A Study to Learn More About the Health of Persons With Down Syndrome After Treatment for Acute Leukemia

Официальное название

Chronic Health Conditions in Down Syndrome-Associated Acute Leukemia: The Down Syndrome Phenotyping Acute Leukemia Study in Survivors (DS-PALS Survivors)

Обзор

This study attempts to learn more about the health of persons with Down syndrome after treatment for acute leukemia. Children with Down syndrome are at increased risk for side effects during treatment for acute leukemia, but it is unclear of their risk for long-term effects of cancer treatment. By learning more about the factors that may contribute to chronic health conditions and long-term effects after treatment for leukemia in persons with Down syndrome, clinical practice guidelines for survivorship care can be developed to help improve their quality-of-life.

Подробное описание

PRIMARY OBJECTIVE:

I. To determine the prevalence, type, and severity of chronic health conditions (CHC) in survivors of Down syndrome-associated acute leukemia (DS-AL), and to compare CHC with frequency-matched DS individuals that have no cancer history.

SECONDARY OBJECTIVES:

I. To characterize post-treatment clinical outcomes of DS-AL by prospective, in-person assessment.

II. To determine the prevalence and severity of parent-reported adverse neuropsychological (NP) outcomes in survivors of DS-AL, compared with frequency-matched DS individuals with no cancer history.

III. To determine health-related quality of life (HRQOL) in survivors of DS-AL, compared with frequency-matched DS individuals with no cancer history.

IV. To identify clinical risk determinants of CHC, NP, and clinical outcomes in survivors of DS-AL.

V. To establish a well-annotated cohort of survivors of DS-AL and associated biobank as a resource for future investigations.

EXPLORATORY OBJECTIVES:

I. For DS-acute lymphoblastic leukemia (DS-ALL), test if structural birth defects and genetic associations with etiology extend to CHC.

II. For DS-ALL, test if telomere length determined by polygenic risk score and telomere flow-fluorescence in situ hybridization (FISH) are associated with outcomes from in-person NP assessment.

OUTLINE:

Patients undergo an optional saliva/buccal swab in part 1 and clinical assessment in part 2 of the study. Patients with DS-ALL may then undergo blood sample collection and neurocognitive assessment in part 3 of the study.

Вмешательства

• Процедура Biospecimen Collection
  Patients undergo saliva/buccal and blood sample collection
• Другое Clinical Evaluation
  Undergo a clinical assessment
• Другое Neurocognitive Assessment
  Undergo neurocognitive assessment
• Другое Questionnaire Administration
  Ancillary studies
• Другое Survey Administration
  Ancillary studies

Первичные конечные точки

• Prevalence, type, and severity of chronic health conditions (CHC) [Срок оценки: Up to study completion]

Вторичные конечные точки (5)

• Post-treatment clinical outcomes [Срок оценки: Up to study completion]
• Prevalence and severity of parent-proxy neuropsychological (NP) outcomes [Срок оценки: Up to study completion]
• Health-related quality of life (HRQOL) [Срок оценки: Up to study completion]
• Clinical risk determinants of CHC, NP, and clinical outcomes [Срок оценки: Up to study completion]
• Well-annotated cohort of Down syndrome phenotyping acute leukemia study (DS-PALS) survivors and associated biobank [Срок оценки: Up to study completion]

Критерии участия

Критерии включения

• Patients age >= 6 and < 40 years at the time of enrollment
• A diagnosis of Down syndrome is required, and may include any of the three recognized types: trisomy 21 resulting from chromosomal nondisjunction (most common), translocation (the patient has 46 chromosomes, but all or part of an additional copy of chromosome 21 is attached to another chromosome), or mosaicism (trisomy 21 that is present in only a fraction of cells)
• All patients must be DS-AL survivors (acute lymphoblastic leukemia \[ALL\] or acute myeloid leukemia \[AML\])

• Note 1: Myeloid leukemia of Down syndrome (ML-DS) is included in the AML category above. Per the World Health Organization (WHO) definition of ML-DS, this diagnosis encompasses both myelodysplastic syndrome (MDS) and overt AML. Also, note that survivors of relapsed disease are eligible, so long as the patient otherwise meets eligibility criteria, i.e., treatment for relapse was completed at least 36 calendar months prior to enrollment and did not include stem cell transplant
• Note 2: A diagnosis of transient abnormal myelopoiesis (TAM), also known as transient myeloproliferative disease (TMD), is not alone sufficient for inclusion in this study
• Patients must have been treated for ALL or AML

• Note: History of COG therapeutic trial participation is not required. As a reminder ML-DS would be included under the AML category here above
• All cancer treatment (oral or intravenous) must have been completed at least 36 calendar months prior to enrollment
• Patients must have a life expectancy of > 1 year
• Patient and parent of subject must be either English or Spanish speaking. At least one parent or guardian must be able to read and write in English or Spanish

• Note: Parents or guardians are responsible for completing all questionnaires, even in the case of subjects that are >= 18 years old
• All patients and/or their parents or legal guardians must sign a written informed consent
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

Критерии исключения

• Patients with history of hematopoietic stem cell transplant (HSCT) are excluded

• Note: Patients with previous chimeric antigen receptor T-cell (CAR T-cell) therapy, and other cellular cancer therapies can participate, as long as all other eligibility criteria are satisfied
• Patients with a history of cancers prior to their ALL or AML diagnosis are excluded. Patients that developed a subsequent malignant neoplasm following their ALL or AML diagnosis are also excluded

• Note: Prior history of transient abnormal myelopoiesis is allowed, but is not sufficient for eligibility
• Patients whose parents or guardians are unable to complete the required forms are excluded

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Другое

Центры проведения

США · 69 центров

• Children's Hospital of Alabama — Birmingham
• Phoenix Childrens Hospital — Phoenix
• Valley Children's Hospital — Madera
• UCSF Benioff Children's Hospital Oakland — Oakland
• Kaiser Permanente-Oakland — Oakland
• University of California Davis Comprehensive Cancer Center — Sacramento
• Rady Children's Hospital - San Diego — San Diego
• UCSF Medical Center-Mission Bay — San Francisco
• … и ещё 61 центр

Канада · 1 центр

• Children's Hospital — London

Публикации

• Gramatges MM, Sanclemente LN, Hall L, Taylor OA, Nuno MM, Bhatia S, Chow EJ, Getz KD, Hitzler JK, Li AM, McCloskey K, Nathan PC, O'Brien MM, Patel S, Verma A, Yarbrough AR, Richard MA, Rosser TC, Jacola LM, Lupo PJ, Rabin KR. A multicenter observational cohort study in survivors of Down Syndrome-associated acute leukemia (ALTE22C1): a report from the Children's Oncology Group. BMC Cancer. 2025 Oct PMID 41116168

Идентификаторы

NCT: NCT05702645 · ALTE22C1 · NCI-2022-09410 · ALTE22C1 · COG-ALTE22C1 · ALTE22C1 · R01CA263000 · UG1CA189955

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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