UF Health Cancer Institute - Gainesville

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 evaluating the safety, side effects, and best dose of a combination of two anti-cancer drugs, neratinib and trastuzumab deruxtecan, in patients with advanced solid tumors that have spread or cannot be removed by surgery and have changes in the HER2 gene. This phase I trial aims to determine the maximum tolerated dose, assess tumor response, and study the drugs effects on tumor markers and overall survival in this patient population. Participants receive neratinib orally once daily on specified days of each 21-day cycle and trastuzumab deruxtecan intravenously over 30 to 90 minutes on day 1 of each cycle. Treatment cycles repeat every 21 days unless disease progression or unacceptable side effects occur. The study includes dose-escalation and dose-expansion phases, with possible tissue biopsies at various points to evaluate tumor changes and drug impact. Throughout the study, participants undergo blood sample collection, CT scans, and heart function tests via echocardiography or MUGA scans. Biopsies may be performed at baseline, during the study, or at disease progression depending on the cohort. After treatment ends, patients are followed every three months for at least one year to monitor health and survival outcomes. Researchers measure treatment safety, tumor response rates, duration of response, progression-free survival, and drug concentrations over this period.

Researchers are evaluating the effects of pembrolizumab alone compared to the standard treatment of chemotherapy cisplatin or carboplatin combined with radiation therapy after surgery in patients with recurrent or second primary head and neck squamous cell carcinoma. This phase II trial aims to assess overall survival, disease control, metastasis rates, and side effects in high-risk patients, while also exploring whether high PD-L1 expression predicts better responses to pembrolizumab. Participants are randomly assigned to one of two treatment groups. One group receives intravenous cisplatin or carboplatin weekly for six cycles alongside daily intensity-modulated radiation therapy or proton beam radiation totaling 30 sessions. The other group receives intravenous pembrolizumab every six weeks for up to nine cycles. Throughout treatment, participants undergo CT or MRI scans to monitor their condition. After completing treatment, participants are followed up 30 days later and then every six months for up to five years. During the study, researchers evaluate overall survival from randomization to death, adverse events for up to five years, disease-free survival, and PD-L1 expression levels. The study monitors patients closely with imaging and clinical assessments to track treatment effects and safety 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 evaluating the safety, side effects, and best dose of a combination treatment using entinostat and ZEN003694 for patients with advanced or refractory solid tumors, including pancreatic cancer. Entinostat is a histone deacetylase inhibitor that may stop tumor growth by blocking enzymes needed for cell growth, while ZEN003694 is a bromodomain and extra-terminal BET protein inhibitor that may prevent tumor growth by targeting BET protein overproduction. This phase III trial aims to find the maximum tolerated dose and assess the overall response rate in pancreatic cancer patients. Participants receive oral doses of entinostat and ZEN003694 in repeated 28-day cycles unless disease progression or unacceptable toxicity occurs. The current dosing schedule involves entinostat given once weekly on days 1, 8, 15, and 22, and ZEN003694 given orally on specific days within each cycle days 1-5, 8-12, 15-19, and 22-26. Patients undergo computed tomography scans throughout the study to monitor tumor status. Earlier dosing schedules and biopsy procedures are no longer used as of an amendment dated June 12, 2025. During the study, participants undergo regular assessments including CT scans and blood tests to monitor safety and response. The research team measures maximum tolerated dose, recommended phase 2 dose, objective response rates, and survival outcomes. Patients are followed for 30 days after completing treatment to observe safety and any lasting effects, ensuring close monitoring throughout their participation, which lasts as long as treatment continues or until progression or toxicity occurs.

Researchers are evaluating whether the drugs mosunetuzumab and polatuzumab vedotin can provide benefits for patients with diffuse large B-cell lymphoma or grade 3b follicular lymphoma that has returned or not responded to treatment. This study focuses on patients who have already received chemotherapy with fludarabine and cyclophosphamide, followed by CAR T-cell therapy. The goal is to determine if adding these drugs after previous treatments may better control or shrink the cancer. Participants first receive lymphodepleting chemotherapy with fludarabine and cyclophosphamide, followed by one of three CAR T-cell therapies given by infusion tisagenlecleucel, axicabtagene ciloleucel, or lisocabtagene maraleucel. After this, participants are randomly assigned to one of four groups receiving mosunetuzumab alone, polatuzumab vedotin alone, both drugs together, or observation without further drug treatment. Those in the observation group who experience disease progression within 12 months may switch to receive both drugs. Throughout the study, patients undergo PET-CT or CT scans and provide blood and tissue samples for research. Participants will be monitored regularly with imaging scans and laboratory tests to assess disease status and treatment effects. Researchers will measure progression-free survival, overall survival, remission rates, and treatment side effects up to two years. The study also includes collecting and banking tissue samples and PET-CT images for future research. The total length of involvement depends on individual timelines but includes treatment and follow-up periods extending up to two years or more.

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.

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 dose-escalated radiation therapy compared to usual care for patients with locally advanced unresectable pancreatic ductal adenocarcinoma who have already completed 4 to 6 months of chemotherapy. The study is a phase III trial aiming to determine if higher doses of radiation can improve 3-year overall survival, as well as assess effects on tumor progression, chemotherapy-free intervals, treatment toxicity, and quality of life. This trial is led by NRG Oncology and focuses on patients without disease progression after initial chemotherapy. Participants are randomly assigned to one of two groups. The first group receives standard care, which may include additional chemotherapy options such as FOLFIRINOX, NALIRIFOX, or gemcitabinenab-paclitaxel, standard radiation therapy with concurrent chemotherapy, or observation. The second group receives dose-escalated radiation therapy delivered in either 5 or 25 fractions, with or without concurrent chemotherapy for the 25-fraction option. After radiation, patients who have not completed 6 months of chemotherapy are encouraged to finish it, with treatment beyond 6 months at the physicians discretion. Imaging and tissue biopsies are performed throughout the study. Participants undergo various assessments including blood sample collection, CT scans, MRI, and optional PETCT scans before and during treatment. Tumor tissue biopsies are also collected. Follow-up visits occur every 3 months for the first 2 years and annually for an additional 3 years. Researchers measure overall survival, local disease progression, progression-free survival, chemotherapy-free intervals, and adverse events. Quality of life is evaluated using specific questionnaires over the course of participation.