology-Oncologycollaborator
- International BFM Study Groupcollaborator
Study Sites (1)
Schneider Children's Medical Center
Petah Tikva, 4920235, Israel
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Sarah Elitzur, MD
Schneider Children's Medical Center, Israel
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- RETROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 21, 2019
First Posted
July 30, 2019
Study Start
July 28, 2019
Primary Completion
October 28, 2021
Study Completion
December 30, 2021
Last Updated
October 1, 2021
Record last verified: 2021-08