Treatment of Leukemia and Lymphoma in Children With Ataxia Telangiectasia

NCT ID: NCT04037189

Last Updated: 2021-10-01

Basic Information

• Recruitment Status: UNKNOWN
• Total Enrollment: 250 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2019-07-28
• Study Completion Date: 2021-12-30

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.

Detailed Description

Ataxia telangiectasia (A-T) is a multisystem disease with diverse manifestations, including progressive neurodegeneration, immunodeficiency, respiratory disease, and genomic instability. A-T is caused by biallelic mutations in the ATM gene, a major activator of the cellular response to DNA double strand breaks. One of the most important features of A-T is the increased predisposition to cancer. Lymphoid malignancies represent the majority of cancers. The treatment of cancer in children with A-T is extremely challenging, due to severe co-morbidities and a significantly increased risk of cancer therapy-related toxicities. 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 optimal treatment approach is controversial; some advocate treatment by standard chemotherapeutic protocols, while others advise initial protocol modifications with chemotherapy dose reductions. Due to the rarity of this disorder, there is an unmet need for an international collaboration for data collection concerning treatment, toxicity and outcome in children with cancer and A-T. Data will be collected from patient files, including patient characteristics and history, AT manifestations, malignancy characteristics, treatment, chemotherapy doses, treatment response, toxicity and outcome.

The aims of the 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.

This study will not involve the use of specimens or participant contact. All the data required have already been collected during the treatment of the participants, and is available in patient records.

Conditions

Ataxia Telangiectasia; Leukemia; Lymphoma

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: RETROSPECTIVE

Eligibility Criteria

Inclusion Criteria

• Individuals diagnosed with ataxia telangiectasia and leukemia or lymphoma
• Age 0-21

Exclusion Criteria

-Age greater than 21 years

• Minimum Eligible Age: 0 Years
• Maximum Eligible Age: 21 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Israeli Society for Pediatric Hematology-Oncology

OTHER

Sponsor Role: collaborator

International BFM Study Group

NETWORK

Sponsor Role: collaborator

Rabin Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Sarah Elitzur, MD

Role: PRINCIPAL_INVESTIGATOR

Schneider Children's Medical Center, Israel

Locations

Schneider Children's Medical Center

Petah Tikva, , Israel

Site Status: RECRUITING

Countries

Israel

Central Contacts

Sarah Elitzur, MD

Role: CONTACT

sarhae@clalit.org.il

97239253669

Naomi Litichever, PhD

Role: CONTACT

naomilitichever@clalit.org.il

Facility Contacts

Sarah Elitzur, MD

Role: primary

sarhae@clalit.org.il

97239253669

Naomi Litichever, PhD

Role: backup

naomilitichever@clalit.org.il

Other Identifiers

0132-19-RMC

Identifier Type: -

Identifier Source: org_study_id