Ataxia TelangiectasiaNCT04037189
Treatment of Leukemia and Lymphoma in Children With Ataxia Telangiectasia
An observational study of Ataxia Telangiectasia and Leukemia, run by Rabin Medical Center at 1 site in Israel.
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. Read more about this study
- Who is it for?
- Ages 0 to 21 years
- Conditions the study is for
- Ataxia Telangiectasia, Leukemia, Lymphoma
- How many people
- 250 participants
- Where
- 1 siteIsrael
- Study dates
- 28 Jul 2019to 30 Dec 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Children and young adults diagnosed with ataxia telangiectasia and leukemia or lymphoma
Who may be able to join
- Individuals diagnosed with ataxia telangiectasia and leukemia or lymphoma
- Age 0-21
Who cannot join
- -Age greater than 21 years
Trial locations and contacts
1 site in 1 country.
Naomi Litichever, PhD contact
Israel1 site, 1 recruiting
- Recruiting
Schneider Children's Medical Center
Petah Tikva, 4920235
Sarah Elitzur, MD, 97239253669, sarhae@clalit.org.il
Naomi Litichever, PhD, naomilitichever@clalit.org.il
About this study
Official title: Treatment of Leukemia and Lymphoma in Children With Ataxia Telangiectasia- A Retrospective Study
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.
Read the 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
- Medical subject headings
- Ataxia Telangiectasia, Leukemia, Lymphoma
What is being measured
The primary outcome is the main question the study is designed to answer.
Event-free survival
Assess 5 and 3-year event-free survival
Measured: 5 years
Overall survival
Assess 5 and 3-year overall survival
Measured: 5 years
Cumulative incidence of relapse
Assess 5-year cumulative incidence of leukemia/lymphoma relapse
Measured: 5 years
Cumulative incidence of treatment-related mortality
Assess 2-year cumulative incidence of treatment-related mortality
Measured: 2 years
Cumulative incidence of second malignancies
Assess 5-year cumulative incidence of second malignancies
Measured: 5 years
Secondary outcomes (2)
Cause and timing of death
Determine cause of death and timing of death in relation to specific elements of leukemia/lymphoma therapy (by questionnaire)
Measured: 5 years
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
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jul 2019
- Study start28 Jul 2019
- Primary completion28 Oct 2021 (estimated)
- Study completion30 Dec 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ataxia TelangiectasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 250 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Rabin Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Israeli Society for Pediatric Hematology-Oncology, International BFM Study Group
- Responsible party
- Sponsor
- Investigators
- Sarah Elitzur, MD, Principal investigator, Schneider Children's Medical Center, Israel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04037189
- Sponsor's protocol ID
- 0132-19-RMC
- Status last verified
- Aug 2021
- First submitted
- 21 Jul 2019
- Last updated
- 1 Oct 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Leukemia and Lymphoma in Children With Ataxia Telangiectasia- A Retrospective Study
- ClinicalTrials.gov ID
- NCT04037189
- Acronym / sponsor ID
- 0132-19-RMC
- Conditions
- Ataxia Telangiectasia, Leukemia, Lymphoma
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 250 participants (estimated)
- Lead sponsor
- Rabin Medical CenterUniversity, hospital or other
- Collaborators
- Israeli Society for Pediatric Hematology-Oncology, International BFM Study Group
- Study start
- 28 Jul 2019 (actual)
- Primary completion
- 28 Oct 2021 (estimated)
- Study completion
- 30 Dec 2021 (estimated)
- First posted
- 30 Jul 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- 0 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Children and young adults diagnosed with ataxia telangiectasia and leukemia or lymphoma
Key inclusion criteria (2 listed)
- Individuals diagnosed with ataxia telangiectasia and leukemia or lymphoma
- Age 0-21
Key exclusion criteria (1 listed)
- -Age greater than 21 years
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Event-free survival
Time frame: 5 years
Assess 5 and 3-year event-free survival
Overall survival
Time frame: 5 years
Assess 5 and 3-year overall survival
Cumulative incidence of relapse
Time frame: 5 years
Assess 5-year cumulative incidence of leukemia/lymphoma relapse
Cumulative incidence of treatment-related mortality
Time frame: 2 years
Assess 2-year cumulative incidence of treatment-related mortality
Cumulative incidence of second malignancies
Time frame: 5 years
Assess 5-year cumulative incidence of second malignancies
2 secondary outcomes
Cause and timing of death
Time frame: 5 years
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2021.
Related trials for Ataxia Telangiectasia
Active, not recruitingPhase 3
A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)
Ataxia-Telangiectasia, Ataxia-Telangiectasia (A-T)
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingPhase 1/2
A Study of ART0380 for the Treatment of Advanced or Metastatic Solid Tumors
Advanced Cancer, Metastatic Cancer
RecruitingPhase 3
The Cancer of the Pancreas Screening-5 CAPS5)Study
Pancreas Cancer, Peutz-Jeghers Syndrome (PJS)
Active, not recruitingObservational study
The Pancreas Interception Center (PIC) for Early Detection, Prevention, and Novel Therapeutics
Pancreatic Ductal Adenocarcinoma, Pancreatic Cyst
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Naomi Litichever, PhD contact
Sarah Elitzur, MD principal investigator
Schneider Children's Medical Center, Israel
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Ataxia Telangiectasia and Leukemia, run by Rabin Medical Center at 1 site in Israel.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 1 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Sarah Elitzur, MD (97239253669, sarhae@clalit.org.il), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Event-free survival (time frame: 5 years). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Oct 2021.