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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT02670525
Other study ID # 15-384
Secondary ID
Status Active, not recruiting
Phase N/A
First received
Last updated
Start date August 17, 2016
Est. completion date May 2027

Study information

Verified date May 2024
Source Dana-Farber Cancer Institute
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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.


Description:

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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 338
Est. completion date May 2027
Est. primary completion date May 16, 2022
Accepts healthy volunteers No
Gender All
Age group N/A to 30 Years
Eligibility Inclusion Criteria: - 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. Exclusion Criteria: - 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.

Study Design


Related Conditions & MeSH terms


Intervention

Genetic:
Leukemia Profiling
Genetic profiling of leukemia cells will be performed and analyzed by an expert panel. Matched targeted therapy recommendation based on profiling results will be made if available. The recommendation, if available, will be communicated to the primary oncologist.

Locations

Country Name City State
United States Children's Healthcare of Atlanta Atlanta Georgia
United States Children's Hospital Colorado Aurora Colorado
United States Johns Hopkins Hospital Baltimore Maryland
United States Dana Farber Cancer Institute Boston Massachusetts
United States The Children's Hospital at Montefiore Bronx New York
United States Roswell Park Comprehensive Cancer Center Buffalo New York
United States The University of Chicago Chicago Illinois
United States Children's Hospital of Wisconsin Milwaukee Wisconsin
United States Children's Hospital's and Clinics of Minnesota Minneapolis Minnesota
United States Columbia University Medical Center New York New York
United States Memorial Sloan Kettering Cancer Center New York New York
United States Children's Hospital of Philadelphia Philadelphia Pennsylvania
United States Washington University at St. Louis School of Medicine Saint Louis Missouri
United States UCSF Helen Diller Family Comprehensive Cancer Center San Francisco California
United States Seattle Children's Hospital Seattle Washington

Sponsors (1)

Lead Sponsor Collaborator
Dana-Farber Cancer Institute

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Rate of Patients With Actionable Alterations 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%. 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.
Secondary Rate of Somatic Genomic Alterations This Secondary Outcome will be addressed by describing the somatic genomic alterations in Cohort 1 (Relapsed/Refractory Leukemia arm) and Cohort 2 (New Diagnosis arm) that are discovered by genomic analyses. 2 Years
Secondary Rate of Results Reporting This Secondary Outcome will be addressed by a description of the time of results reporting. Documentation of the time of sample receipt and processing, resources and personnel utilized at each step of the process, time to results reporting, interpretation and review by Expert panel and communication of results to the primary oncologist will be captured. 2 Years
Secondary Parent's Feelings and Understanding of Genomic Testing This Secondary Outcome will be addressed by describing the hopes and concerns of parents of children with recurrent/refractory/high-risk de novo leukemia regarding genomic testing of their child's leukemia as well as their understanding of the testing and evaluate whether the hopes and concerns were realized following the return of results. 2 Years
Secondary Analysis of Primary Leukemia Sensitivity Testing and Establishment of Xenograft Models This Secondary Outcome will be addressed by completing analysis of the primary leukemia sensitivity testing to a panel of drugs or shRNA and establishing xenograft models in dedicated participating research laboratories and conducting co-clinical trials with the recommended matched therapy once the animal model is established. 2 Years