80 Clinical Trials for Various Conditions
This study utilizes a multi-institutional registry to describe the natural history of medullary thyroid cancer that has spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced) in understanding disease management. The goal of this study is to learn about how medullary thyroid cancer develops and progresses.
The reason for this study is to see if the study drug selpercatinib is safe and more effective compared to a standard treatment in participants with rearranged during transfection (RET)-mutant medullary thyroid cancer (MTC) that cannot be removed by surgery or has spread to other parts of the body. Participants who are assigned to the standard treatment and discontinue due to progressive disease have the option to potentially crossover to selpercatinib.
This trial develops and studies how well a patient decision aid works in supporting decision-making about when to start or stop new drugs, join clinical trials, or continue active cancer monitoring for patients with medullary thyroid cancer and their caregivers. Developing a patient decision aid may help patients with medullary thyroid cancer make well-informed decisions about their cancer care and be able to discuss their preferences with their doctors.
This is an open-label, first-in-human study designed to evaluate the safety, tolerability, pharmacokinetics (PK) and preliminary anti-tumor activity of selpercatinib (also known as LOXO-292) administered orally to participants with advanced solid tumors, including rearranged during transfection (RET)-fusion-positive solid tumors, medullary thyroid cancer (MTC) and other tumors with RET activation.
This study is conducted in the United States of America (USA). The aim of the study is to monitor the number of annual new adult cases of medullary thyroid carcinoma (MTC) and to establish a registry for these new cases in order to identify any possible increase related to the introduction of liraglutide, exenatide once-weekly, and other GLP-1 receptor agonists into the US market.
The purpose of this study is to give patients with medullary thyroid cancer either 300mg/day or 150mg/day vandetanib and compare how well each dose affects how their cancer responds. It will also help the investigators understand the side effects of different doses in these patients.
Background: Medullary thyroid carcinoma (MTC) is a rare malignancy, occurring either as a sporadic disease (75% of cases), or in a hereditary pattern as multiple endocrine neoplasia (MEN) type 2 (MEN2A or MEN2B) or familial medullary thyroid carcinoma (FMTC). The MTC arises from the neural crest C-cells and in hereditary cases the first pathological disorder is C-cell hyperplasia (CCH) Most patients with MTC have advanced disease at the time of diagnosis. Chemotherapy and external beam radiotherapy have been minimally effective. Molecular targeted therapeutics (MTTs) and other receptor kinases in patients with advanced MTC have demonstrated activity. Despite some clinical responses, the collection of tumor tissues and autologous normal tissues has been virtually non-existent. Thus, laboratory studies defining affected molecular targets and downstream pathways, and molecular data providing direction for future clinical trials has yet to occur. Data from molecular studies of tumor tissue of hereditary or sporadic MTC patients will assist in predicting clinical behavior and the biology of MTC in predicting response to a given MTT, and in designing combination clinical trials. Objectives: Clarify how normal molecular pathways are altered by mutations in the RET protooncogene. Including additional genetic mutations and unidentified chromosomal translocations. Correlate results from molecular analyses of MTC tissue with patient s clinical course. Define how the molecular and clinical data will be useful in designing targeted therapy for patients with MTC. Eligibility: Patients must have confirmed diagnosis of C-cell hyperplasia, primary MTC, or metastatic MTC with archived pathology specimens available at Washington University. Design: Paraffin blocks of MTC tissues from archival samples at Washington University Department of Pathology will be selected. H\&E slide from selected tissue blocks will be examined for molecular study suitability. Necessary tissue samples from blocks will have molecular studies, including, gene arrays, array comparative genomic hybridization, immunohistochemistry, and sequencing. Retrospective chart review will occur to obtain relevant clinical information.
Background: * The combination of anti-cancer drugs vandetanib (given orally) and bortezomib (given intravenously) has not been used in humans. However, both drugs have been studied separately. Bortezomib has been approved by the U.S. Food and Drug Administration (FDA) for treating multiple myeloma and mantle cell lymphoma, while vandetanib is still under investigation pending FDA approval. * Both bortezomib and vandetanib are under investigation for use in treating certain kinds of cancer. Researchers hope that the combination of these two drugs will be more effective than either of them alone. Objectives: * To determine if the combination of vandetanib and bortezomib will decrease the amount of the cancer and, if it does, to determine how long the response will last. * To determine any side effects that may occur with this combination of treatments. * To determine what doses of each drug are well tolerated and safe when given together. * To study genetic mutations in tumors to better understand how tumors grow and how these drugs interact with the tumor. Eligibility: * Patients 18 years of age and older with solid tumors that cannot be surgically removed and have either recurred or shown further growth. The tumor(s) must be able to be evaluated by X-ray, MRI (magnetic resonance imaging), and CT (computerized tomography) scanning. * Patients who have been diagnosed with medullary thyroid cancer will participate in Phase II of the study. Design: * Tumor samples may be taken at the start of the study for research purposes. * Phase I: Patient groups will be treated on an outpatient basis with vandetanib and bortezomib, given at increasing doses over four different levels to determine the maximum tolerated dose calculated by height and weight: * Doses will be given on Days 1, 4, 8, and 11 for each 28-day cycle. * Two additional levels (Level 1A and Level 1B) may be included in the study, depending on side effects at various levels. * Phase II: Patients with medullary thyroid cancer will be divided into two groups, with two patients in Group A for every one patient in Group B. No placebo will be involved in this study. * Group A: Patients will be treated with vandetanib and bortezomib at the maximally tolerated dose of the Phase I study. * Group B: Patients will be treated with bortezomib alone. * A second tumor sample may be taken. In patients with thyroid cancer, the second biopsy will be done at the 6-week evaluation (approximately 42 days after beginning). In patients with cancer other than thyroid cancer, the second biopsy will be obtained on Day 4 of either the first or second cycle, after the bortezomib infusion. * The effects of the drugs will be studied through blood samples and CT scans taken during and after various drug cycles.
Background: * Medullary thyroid carcinoma (MTC) is common in people with a genetic disorder called multiple endocrine neoplasia (MEN). * Vandetanib is an experimental drug that blocks a defective protein receptor (rearranged during transfection (RET) receptor) found on the surface of cancer cells in people with MEN. It is thought that this protein is a primary cause of MTC in people with MEN. Objectives: * To study the activity of Vandetanib in children and adolescents with MEN-related MTC by measuring the change in tumor size, in blood levels of proteins produced the tumor (calcitonin and carcinoembryonic antigen (CEA) and in tumor-related diarrhea. * To determine the safety and tolerability of Vandetanib in children and adolescents. * To study how the body handles Vandetanib in children and adolescents. * To determine the effect of Vandetanib on the survival of children and adolescents with MTC. Eligibility: -Children and adolescents 5 to 18 years of age with MTC whose tumor cannot be surgically removed or has grown back after treatment or has metastasized (spread beyond the thyroid gland). Design: * Patients take Vandetanib once a day in 28-day cycles. The first patients enrolled in the study are started on a low dose of Vandetanib to determine tolerability. * Patients have periodic blood tests, electrocardiograms, and blood pressure measurements to look for side effects of Vandetanib. * Blood tests and imaging scans (magnetic resonance imaging (MRI), computed tomography (CT), bone and octreoscan) are done every 8 weeks for the first 32 weeks of treatment and then every 16 weeks for the duration of the treatment period. * Patients who have tumor-related diarrhea keep a daily record of the number and consistency of bowel movements.
This phase II trial studies how well sorafenib tosylate works in treating patients with medullary thyroid cancer that has spread to other parts of the body (metastatic), spread to the tissue surrounding the thyroid (locally advanced), or has returned after a period of improvement (recurrent). Sorafenib tosylate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth and by blocking blood flow to the tumor.
Objectives: Primary objectives: To determine the maximum tolerated doses (MTD) for the combination of imatinib mesylate, capecitabine, and dacarbazine in patients with solid tumors. To determine the overall tumor response rate to imatinib mesylate in combination with capecitabine and dacarbazine as first line and second line therapy in advanced metastatic medullary thyroid carcinoma. To determine the tolerability (toxicity) of this regimen. Secondary objectives: To determine the median overall survival (OS) and time to progression (TTP) for patients treated with this combination.
This study is a retrospective study trying to find the predictive factors for medullary thyroid aggressiveness in terms of tumor metastasis and patients' survival.
Clinical studies, with a distinct emphasis on medullary thyroid cancer, play a pivotal role in evaluating the safety and effectiveness of novel treatments for this condition. These trials serve as essential tools to determine whether new medications surpass conventional therapies, providing substantial evidence to endorse their broader adoption. The primary objective is to meticulously examine trial completion rates and voluntary withdrawals within this specific patient group. By actively participating in this observational study plays a critical role in pushing medical knowledge forward and advancing care for individuals suffering from the medullary thyroid cancer.
Background: Medullary thyroid cancer (MTC) is a tumor of the thyroid gland. Surgery is the only current treatment to cure it. The drug pembrolizumab (MK-3475) is a new type of cancer therapy. It works by allowing the immune system to detect and kill tumor cells. Objective: To test how pembrolizumab affects people with MTC and if it can offer them clinical benefit. Eligibility: People ages 18 and older with MTC Patients who have recurrent or metastatic MTC, for whom surgery is not a curative option Patients with some imaging evidence of MTC Patients with minimal symptoms related to MTC Design: Participants will be screened with: * Medical history * Physical exam * Blood, urine, and heart tests * Computed tomography (CT) scan or magnetic resonance imaging (MRI): They lie in a machine that takes pictures of the body. * Bone scan Participants will be put in a group based on their treatment history: * Group 1 if they have had an immune stimulating cancer vaccine * Group 2 if they have had no vaccine Participants will receive the study drug as a 30-minute intravenous (IV) infusion every 3 weeks. Treatment will continue for up to 2 years as long as they tolerate it and their disease does not get worse. Participants will have physical exams and blood tests on the day of each infusion. They will have CT and bone scans every 3 months. Participants may save biopsies before treatment and after starting treatment. Participants will have a final visit 3-4 weeks after stopping treatment. This will include a physical exam and blood and heart tests. After this study, participants can join a long-term follow-up study.
This research study is studying a targeted therapy as a possible treatment for thyroid cancer. A targeted therapy is a type of treatment that uses drugs or other substances to identify and attack specific types of cancer cells with less harm to normal cells. - The name of the study intervention involved in this study is regorafenib.
The purpose of this Phase 2 study is to find out what effect, good and/or bad, external beam radiation therapy, has on the patient and their thyroid cancer where surgery is not an option or where despite surgery, the disease is still present.
Background: - GI-6207 is an experimental cancer vaccine made with baker's yeast. The yeast has been modified to help the immune system target a protein called CEA. CEA is found on the surface of some kinds of tumor cells, including thyroid cancer cells. Researchers want to see if GI-6207 can encourage the body's immune system to attack and kill tumor cells that contain the CEA protein. They will test to see whether this vaccine is a safe and effective treatment for medullary thyroid cancer that has not responded to earlier treatments. Objectives: - To test the safety and effectiveness of the GI-6207 vaccine for advanced medullary thyroid cancer. Eligibility: - Individuals at least 18 years of age who have medullary thyroid cancer that has not responded to earlier treatments. Design: * Participants will be screened with a physical exam and medical history. They will provide blood and tumor samples and have an imaging study of the neck and chest. They will also have a skin test to make sure that they are not allergic to the yeast in the vaccine. * Participants will be divided into two groups. One group will start to take GI-6207 immediately for 1 year. The second group will have 6 months of monitoring and tests with no vaccine, and then will take GI-6207 for 1 year. * GI-6207 will be given every other week for the first seven visits (about 3 months), and then monthly for the remaining year of treatment. It will be given as injections beneath the arm and in the upper thigh. These locations will help the vaccine enter the lymph nodes and reach the immune system more quickly. * Participants will be monitored with frequent blood and urine tests and imaging studies. * Participants will have regular follow-up visits after their year of study vaccines.
Background: * Medullary thyroid cancer (MTC) represents 5% of thyroid cancers and presents as a hereditary (25% of cases) or sporadic (75% of cases) neuroendocrine malignancy. * MTC arises from the parafollicular C-cells of the thyroid. * Germline mutations in the rearranged during transfection (RET) proto-oncogene occur in virtually all of hereditary MTC cases, and somatic RET mutations occur in 50% of sporadic cases. * Drugs targeting RET kinase such as vandetanib and cabozantinib have shown efficacy in the treatment of advanced or metastatic MTC, however, more effective RET inhibitors are needed for previously untreated patients as well as patients who have become refractory to other molecular targeted therapeutics (MTTs). * Ponatinib, a drug that is Food and Drug Administration (FDA) approved as a therapy for chronic myelogenous leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL), is a potent inhibitor of RET kinase. Primary Objective: -To determine the objective overall response rate (complete response \[CR\] + partial response \[PR\] by Response Evaluation Criteria in Solid Tumors (RECIST) to ponatinib in the treatment of patients with advanced or metastatic MTC previously treated with cabozantinib and vandetanib who: 1) have tumors with RET mutations and 2) have tumors without RET mutations. Eligibility: * Patients must have histologically confirmed, unresectable, locally advanced or metastatic MTC, with measurable disease by RECIST criteria. * Patients must have disease amenable to biopsy and be willing to undergo biopsy for molecular analysis, and also have adequate archival material from their thyroidectomy or from a tumor biopsy obtained prior to beginning any systemic therapy. * Patients must have failed or been intolerant to prior treatment with both cabozantinib and vandetanib. * The last dose of prior systemic therapy must be more than 28 days prior to the first dose of ponatinib * Radiation therapy is permitted if the last treatment was received more than 28 days prior to the first dose of ponatinib. Design: * Open label phase II trial with 2 treatment groups: * RET mutation positive MTC, previously treated with vandetanib and cabozantinib * RET mutation negative MTC, previously treated with vandetanib and cabozantinib * Patients will receive ponatinib 30 mg orally daily until disease progression or until the development of intolerable side effects. * Tumor response will be assessed by RECIST 1.1 criteria at 8 weeks and then every 12 weeks thereafter. After one year on study, tumor response will be assessed every 16 weeks. * Patients will have a biopsy of their MTC for molecular analysis prior to initiating treatment with ponatinib. Patients will also have a biopsy of their MTC at the time of tumor progression, should that occur.
Diarrhea in patients with MTC (Medullary Thyroid Cancer) can be debilitating and, in some cases life threatening. Findings in such patients include volume depletion, renal insufficiency, and electrolyte disorders. Diarrhea can also lead to increased cost of care, reduced quality of life, and treatment delays. Not all patients benefit from conventional anti-diarrheal therapy. CASAD is proven to reduce diarrhea in humans and animals. Clays have water-binding effects, increase the absorptive capacity of the intestinal mucosa, and absorb the excess cytokines which are possible mechanisms of diarrhea in MTC. In this study, we will investigate if starting 1 g CASAD three times a day will ameliorate the severity of diarrhea in patients with MTC. Diarrhea in patients with MTC can be debilitating and, in some cases life threatening. Findings in such patients include volume depletion, renal insufficiency, and electrolyte disorders. We hypothesize that adding CASAD 3 grams/day will reduce the incidence and ameliorate the severity of diarrhea in patients with MTC.
The objective of this study is to provide access to cabozantinib for eligible subjects with medullary thyroid cancer (MTC) pending approval of Exelixis' New Drug Application (NDA) by the FDA and commercial availability of cabozantinib.
Background: - Medullary thyroid cancer (MTC) is a rare cancer of the thyroid gland. In children and adults, it is often part of a condition called Multiple Endocrine Neoplasia 2 (MEN2). MEN2 is usually caused by a genetic mutation, and it can cause a number of problems in addition to MTC. These problems include adrenal gland tumors, hormone changes, and problems with the bones and other organs. Not much is known about how MTC develops over time, especially in people with MEN2. Researchers want to study MTC in children and adults and see how it affects their growth and development. Objectives: - To study how medullary thyroid cancer affects children and adults over time. Eligibility: - Children and adults who have medullary thyroid cancer. Design: * Participants will be screened with a brief physical exam and medical history. Blood and tissue samples will be collected to see whether participants have the MEN2 genetic mutation. * Treatment will not be provided as part of this study. However, participants will be receiving standard care for MTC. They may be eligible for other clinical trials at the National Institutes of Health. * Participants will have regular study visits every 6 to 12 months to evaluate their MTC and any treatment. Blood tests, imaging studies, and other tests may be performed as needed to monitor the disease. * Participants and their parents/guardians will also complete questionnaires about their health and emotions during the study.
The purpose of this study is to determine the effectiveness of two anticancer drugs, everolimus and pasireotide, in patients with thyroid cancer when the cancer is no longer responding to treatment with radioiodine or where it is deemed unsafe for the patient to receive additional radioiodine treatment. The investigators also want to establish the best manner of taking the two medications when used together to treat thyroid cancer. In particular, the investigators want to know if it is better to give both at the same time or add a second medication after the first one has stopped working. This study will also look at specific substances called biomarkers in your blood, and in the tumor tissue which are involved in the growth of tumor cells, and determine if the levels of these biomarkers are related to your response to treatment or development of side effects. Everolimus, also known by the brand name, Afinitor, is a biologic drug approved by the Food and Drug Administration (FDA) for the treatment of kidney cancer. It works by preventing cancer cells from multiplying and it also makes them more likely to die from the treatment. Pasireotide also known by the name, SOM230 is a new medication that is not yet approved by the FDA for the treatment of cancer. It is a newer form of a drug called octreotide, which is approved for the treatment of cancer arising from endocrine organs. Pasireotide works by binding to a protein called somatostatin receptor, which is expressed in many tissues throughout the body including thyroid cancer cells. Pasireotide prevents the action of somatostatin by binding to these receptors.
Background: - Researchers are studying types of thyroid cancer that seem to cluster in families. Non-medullary thyroid cancer accounts for the vast majority of all types of thyroid cancer, but little is known about possible genes that may cause the cancer. More research is needed to develop the best ways to screen for familial non-medullary thyroid cancer (FNMTC) so that it can be diagnosed and treated at an early stage. Objectives: * To evaluate the natural history of FNMTC. * To determine the best screening strategy for FNMTC. * To identify genes that may indicate susceptibility to FNMTC. Eligibility: - Individuals at least 7 years of age who have two first-degree relatives (e.g., parents, children, siblings) who have or have had non-medullary thyroid cancer or a documented diagnosis of non-medullary thyroid cancer and one living relative with documented non-medullary thyroid cancer. Design: * Participants will be evaluated by family history pedigree, physical examination, imaging (including possible neck ultrasound and radioactive iodine scans), and laboratory testing. * Participants who agree to have blood or other biological samples collected will be asked to enroll in an additional study to provide the appropriate samples and tissues. * After the initial study evaluation, participants who are not found to have a malignant thyroid tumor will be re-screened every year with non-invasive imaging studies. Participants who are found to have a malignant thyroid tumor will be informed of possible treatment options....
The purpose of this research study is to evaluate the progression-free survival (PFS) with XL184 as compared with placebo (an inactive substance) in subjects with unresectable, locally advanced, or metastatic medullary thyroid cancer (MTC). Subjects will be randomized to receive XL184 or placebo in a 2:1 ratio. XL184 is an investigational drug that inhibits VEGFR2, MET and RET, kinases implicated in tumor formation, growth and migration. The Clinical Steering Committee for this study, comprised of study doctors who specialize in medullary thyroid cancer, has provided guidance regarding the design of the study. The committee includes: Douglas Ball, MD, Barry Nelkin, PhD, Martin Schlumberger, MD and Steven Sherman, MD.
Primary objective is to evaluate the tumor response rate of patients with MTC treated with Lithium carbonate
The purpose of this study is to learn how hereditary or sporadic medullary thyroid cancer patients, treated with ZD6474, react to the drug, what happens to ZD6474 in the human body, about the side effects of ZD6474, and if ZD6474 can decrease or prevent the growth of tumors.
This will be a Phase II, open label study to establish the effect of once-daily oral doses of ZD6474 100mg in subjects with locally advanced or metastatic hereditary medullary thyroid cancer in whom no standard therapeutic option is available.
This is an open-label phase 1 study to assess the safety and feasibility of autologous T cells expressing a single-chain scFv targeting GFRα4 with tandem TCR/CD3ζ and 4-1BB (TCRζ/4-1BB) co-stimulatory domains (referred to as "CART-GFRa4 cells") in patients with incurable medullary thyroid cancer (MTC).
This is an open-label, multi-center Phase 1/2 study of oral LOXO-292 in pediatric participants with an activating rearranged during transfection (RET) alteration and an advanced solid or primary CNS tumor.
The Personalized Discovery Process is the only program offering patients treatment recommendations based on an empirically constructed Drosophila "fly" model of their disease. Special committee selects one of the one of the few 2-3 FDA approved drug combinations or single agents that improved survival in the fly cancer model.