Treatment of Leukemia and Lymphoma in Children With Ataxia Telangiectasia

Lymphoma Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04037189
• Other study ID #: 0132-19-RMC
• Status: Recruiting
• Start date: July 28, 2019
• Est. completion date: December 30, 2021

Study information

• Verified date: August 2021
• Source: Rabin Medical Center
• Contact: Sarah Elitzur, MD
• Phone: 97239253669
• Email: sarhae[@]clalit.org.il
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 250
• Est. completion date: December 30, 2021
• Est. primary completion date: October 28, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 21 Years

Eligibility

Inclusion Criteria:

• Individuals diagnosed with ataxia telangiectasia and leukemia or lymphoma

• Age 0-21

Exclusion Criteria:

-Age greater than 21 years

Related Conditions & MeSH terms

• Ataxia
• Ataxia Telangiectasia
• Cerebellar Ataxia
• Leukemia
• Lymphoma
• Telangiectasis

Locations

Country	Name	City	State
Israel	Schneider Children's Medical Center	Petah Tikva

Sponsors (3)

Lead Sponsor	Collaborator
Rabin Medical Center	International BFM Study Group, Israeli Society for Pediatric Hematology-Oncology

Country where clinical trial is conducted

Israel, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Event-free survival	Assess 5 and 3-year event-free survival	5 years
Primary	Overall survival	Assess 5 and 3-year overall survival	5 years
Primary	Cumulative incidence of relapse	Assess 5-year cumulative incidence of leukemia/lymphoma relapse	5 years
Primary	Cumulative incidence of treatment-related mortality	Assess 2-year cumulative incidence of treatment-related mortality	2 years
Primary	Cumulative incidence of second malignancies	Assess 5-year cumulative incidence of second malignancies	5 years
Secondary	Cause and timing of death	Determine cause of death and timing of death in relation to specific elements of leukemia/lymphoma therapy (by questionnaire)	5 years
Secondary	Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	2 years