Clinical Research Directory

Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...

Background: \- Some people with cancer have solid tumors. Others have refractory leukemia. This may not go away after treatment. Researchers want to see if a drug called TURALIO(R) can shrink tumors or stop them from growing. Objectives: \- To find the highest safe dose and side effects of TURALIO(R). To see if it helps treat certain types of cancer. Eligibility: \- People ages 3-35 with a solid tumor or leukemia that has returned or not responded to cancer therapies. Design: * Individuals will be screened with: * Medical history * Physical exam * Blood and urine tests * Heart tests * Scans or other tests of the tumor * Individuals will take TURALIO(R) as a capsule once daily for a 28-day cycle. They can do this for up to 2 years. * During the study, participants will have many tests and procedures. They include repeats of the screening tests. Individuals will keep a diary of symptoms. * Individuals with solid tumors will have scans or x-rays. * Individuals with leukemia will have blood tests. They may have a bone marrow sample taken. * Some individuals may have a biopsy. * When finished taking TURALIO(R), individuals will have follow-up visits. They will repeat the screening tests and note side effects.

Activation Sarcoma: PROMIS and Mobility Evaluation in Sarcoma Patients

Sarcoma is a rare form of bone and soft tissue cancer, making up about 1% of all adult cancers. Management typically involves radiation therapy, chemotherapy, and surgical resection. Surgery is central to the treatment of bone and soft tissue sarcoma, yet post-surgical recovery is difficult to measure because of disease rarity, multiple different tumor types, and treatment variation. Therefore, the overall goal of the study is to use patient reported outcome measures and mobility data to define recovery trajectories in sarcoma patients undergoing surgery, and to evaluate a novel smartphone application (app) entailed Activation Health for collecting these data. Participants will download the Activation Health app on their smartphone. A demographics survey and multiple patient reported outcome measures will be administered remotely through the Activation Health app. Surveys will be completed preoperatively and at 6 weeks, 12 weeks, 6 months 12 months, 16 months, 20 months, and 24 months postoperatively. Additionally, the app will passively track mobility metrics such as daily step count, stairs climbed, and gait parameters throughout the study period. At the end of study participation, an additional surveys will be administered assess participant phone use habits and app function.

Combination Immunotherapy Targeting Sarcomas

The aim of this clinical trial is to assess the feasibility, safety and efficacy of a combination low dose chemotherapy and immunotherapy in patients who have sarcoma that is relapsed or late staged. Another goal of the study is to assess the safety and efficacy of the therapy that combines multiple CAR T cells followed by sarcoma vaccines.

Safety and Efficacy Evaluation of 4th Generation Safety-engineered CAR T Cells Targeting Sarcomas

The aim of this clinical trial is to assess the feasibility, safety and efficacy of CAR T cells immunotherapy in patients who have sarcoma that is relapsed or late staged. Another goal of the study is to assess the safety and efficacy of the therapy that combines CAR T cells and IgT cells to treat sarcoma.

18F-Fibroblast Activation Protein Inhibitor ([18F]FAPI-74) PET Imaging for Cancer Detection

Background: Fibroblast-activation protein (FAP) is an enzyme that appears in high numbers in cancer-associated fibroblasts of certain cancer types. \[18F\]FAPI-74 is a new PET (positron emission tomography) tracer, a substance that is injected into a person s body before an imaging scan. Researchers believe that \[18F\]FAPI-74 PET imaging may be able to visualize cancer more effectively than the approved tracers. If so, the new tracer would make it easier to find FAP-positive tumors in the body. Objective: To see if \[18F\]FAPI-74 PET scan is as good or better than other imaging methods for detecting certain cancers. Eligibility: People aged 18 years or older with one of these cancer types: pancreatic ductal adenocarcinoma (PDAC), cholangiocarcinoma, hepatocellular carcinoma (HCC), gastric cancer, bladder cancer, ovarian cancer, pheochromocytoma/paraganglioma (PPGL), small cell lung cancer (SCLC) or extrapulmonary neuroendocrine cancer (EP-NEC), mesothelioma or sarcoma. Participants must be scheduled or intended to receive treatment for cancer. Design: Participants will have 2 baseline scans: an \[18F\]FAPI-74, and the approved tracer \[18F\]-FDG. The \[18F\]FAPI-74 will be infused through a needle inserted into a vein. About 1 hour later, the participant will undergo imaging. Within 1 week, participants will undergo the same scanning procedures with the approved tracer. If the baseline scan with \[18F\]FAPI-74 shows the tumor(s), scans with this tracer will be repeated when their regular treatment regimen calls for scans again. If the scan with the regular FDG also show tumors, this scan will be repeated within the same week as the repeated \[18F\]FAPI-74 scan. If \[18F\]-FAPi PET scan shows no tumor(s), scans will not be repeated. If the participant's cancer progresses within 2 years, scans may be repeated. Follow-up calls will continue for 2 years.

Safety, Tolerability and Pharmacokinetics of Oral CPL304110, in Adult Subjects With Advanced Solid Malignancies

The purpose of the study is to determine to evaluate safety and tolerability of CPL304110 when administered once daily to adults with advanced solid malignancies.

my.naviGATE: A Guide to After-Treatment Effects for Adolescents and Young Adults

This study aims to design and test a novel, personalized digital intervention-my.naviGATE-for adolescent and young adults (AYA) with cancer. my.naviGATE is a mobile app that provides personalized survivorship education, access to virtual peer navigation, and responsive participant-reported outcomes (PROs).

Ovarian-Sparing Adaptive Radiotherapy in Young Adult Women

Female patients with early onset (\<50 years old) pelvic malignancies such as uterine and rectal cancers are rising in incidence, which often requires pelvic radiation; many of these patients are premenopausal and at a high risk of premature ovarian failure from radiotherapy. Premature ovarian failure carries significant cardiac, musculoskeletal, sexual, and psychosocial morbidity. Ovarian transposition carries variable success rates, is not readily accessible to the general population, and can still be at risk of clinically significant radiotherapy doses. There is an unmet need for innovative techniques to protect ovarian function.

NK White Blood Cells and Interleukin in Children and Young Adults With Advanced Solid Tumors

BACKGROUND: * Despite progress, some children and young adults with solid tumors still experience poor survival. * Activated NK cells potently kill autologous pediatric solid tumors, and clinical grade procedures are available to generate large numbers of activated NK cells for adoptive cell therapy. OBJECTIVES: * Primary objectives are: 1) to assess the feasibility of harvesting and expanding activated NK cells to meet escalating dose goals in Cohort A, 2) to assess the toxicity of infusing escalating doses of activated NK cells following lymphodepleting chemotherapy without rhIL15 (cohort A), and 3) to assess the toxicity of infusing NK activated cells with escalating doses of rhIL15 (cohort B) in pediatric patients with refractory malignant solid tumors. * Secondary objectives are: 1) to identify biologically active doses of activated autologous NK cells plus or minus rhIL15 by monitoring changes in NK cell number, phenotype and function, 2) to assess pharmacokinetics and immunogenicity of rhIL15 in a pediatric population, and 3) assess antitumor effects and changes in FDG-PET following administration of activated NK cells to lymphopenic hosts plus or minus rhIL15. 4) to evaluate saftey and efficacy of subsequent cycles of autologous NK cell infusions in patients in cohort A who received benefit from the first NK cell infusion. ELIGIBILITY: * Patients in Cohort A: 2-29 years with with refractory pediatric malignant solid tumors, Patients in Cohort B: 2-25 years with refractory pediatric malignant solid tumors. * Adequate performance status and organ function, recovered from toxic effects of prior therapy, no requirement for systemic corticosteroids and no history of allogeneic stem cell transplantation. DESIGN: * All patients receive pre-NK lymphodepleting chemotherapy with cyclophosphamide. * Cohort A receives escalating doses of activated autologous NK cells to identify feasibility of generating cells and tolerability, and potentially identify an MTD. * A1: 1x10(6) NK cells/kg * A2: 1 x 10(7) NK cells/kg * A3: 1 x 10(8) NK cells/kg * If feasibility and acceptable toxicity is demonstrated for all doses in Cohort A, patients enrolled on cohort B will receive activated autologous NK cells plus escalating doses of rhIL15 using the following schema: * B1: 1 x 10(7) NK cells/kg + rhIL15 0.25 mcg/kg/d IV x 10 * B2: 1 x 10(7) NK cells/kg + rhIL15 0.5 mcg/kg/d IV x 10 * B3: 1 x 10(7) NK cells/kg + rhIL15 1 mcg/kg/d IV x 10 * B4: 1 x 10(7) NK cells/kg + rhIL15 2 mcg/kg/d IV x 10 * Three patients will be enrolled at each dose level, with the dose level expanded to 6 if dose-limiting toxicity occurs. An expanded group of 12 patients will be treated at the highest tolerable dose level. DLT toxicity monitoring will continue for 21 days after the NK infusion, or 14 days after the last rhIL15 dose in Cohort B (whichever is later).

The Clinical Application Value of SPECT Imaging Targeting LRRC15 in Malignant Tumors

Here's a polished version of the text: "This project involves patients with high-expression LRRC15 malignant tumors who are either clinically suspected of or diagnosed with pancreatic cancer, breast cancer, lung cancer, ovarian cancer, head and neck tumors, colorectal cancer, melanoma, etc., as well as healthy volunteers. Using a targeted LRRC15-specific SPECT imaging agent, SPECT/CT imaging is performed to achieve the following objectives: For malignant tumor patients: To support disease diagnosis and staging. Diagnostic performance will be evaluated against the gold standard of pathological diagnosis, clarifying the presence or absence of lesions and determining their location and nature. To enable accurate staging and assess tumor burden by comparison with \[18F\]FDG PET/CT, thereby aiding in the determination of treatment plans. For healthy volunteers: To conduct pharmacokinetic analysis, elucidating the distribution, metabolism, and safety profile of the agent in the body."

Lead-212 PSV359 Therapy for Patients With Solid Tumors

Phase I/IIa image-guided, alpha-particle therapy study of \[203Pb\]Pb-PSV359 and \[212Pb\]Pb-PSV359 in patients with solid tumors that are known to be Fibroblast Activation Protein (FAP)-positive.

A Study of MGC026 in Participants With Advanced Solid Tumors

The study is designed to understand the safety, tolerability, pharmacokinetics, immunogenicity, and preliminary antitumor activity of vobramitamab tavatecan (MGC026) in participants with relapsed or refractory, unresectable, locally advanced or metastatic solid tumors The study has a dose escalation portion and a cohort expansion portion in Module A of the study. Combination therapy with pembrolizumab will occur in Module B of the study Participants will receive vobramitamab tavatecan by intravenous (IV) infusion. The dose of vobramitamab tavatecan will be assigned at the time of enrollment. Pembrolizumab will be administered at 200 mg every 3 weeks. Participants may receive up to 35 treatments if there are no severe side effects and as long as the cancer does not get worse. Participants will be monitored for side effects, and progression of cancer, have blood samples collected for routing laboratory work, and blood and tumor samples collected for research purposes.

Exercise Rehabilitation to Improve Long-term Outcomes in Sarcoma Survivors.

People who have had a sarcoma removed from the leg often live with weak muscles, stiff joints, and trouble walking. These problems can last for years. Formal medical follow-up usually ends about five years after treatment. Few studies have tested exercise at this stage. This study tested an 8-week supervised exercise program. Participants were adults aged 18 to 67 who were at least 5 years past limb-sparing (wide resection) surgery for a leg sarcoma. They attended a clinic in Perth, Western Australia, twice a week for one hour. The program built leg strength, joint movement, balance, walking, and fitness. It progressed through three phases and was matched to each person's surgery and ability. All participants received the same program type. There was no comparison group. Each person was tested twice before starting to confirm stable function. Testing was repeated at the end of the 8 weeks. It was repeated again 8 weeks later, after a period with no supervised exercise. The main outcome was high-level mobility, measured by the High-level Mobility Assessment Tool (HiMAT). This tool scores walking, running, hopping, and stair climbing. Other outcomes were six-minute walk distance, leg strength, joint range of motion, and self-reported physical function and quality of life (TESS and SF-36 questionnaires). The study aimed to learn two things. First, whether exercise improves strength and mobility years after sarcoma surgery. Second, whether those gains last after supervision stops.

STC-15 as a Part of Combination Therapy With Toripalimab in Selected Advanced Cancers and as Monotherapy in Participants With Selected Sarcomas

This early phase oncology trial will be conducted at various study centers to investigate the safety, tolerability, and antitumor activity of STC-15 (a METTL3 inhibitor) in combination with toripalimab (anti- programmed cell death 1 \[PD-1\]) in advanced unresectable or metastatic tumors. The Phase 2 Monotherapy part is an open-label, non-randomized, multicenter Simon's 2-stage design that investigates the safety, tolerability, and antitumor activity of STC-15 in participants with selected, relapsed sarcomas subtypes, dedifferentiated (DD) liposarcoma and leiomyosarcoma (uterine and non-uterine).

A Study of Etrumadenant and Zimberelimab in People With Dedifferentiated Liposarcoma

Participants will have a diagnosis of dedifferentiated liposarcoma (DDLS) that has spread beyond its original location (advanced). In addition, their DDLS either has come back after treatment (recurrent), has spread to different parts of your body (metastatic), or is unable to be removed surgically (unresectable). The purpose of this study is to find out whether the combination of etrumadenant and zimberelimab is an effective treatment for people with advanced DDLS.

Hyperbaric Oxygen Therapy for Soft Tissue Sarcoma Pilot Study

A parallel-group randomized pilot trial at a single institution (Duke University) on the effect of postoperative hyperbaric oxygen therapy on wound healing in patients with soft tissue sarcoma undergoing surgical resection with neo-adjuvant radiation therapy.. Participants will be allocated to either the treatment group (HBOT) or control (standard of care) by computer-generated randomization, stratified by tumor size (≤10cm and \>10cm). An unequal randomization of 2:1 will be utilized to provide experience prescribing HBOT to more patients.

LCI-SAR-BSTS-CTDNA-001: Circulating Tumor DNA Liquid Biopsy and Spectroscopy-based Molecular Urinalysis in Sarcoma Patients

The purpose of this research study is to see how well Low Pass Whole Genome Sequencing (LP-WGS) can detect circulating tumor deoxyribonucleic acid (ctDNA) in the blood of participants who have bone or soft tissue sarcoma (type of cancer). This study is also looking at a new technology called Rametrix® Molecular urinalysis (RMU). The study is using this technology to evaluate changes in the urine that may indicate the presence of disease or cancer.

Determining the Biodistribution of an Imaging Tracer (68Ga-FAPi-46) in Patients With Solid Tumors or Hematologic Cancers

This phase I trial is evaluating a new imaging tracer (68Ga-FAPi-46) with positron emission tomography (PET)/computed tomography (CT) to determine where and to which degree the tracer (68Ga-FAPi-46) accumulates in normal and cancer tissues (the biodistribution) in patients with solid tumors or hematologic (blood) cancers. PET is an established imaging technique that utilizes small amounts of radioactivity attached to very minimal amounts of tracer, in the case of this research, 68Ga-FAPi-46. Because some cancers take up 68Ga-FAPi-46, it can be seen with PET. CT utilizes x-rays that traverse the body from the outside. CT images provide an exact outline of organs and potential inflammatory tissue where it occurs in a patient's body. Combining a PET scan with a CT scan can help make the image easier to interpret. PET/CT scans are hybrid scanners that combine both modalities into a single scan during the same examination.

Surgical Margin Assessment Using Intra-operative High-resolution PET/CT in Patients With Cancers Referred to Surgical Resection, an Exploratory Pilot Study

Reoperation is often required in surgical oncology because of positive resection margins and is associated with substantial physical, psychological, and economic consequences. Reoperation may also alter or delay the initiation of adjuvant therapy, potentially increasing the risk of local or distant recurrence. When re-resection is not feasible, a positive resection margin may necessitate additional treatment (e.g., radiation therapy or chemotherapy) that might otherwise have been avoided if clear margins had been achieved during the initial operation. Moreover, obtaining a negative margin during reoperation does not necessarily result in improved oncologic outcomes. Therefore, achieving negative margins intraoperatively is a key objective in oncologic surgery. The AURA 10 Specimen PET/CT imaging system, a novel technology developed by a Belgian company, has recently been introduced to the market. This system enables perioperative imaging of resected tumors within approximately 10 minutes, allowing prompt assessment of surgical margins. Preliminary studies have demonstrated the potential clinical utility of this technique in various cancers, including breast, head and neck, and prostate cancers (1-5). A recent multicenter study of patients with breast cancer reported a sensitivity of 91% and a specificity of 86% for assessing surgical margin status in invasive ductal carcinoma (IDC) (4). The aim of this study is to evaluate the clinical utility of perioperative specimen PET/CT imaging for surgical margin assessment and the feasibility of implementing this technology in routine clinical practice. The study will initially focus on breast cancer, head and neck cancer, soft tissue sarcomas, lung cancer, colorectal liver metastases, and hepatobiliary and pancreatic malignancies. Patients will be screened for study eligibility. Those who meet the eligibility criteria will be invited to participate and provide written informed consent. On the day of the scheduled operation, patients will receive an injection of the FDG radiotracer in the Department of Nuclear Medicine approximately 1 hour before surgery. They will then be transfered by authorized personnel to the operating room. Immediately after tumor resection, the surgical specimen will be imaged using the specimen PET/CT imager (AURA 10, Xeos); this process will take approximately 10 minutes. If the surgeon decides to re-excise additional tissue, that tissue will also be imaged. In addition, all resected lymph nodes will be imaged for research purposes only. The specimen will then be transferred to the histopathology laboratory for standard-of-care evaluation. During the operation, the results of specimen PET/CT imaging will not influence the surgeon's intraoperative decision-making. Subsequent clinical decisions will be based on the results of standard-of-care evaluations and the treating physician's clinical judgment. The final surgical margin status, as determined by the pathologist, will be collected for study purposes. Patients will receive treatment and follow-up in accordance with the standard of care, and relevant follow-up data will be collected.

Video Education With Result Dependent dIsclosure

The overall study objective of this trial study is to identify and evaluate strategies to improve the accessibility of the video education with result dependent disclosure (VERDI) model, increasingly utilized as a pre-genetic testing (pretest) education alternative in clinical practice, to better serve a more diverse patient population at risk for hereditary cancers.

Fluorescence-Guided Surgical Resection for Reducing Unexpected Positive Margins in Patients With Sarcoma, SarcoSIGHT Trial

This phase II trial compares standard of care surgical resection to fluorescence-guided surgical resection using indocyanine green and fluorescence imaging for reducing unexpected positive margin rates in patients with sarcoma. Surgical margins are one of the criteria used in assessing the effectiveness of a given surgical procedure. They are the edges or border of the tissue removed in cancer surgery. A negative margin means that the resected tumor is surrounded by normal tissue, which implies there is no disease left in the patient. By contrast, a positive margin means that sarcoma cells are visible at the edge of the tumor. Positive margins may be classified as planned when they are expected pre-operatively due to the preservation of a critical structure, or unexpected when the pathologist reports the presence of tumor at the margin despite the surgeon having intended to remove it in its entirety. Fluorescence-guided surgery is an established surgical method that involves the administration of fluorescent dye (in this case, indocyanine green) into the tumor, which can then be visualized with a special camera during surgery to help surgeons identify tumor versus healthy tissue. Fluorescence-guided surgery may reduce the rate of unexpected positive margins in patients undergoing surgery for sarcoma.

Comprehensive Omics Analysis of Pediatric and Adult Solid Tumors and Establishment of a Repository for Related Biological Studies

Background: \- Laboratory investigators who are studying common childhood cancers are interested in developing a tissue repository to collect and store blood, serum, tissue, urine, or tumors of children who have cancer or adults who have common childhood cancers. To develop this repository, additional samples will be collected from children and adults who have been diagnosed with common childhood cancers such as leukemia and tumors of the central nervous system. Objectives: \- To collect and store blood, serum, tissue, urine, or tumor samples of children who have cancer or adults who have common childhood cancers. Eligibility: * Individuals who have been diagnosed with a common childhood cancer (e.g., leukemia) regardless of patient age. * Children, adolescents, and adults who have been diagnosed with a type of cancer more commonly found in adults. Design: * Extra blood, serum (the liquid part of blood), tissue, urine, or tumor samples will be collected from participants at a time when sampling is required for medical care or as part of a research study. * No additional procedures will be performed for the sole purpose of obtaining additional tumor tissue, aside from what is required for clinical care.

Aerosolized Aldesleukin in Treating Patients With Lung Metastases

This phase I/II trial studies the side effects and best dose of aerosolized aldesleukin and to see how well it works in treating patients with cancer that has spread from the original tumor to the lungs. Biological therapies, such as aerosolized aldesleukin, may stimulate or suppress the immune system in different ways and stop tumor cells from growing.

Evaluating the Use of Educational Videos in Newly Diagnosed Patients in a Medical Oncology Sarcoma Clinic

This study aims to evaluate how sarcoma-specific educational videos improve patients' knowledge of their disease and treatment options before their first clinic appointment. Participants will watch four educational videos before their first appointment with a medical oncologist. Participants will be asked to complete surveys to assess their understanding of sarcoma and its treatment options before and after their first medical oncology clinic appointment.