Matched Targeted Therapy (MTT) Recommendation for Patients With Recurrent, Refractory, or High Risk Leukemias and Myelodysplastic Syndrome
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 338
- 试验地点
- 21
- 主要终点
- Rate of Patients With Actionable Alterations
研究概览
简要总结
This research study is seeking to gain new knowledge about Recurrent, Refractory, or High Risk Leukemias in children and young adults.
This study is evaluating the use of specialized testing called leukemia profiling. Once the profiling is performed, the results are evaluated by an expert panel of physicians, scientists and pharmacists. This may result in a recommendation for a specific cancer therapy or a clinical trial called matched targeted therapy (MTT). The results of the leukemia profiling and, if applicable, the MTT recommendation will be communicated to the participant's primary oncologist.
详细描述
Our tissues and organs are made up of cells. Cancer occurs when the molecules that normally control cell growth are damaged. The damage results in unchecked cell growth which causes a tumor, a collection of cancer cells. The damage is referred to as an alteration. There are different types of cancer-causing alterations. Genes are the part of cells that contain the instructions which tell our cells how to make the right proteins to grow and work. Genes are composed of Deoxyribonucleic Acid (DNA) letters that spell out these instructions.
By participating in this study, the participant's leukemia cells will be tested for cancer causing alterations. This testing is called leukemia profiling. The leukemia profiling will be performed using bone marrow or blood that has already been obtained during a clinical test. Alternately, the profiling may be done on leukemia cells that are planned to be obtained as part of routine clinical care.
This study will determine whether it is possible to use profiling results to determine a matched targeted therapy for patients with leukemia. It will describe the range of mutations found in patients with leukemia with this type of profiling, and describe the clinical outcomes of patients who receive a matched targeted therapy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- — 至 30 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Birth to ≤ 30 years at study entry
- •Diagnosis: Patients will be enrolled in one of the two cohorts based on diagnosis:
- •Cohort 1: Relapsed/refractory leukemia
- •Acute lymphoblastic leukemia (ALL), first or greater relapse
- •Acute myeloid leukemia (AML), first or greater relapse
- •Leukemia refractory to induction chemotherapy
- •Other recurrent leukemia
- •Myelodysplastic syndrome (MDS), first or greater relapse, or refractory to initial therapy
- •Cohort 2: New diagnosis
- •Acute myeloid leukemia (AML), new diagnosis
- •New diagnosis infant mixed-lineage leukemia (MLL)-rearranged ALL or low hypodiploid (<40 chromosomes) ALL
- •Rare leukemia- e.g., juvenile myelomonocytic leukemia (JMML), leukemia of ambiguous lineage
- •Secondary leukemia
- •Myelodysplastic syndrome (MDS) not eligible for stem cell transplant
- •Pathologic Criteria
- •Histologic confirmation of leukemia at the time of diagnosis or recurrence
- •Specimen Samples
- •Sufficient leukemia specimen available for profiling from diagnosis or recurrence OR bone marrow aspirate/blood draw/pheresis/other fresh sample of patient leukemia cells planned for clinical care anticipated to allow collection of minimum specimen for testing.
排除标准
- •Insufficient leukemia specimen available for profiling from diagnosis or recurrence; or bone marrow evaluation/blood draw/other leukemia cell sample NOT planned to be obtained for clinical care; or peripheral blast percentage <20% AND clinical blood draw not planned.
研究组 & 干预措施
New Diagnosis
Cohort 2: New Diagnosis
- Acute myeloid leukemia (AML), new diagnosis (excluding acute promyelocytic leukemia (APL))
- New diagnosis infant mixed-lineage leukemia (MLL)-rearranged ALL or low hypodiploid (<40 chromosomes) ALL
- Rare leukemia- e.g., juvenile myelomonocytic leukemia (JMML), leukemia of ambiguous lineage
- Secondary leukemia
- Myelodysplastic syndrome (MDS) not eligible for stem cell transplant
After the screening procedures confirms eligibility:
- Leukemia Profiling will be performed
- Identifying an actionable genomic alteration and making a matched targeted therapy treatment recommendation.
干预措施: Leukemia Profiling (Genetic)
Relapsed/Refractory Leukemia
Cohort 1: Relapsed/Refractory Leukemia
- Acute lymphoblastic leukemia (ALL), first or greater relapse
- Acute myeloid leukemia (AML), first or greater relapse
- Leukemia refractory to induction chemotherapy
- Other recurrent leukemia
- Myelodysplastic syndrome (MDS), first or greater relapse, or refractory to initial therapy
After the screening procedures confirms patient eligibility:
- Leukemia Profiling will be performed
- Identifying an actionable genomic alteration and making a matched targeted therapy treatment recommendation.
干预措施: Leukemia Profiling (Genetic)
结局指标
主要结局
Rate of Patients With Actionable Alterations
时间窗: Actionable alteration was identified based on a combination of fluorescence in situ hybridization (FISH)/cytogenetics and sequencing. Average time to full results was 2 weeks.
An actionable alteration was defined as a cancer-associated genomic event for which there was a targeted drug available. Actionable genetic alterations were identified by a combination of standard-of-care cytogenetics/fluorescence in situ hybridization (FISH) and sequencing performed as part of the study. This study is considered feasible if at least 13% of participants analyzed have profile data and identifiable actionable alterations. A 90% exact binomial confidence interval is presented for the rate of patients with actionable alterations to compare the observed rate against 13%.
次要结局
- Rate of Somatic Genomic Alterations(2 Years)
- Rate of Results Reporting(2 Years)
- Parent's Feelings and Understanding of Genomic Testing(2 Years)
- Analysis of Primary Leukemia Sensitivity Testing and Establishment of Xenograft Models(2 Years)
研究者
Yana Pikman, MD
Principal Investigator
Dana-Farber Cancer Institute