Laura & Isaac Perlmutter Cancer Center

This research aims to gather health information from infants, children, adolescents, and young adults diagnosed with cancer to improve treatment and ongoing care. It maintains a Childhood Cancer Registry and uses clinical and biological data to identify eligibility for Childrens Oncology Group therapeutic trials. The study also develops a biorepository of biospecimens and clinical data for current and future research, including tumor and host DNA samples, and allows for future contact regarding additional research studies. Participants are assigned to one of two observational protocols. In the Project Every Child Protocol, medical data is reviewed to create a cancer registry, and biospecimens such as tissue, blood, bone marrow, plasma, serum, saliva, cerebrospinal fluid, or urine are collected. In the Project AYA Protocol, biospecimens are collected similarly. These samples are gathered at diagnosis, progression, recurrence, or post-mortem where applicable. During the study, participants undergo medical chart reviews and biospecimen collections. Researchers measure the number of patients consenting to biobanking as a primary outcome. Clinical data including diagnosis, staging, treatment summaries, and outcomes are recorded. The study involves ongoing data collection over time to support future research and screening for trial eligibility, with participation lasting as long as data and samples continue to be collected.

Researchers are studying breast cancer to improve personalized medicine by identifying which new drug agents work best for specific types of breast cancer tumors. The study also aims to find early signs of treatment success by analyzing tumors before surgery using MRI images along with tissue and blood samples. This Phase 2 trial focuses on matching treatments to tumor molecular characteristics to increase the chance of complete tumor response after therapy. Participants receive novel drugs combined with standard chemotherapy in different treatment groups based on their tumor biomarkers. The standard therapy includes weekly paclitaxel for 12 weeks followed by doxorubicin and cyclophosphamide before surgery. Various investigational drugs and combinations are tested in sequences with standard treatments, and treatment groups showing promising results may continue while less effective ones are discontinued. Treatment lasts up to 36 weeks before surgery. During the study, participants undergo tumor biopsies, MRI scans at multiple points, and blood and tissue sample collections to monitor changes and predict treatment outcomes. Researchers measure the pathological complete response after surgery, track relapse-free and overall survival up to five years, and monitor adverse events during treatment and follow-up. The study involves detailed assessments before, during, and after treatment to evaluate safety and effectiveness over time.

Researchers are comparing two treatments for children and young adults aged 2 to 21 with newly diagnosed or untreated low-grade glioma LGG that does not have a BRAFV600E mutation or neurofibromatosis type 1 NF1. The study aims to find out if selumetinib, a drug that blocks enzymes needed for tumor cell growth, works as well as the standard chemotherapy treatment with carboplatin and vincristine CV. It also evaluates which treatment better improves quality of life and patient outcomes. Participants are randomly assigned to one of two groups. One group receives vincristine intravenously on specific days along with carboplatin intravenously on scheduled days, followed by maintenance cycles every 42 days for up to 8 cycles. The other group takes selumetinib orally twice daily in 28-day cycles for up to 27 cycles. Throughout the trial, patients undergo blood collection, MRI scans, and in the selumetinib group, echocardiography at baseline. Treatments continue unless there is disease progression or unacceptable side effects. During participation, patients have various assessments including tumor imaging, blood tests, and evaluations of vision and motor function. Quality of life and cognitive, social, emotional, and behavioral functions are also measured. After treatment, patients are followed every 3 months for the first year, every 6 months for years 2 and 3, and then annually up to 10 years. The main outcome measured is event-free survival over this period.

Researchers are studying very low-risk and low-risk fusion negative rhabdomyosarcoma, a type of soft tissue cancer, to maintain excellent treatment outcomes while reducing therapy burdens. This phase III trial also evaluates how well patients with certain DNA mutations respond to different chemotherapy regimens and examines the use of centralized molecular risk stratification in treatment planning. The study aims to improve outcomes by adjusting therapy intensity based on molecular features. Patients are assigned to one of three treatment regimens based on their risk level and mutation status. Very low-risk patients receive vincristine and dactinomycin over 24 weeks in 8 cycles. Low-risk patients receive 12 weeks of vincristine, dactinomycin, and cyclophosphamide followed by 12 weeks of vincristine and dactinomycin, totaling 8 cycles. Patients with MYOD1 or TP53 mutations switch to an intensified 12-13 cycle regimen including vincristine, dactinomycin, and cyclophosphamide. Radiation therapy may be given at cycle 5 for some patients. Treatments are administered intravenously every 21 days. Throughout the study, participants undergo CT scans, MRI, bone scans, PET scans, and tumor biopsies to monitor disease status. Blood and tissue samples are collected at various times for research purposes. Researchers measure failure-free survival and overall survival over several years to assess treatment success. The trial lasts up to five years with ongoing assessments to track patient outcomes and safety.

Researchers are investigating targeted therapies directed by genetic testing for patients with locally advanced or advanced solid tumors that have spread or progressed after standard treatments. This Phase 2 screening trial, called ComboMATCH, aims to match patients to specific combination treatments based on genetic mutations found in their tumor cells. The study seeks to improve treatment planning by using genetic information to guide therapy choices. Patients undergo tumor genetic screening using previously collected samples or a new biopsy if eligible and aged 18 or older. Based on their tumors genetic profile, patients are assigned to one of multiple treatment subprotocols involving different drug combinations. Treatments include oral, intravenous, and intramuscular drugs such as selumetinib, olaparib, fulvestrant, binimetinib, and others. Treatment cycles generally repeat every 28 days until disease progression or unacceptable toxicity. Patients may also undergo biopsies, blood collection, imaging scans CT, MRI, PET, echocardiograms, and bone marrow procedures as part of the study. Throughout the trial, participants have regular assessments including tumor biopsies, blood draws, imaging tests, and heart function monitoring. These evaluations occur during screening, treatment, and follow-up periods. Researchers measure patient enrollment, assignment to treatment arms, and treatment outcomes over up to 8 years. Safety is monitored via clinical evaluations and diagnostic tests. This comprehensive approach aims to evaluate responses to targeted therapies guided by tumor genetics for advanced solid tumors.

The trial investigates the effectiveness and safety of multiple therapies in participants with resectable stages IB, IIA, IIB, IIIA, or selected IIIB non-small cell lung cancer NSCLC tumors that meet specific biomarker criteria. This phase II study focuses on patients who have not been previously treated and aims to assess various targeted treatments based on tumor characteristics. Participants receive up to 8 weeks of neoadjuvant therapy with drugs such as alectinib, entrectinib, vemurafenib plus cobimetinib, pralsetinib, atezolizumab combined with stereotactic body radiation therapy SBRT, or divarasib before undergoing surgical resection according to standard care. After surgery, eligible participants with no disease progression or pathological response continue with adjuvant treatment, including chemotherapy cycles followed by up to 2 years of the respective study drug or other standard treatments depending on their cohort. Throughout the study, participants undergo clinical staging with CT, PET, and brain MRI scans, and evaluations including pathological response, adverse events, and survival outcomes. Researchers monitor tumor responses, disease-free and overall survival, and safety events for up to nine years. Treatment adherence, imaging, blood tests, and molecular analyses such as circulating tumor DNA clearance are part of the assessments during neoadjuvant, surgical, and adjuvant phases.

Researchers are studying colorectal cancer CRC in participants aged 45 to 70 who have 1 to 2 non-advanced adenomas, which are small colon polyps without high-risk features. The trial compares two surveillance colonoscopy schedules one group receives colonoscopy at 5 and 10 years after polyp detection, while the other group has colonoscopy only at 10 years. This study aims to provide randomized data to guide optimal timing for follow-up colonoscopies to help reduce CRC incidence and improve clinical practices. Participants are randomly assigned to one of two groups the first group undergoes surveillance colonoscopy at both 5 and 10 years following their qualifying colonoscopy, and the second group undergoes colonoscopy at 10 years only. All colonoscopies, including any unscheduled ones, follow current quality guidelines and institutional preparation standards. The qualifying colonoscopy must have been complete and performed within 4 years before randomization, with all polyps fully removed. During the study, participants will undergo colonoscopies according to their assigned schedules. Researchers will monitor the incidence of colorectal cancer over 10 years as the primary outcome. Participants must be able to understand English or Spanish to complete required procedures. The study may last up to several decades, with ongoing monitoring to assess long-term cancer incidence and the effectiveness of different surveillance intervals.

Researchers are evaluating the combination of BP1001 plus venetoclax plus decitabine in adults with acute myeloid leukemia AML who cannot or choose not to receive intensive chemotherapy. The study aims to determine if this combination offers better results, such as complete remission, compared to venetoclax plus decitabine alone or to intensive chemotherapy in patients with untreated or relapsedrefractory AML. This Phase IIa trial focuses on patients who are fragile or ineligible for aggressive treatment, addressing a need for targeted therapies that may improve outcomes while being tolerable. Participants will receive one of three treatment combinations depending on their AML status untreated patients receive BP1001 with venetoclax and decitabine relapsed or refractory patients receive BP1001 with venetoclax and decitabine and a third group of relapsed or refractory patients who are resistant or intolerant to venetoclax receive BP1001 plus decitabine. The study uses an open-label design and will enroll about 19 participants per cohort initially, with potential expansion based on interim data review. The treatment involves multiple drug administrations to assess safety, pharmacokinetics, pharmacodynamics, and efficacy. During the study, participants will undergo regular assessments including bone marrow biopsies or aspirates to measure treatment response over 180 days. Safety will be monitored through evaluations up to 30 days post-treatment, along with pharmacokinetic studies of BP1001. Researchers will also assess minimal residual disease status and overall survival. Participants must meet specific health criteria and will provide informed consent before enrollment. The study is sponsored by Bio-Path Holdings, Inc. and is expected to continue until December 2028.

Researchers are evaluating the safety, side effects, and optimal dose of cabozantinib combined with standard chemotherapy in patients newly diagnosed with osteosarcoma. This phase IIIII trial aims to compare the effects of adding cabozantinib, a kinase inhibitor that may slow tumor growth, to the usual chemotherapy drugs methotrexate, doxorubicin, and cisplatin. The study focuses on patients under 40 years old with high-grade osteosarcoma, including both localized and metastatic cases. Participants receive treatment in two main phases. The feasibility phase now closed tested cabozantinib with chemotherapy in metastatic patients with resectable tumors, using cycles of oral cabozantinib and intravenous chemotherapy drugs over several months. The ongoing efficacy phase randomly assigns patients to either standard chemotherapy alone or chemotherapy combined with cabozantinib, with treatment divided into induction, consolidation, and maintenance cycles lasting 35 or 28 days each. Imaging scans, blood samples, and surgery are part of the treatment and evaluation process. During the study, participants undergo regular assessments including X-rays, CT scans, MRI, PET or bone scans, and blood sample collection at diagnosis and throughout treatment. Researchers monitor event-free survival, overall survival, symptom burden, and side effects reported by patients. The trial lasts up to 5 years after treatment to assess long-term outcomes, gather tumor and blood samples for further biological studies, and evaluate the impact of cabozantinib addition on patient health and response.

Researchers are evaluating the safety, side effects, and best dose of novobiocin in patients with solid tumors that have alterations in DNA repair genes. Novobiocin is an antibiotic that blocks a protein called DNA polymerase theta, which helps repair damaged DNA in cells. This study focuses on patients with tumors carrying specific gene mutations related to DNA repair, who may or may not have received prior PARP inhibitor treatment. Participants receive novobiocin orally once daily for 5 days followed by 2 days off each week, with treatment cycles repeating every 28 days if the disease does not progress or toxicity is unacceptable. Throughout the study, patients undergo tumor biopsies, medical imaging scans, and blood sample collection. After completing treatment, patients are followed up every 3 to 6 months for 2 years to monitor outcomes. During the trial, researchers monitor the maximum tolerated dose and recommend a phase 2 dose of novobiocin. They also assess anti-tumor activity, safety, tolerability, and pharmacokinetics. Additional analyses include genetic sequencing and gene expression studies on tumor biopsies. Outcome measures include radiological response, progression-free survival, duration of response, and biological effectiveness over up to 2 years of observation.