Clinical trials (also known as clinical research) are research studies in human volunteers that investigate treatments and observe patient performance with new treatments. They play an important role in developing new treatment options for a variety of diseases. Before any new treatment can be tested in humans, it must have shown positive results in the laboratory and/or in animal studies. Interventional trials determine whether experimental treatments or new ways of using known therapies are safe and effective under controlled environments. Observational trials address health issues in large groups of people or populations in natural settings. The United States Food and Drug Administration (FDA) monitors most clinical trials to protect the participants and the general public. Ultimately, the FDA reviews and analyzes data from successful clinical trials to determine whether the experimental treatment should be approved for the treatment of a specific disease or disorder. Clinical trials may be carried out using completely new treatment options or using treatments that are already available. Clinical trials are the safest and quickest way to confirm whether new treatments are truly beneficial for patients.
Why Are Clinical Trials Important?
Clinical trials offer a look at future treatments. They determine whether a new and potentially better treatment is safe and effective in humans. Clinical trials may provide opportunities for you to receive a promising drug or treatment. Typically, currently approved therapies are compared with new therapies and your doctor may feel this is an appropriate treatment for you.
Things You Should Know Before Participating in A Clinical Trial
Talk To Your Doctor
You should always discuss the benefits and risks of participating in clinical trials with your doctor.
Eligibility
Each clinical trial will have its own set of eligibility requirements. Your doctor will be able to help assess your eligibility for a specific trial.
Informed Consent
The FDA requires that an individual read and sign an informed consent form before being enrolled in a clinical trial. Informed consent is a process that ensures patients are given complete information about a clinical trial prior to their participation. The key facts of the study are included in a written informed consent form for participants to read and discuss with their families and doctors. Be sure you understand all the facts before you sign the consent form. Don’t be afraid to ask questions!
Benefits And Risks of Clinical Trials
Potential Benefits:
There is the possibility that drugs or treatment program used in the trial will be better than currently approved treatment options
Patient care is provided by top doctors and researchers at leading healthcare facilities
You will have access to new treatment strategies that may not otherwise be available
Your progress will be closely monitored
Your participation may help to advance the treatment of the disease
Potential Risks:
There is the possibility of unknown or unexpected side effects
There is always the chance that the new drug or treatment may be ineffective or less effective than current options
Participation in the clinical trial may require a greater time commitment for treatment due to extra clinic visits for treatments or monitoring
You need to factor in the potential of additional costs that may be involved, such as housing and transportation, insurance payment for treatment outside covered network, etc.
Clinical Trial Phases
Phase I studies assess the safety of a drug or device. This initial phase of testing, which can take several months to complete, usually includes a small number of healthy volunteers (20 to 100), who are generally paid for participating in the study. The study is designed to determine the effects of the drug or device on humans including how it is absorbed, metabolized, and excreted. This phase also investigates the side effects that occur as dosage levels are increased. About 70% of experimental drugs pass this phase of testing.
Phase II studies test the efficacy of a drug or device. This second phase of testing can last from several months to two years and involves up to several hundred patients. Most phase II studies are randomized trials where one group of patients receives the experimental drug, while a second “control” group receives a standard treatment or placebo. Often these studies are “blinded” which means that neither the patients nor the researchers know who has received the experimental drug. This allows investigators to provide the pharmaceutical company and the FDA with comparative information about the relative safety and effectiveness of the new drug. About one-third of experimental drugs successfully complete both Phase I and Phase II studies.
Phase III studies involve randomized and blind testing in several hundred to several thousand patients. This large-scale testing, which can last several years, provides the pharmaceutical company and the FDA with a more thorough understanding of the effectiveness of the drug or device, the benefits, and the range of possible adverse reactions. 70% to 90% of drugs that enter Phase III studies successfully complete this phase of testing. Once Phase III is complete, a pharmaceutical company can request FDA approval for marketing the drug.
Phase IV studies, often called Post Marketing Surveillance Trials, are conducted after a drug or device has been approved for consumer sale. Pharmaceutical companies have several objectives at this stage: (1) to compare a drug with other drugs already in the market; (2) to monitor a drug’s long-term effectiveness and impact on a patient’s quality of life; and (3) to determine the cost-effectiveness of a drug therapy relative to other traditional and new therapies. Phase IV studies can result in a drug or device being taken off the market or restrictions of use could be placed on the product depending on the findings in the study.
Clinical Trial Resource Center
Welcome to the National Pancreas Foundation Clinical Trials Resource Center, presented in partnership with CenterWatch, the leading publisher of information on clinical research for patients, their advocates and healthcare professionals. Please click on the links below to learn more about clinical research and about new medical therapies for treating pancreas disease and related indications.
Clinical Trials Resources
Animated Pancreas Patient: Before enrolling, learn what clinical trials are, how they are conducted, and why they are important for patients with diseases like pancreatic cancer. The animation also provides an overview of study design, eligibility criteria, informed consent, safeguards, different phases of clinical trials, and the potential benefits and potential risks of participation. The videos and animations are easy to digest and answer many patient questions on the importance of clinical trials.
Center Watch: Search and browse to find a trial relevant to you. Read the trial details on eligibility, location, and participation. You can also register to be notified when a trial you’re searching for begins.
Clinicaltrials.gov: ClinicalTrials.gov is a resource provided by the U.S. National Library of Medicine.Explore 434,833 research studies in all 50 states and in 221 countries.
National Institutes of Health: This listing includes government-funded clinical research studies being conducted by the various National Institutes of Health.
Lustgarten and Let’s Win: Explore which clinical trials may be available to you by using this free, unbiased matching service.
NIH Clincal Center Trails: The National Institutes of Health (NIH) Clinical Center Search the Studies site is a registry of publicly supported clinical studies conducted mostly in Bethesda, MD.
This is a comprehensive listing of drugs that have recently been approved by the Food and Drug Administration. The drugs are organized by therapeutic area.
Here you’ll find links to news articles and reports on recent advances in clinical research. This section is updated weekly, and the information has been published in CenterWatch publications during the past weeks. There may not always be gastroenterology-related material included.
Contains a variety of reports, brochures, and publications that patients and their advocates can use to learn more about the clinical trials industry and how to identify and volunteer for clinical trials.
Cecilia Shares Her Clinical Trial Journey
Ever wondered what goes on in a clinical trial? Cecilia Petricone shares her experience being part of the “Anti-inflammatory therapy to improve outcomes in patients with chronic pancreatitis undergoing TPIAT” clinical trial with the University of Minnesota.
This research study is being conducted by the Type 1 Diabetes in Acute Pancreatitis Consortium (T1DAPC) which is funded by the National Institute of Diabetes and Digestive and Kidney Diseases. The DREAM study will enroll adult participants between the ages of 18 and 75 years old who have been recently diagnosed with acute pancreatitis. Participants with or without a diagnosis of diabetes may be eligible to participate. The purpose of this study is to find out if having acute pancreatitis increases your risk for diabetes. Eligible participants will receive compensation for completing study visits.
This research study—called the DETECT* Study – is funded by the National Institutes of Health (NIH) and is seeking to develop a blood test that can separate diabetes caused by pancreatic cancer or chronic pancreatitis from type 2 diabetes.
The study includes participants from 8 research centers in the United States. If you have pancreatic cancer or chronic pancreatitis, and have also been diagnosed with diabetes, and are 40 to 85 years old, you may be eligible after review of other factors.
This NIH-sponsored Randomized Clinical Trial is currently enrolling subjects to learn whether an Endoscopic Retrograde CholangioPancreatography (ERCP) with sphincterotomy is beneficial in patients with acute recurrent pancreatitis and pancreas divisum.
The purpose of this study is to determine if a ERCP with sphincterotomy reduces the risk of pancreatitis or the number of recurrent pancreatitis episodes in patients with pancreas divisum. ERCP with sphincterotomy is a procedure where doctors used a combination of x-rays and an endoscope (a long flexible lighted tube) to find the opening of the duct where fluid drains out of the pancreas. People who have been diagnosed with pancreas divisum, have had at least two episodes of pancreatitis, and are candidates for the ERCP with sphincterotomy procedure may be eligible to participate. Participants will be randomly assigned to either have the ERCP with sphincterotomy procedure, or to have a “sham” procedure. Participants will have follow up visits 30 days after the procedure, 6 months after the procedure, and continuing every 6 months until a maximum follow-up period of 48 months.
You may qualify if:
If you are at least 18 years old
You have had more than one attack of acute pancreatitis
No clear cause of your pancreatitis has been identified
Chronic pancreatitis is a rare but debilitating condition associated with chronic abdominal pain, diarrhea, diabetes, and an 8-fold increased risk for the development of pancreatic cancer. Unfortunately, there is no available treatment to prevent the progression of chronic pancreatitis, and most subjects require narcotic medications to control the pain. A receptor protein call the CCK-B receptor becomes activated in chronic pancreatitis and is in part responsible for the scar tissue or fibrosis that occurs and responsible for the cancer risk. In mice with chronic pancreatitis, the inflammation and damage were reversed with an old drug called proglumide that blocks the activation of the CCK-B receptor. Proglumide has also been shown to possibly reduce pain.
This protocol involved a 2-Part study to test the safety of oral proglumide in those with confirmed chronic pancreatitis and the second goal is to determine if proglumide improves pain and function of the pancreas. Part-1 is an open-labelled Lead-in Study of N=8 subjects over a 12-week treatment period. Part-2 is a randomized double-blind pseudo cross over study where subjects will be treated in Arm A (placebo for 12 weeks followed by 12 weeks of proglumide) and Arm B (proglumide for 24 weeks).
This research study is for children with pain from chronic pancreatitis. Researchers at Seattle Children’s Hospital want to learn more about pain management strategies to help youth with pancreatitis! Children ages 10-18 and their parents can earn up to $300 for their time. Study participation is completely online.
Answer questions about your pain,
mood, sleep, and everyday life at 3
times over 9 months.
Use a website for 8-12 weeks, to
learn pain coping skills, which might
help with managing pain
This study may be a good fit if:
Your child is between 10 and 18 years old
You child has frequent abdominal pain
Your child has chronic or acute recurring pancreatitis
You both have access to the internet
To participate in this study or learn more, please fill out the form located at https://is.gd/webmapcpcommunity. If you or a family member have questions about the study, please contact Seattle Children’s staff at 1-855-932-6272 (toll-free) or palermolab@seattlechildrens.org.
This research study—called the DETECT* Study – is funded by the National Institutes of Health (NIH) and is seeking to develop a blood test that can separate diabetes caused by pancreatic cancer or chronic pancreatitis from type 2 diabetes.
The study includes participants from 8 research centers in the United States. If you have pancreatic cancer or chronic pancreatitis, and have also been diagnosed with diabetes, and are 40 to 85 years old, you may be eligible after review of other factors.
This phase II trial investigates how well the addition of olaparib following completion of surgery and chemotherapy works in treating patients with pancreatic cancer that has been surgically removed (resected) and has a pathogenic mutation in BRCA1, BRCA2, or PALB2. Olaparib is an inhibitor of PARP, an enzyme that helps repair deoxyribonucleic acid (DNA) when it becomes damaged. Blocking PARP may help keep tumor cells from repairing their damaged DNA, causing them to die. PARP inhibitors are a type of targeted therapy.
Inclusion Criteria: Resected pancreatic cancer w/o evidence of disease recurrence
Received ≥3mo multi-agent chemotherapy in the perioperative setting
Within 8 weeks of therapy completion
Pathogenic germline or somatic variant in BRCA or PALB2 (by local lab; confirmed by central review)
If variant identified in tissue, agreement to undergo germline testing
Exclusion Criteria: Any history of progressive disease during platinum therapy
Uncontrolled gastrointestinal disorder that would interfere with the absorption of Olaparib. History of MDS/AML.
Inclusion Criteria: Adults aged 18 years or older. Diagnosed within 8 weeks with upfront resectable, borderline resectable, or locally advanced unresectable PDAC as defined by the National Comprehensive Cancer Network (NCCN). Planning to undergo TNT at MGH. Planning to receive modified FOLFIRINOX for neoadjuvant chemotherapy. Planning to undergo surgical resection of PDAC at MGH. Verbal fluency in English.
Exclusion Criteria: Adults aged 18 years or older. Diagnosed within 8 weeks with upfront resectable, borderline resectable, or locally advanced unresectable PDAC as defined by the National Comprehensive Cancer Network (NCCN). Planning to undergo TNT at MGH. Planning to receive modified FOLFIRINOX for neoadjuvant chemotherapy. Planning to undergo surgical resection of PDAC at MGH. Verbal fluency in English.
This phase III trial compares perioperative chemotherapy (given before and after surgery) versus adjuvant chemotherapy (given after surgery) for the treatment of pancreatic cancer that can be removed by surgery (removable/resectable). Chemotherapy drugs, such as fluorouracil, irinotecan, leucovorin, and oxaliplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving chemotherapy before and after surgery (perioperatively) may work better in treating patients with pancreatic cancer compared to giving chemotherapy after surgery (adjuvantly).
Subjects will be offered the opportunity to participate in a randomized, controlled, 2-arm, unblinded multicenter trial (RCT). There will be 2 study arms: the control arm receiving chemotherapy with the modified FOLFIRINOX regimen alone; and the irreversible electroporation (IRE) arm, receiving chemotherapy with the modified FOLFIRINOX regimen followed by IRE with the NanoKnife System using either an open or a percutaneous approach. All subjects will be treated with the modified FOLFIRINOX regimen for at least 3 months; randomization to either control or IRE arm will take place at the time of completion of the 3-month modified FOLFIRINOX chemotherapy regimen. Randomization will be conducted centrally. Subjects will be randomized in a 1:1 ratio and must be found to have no evidence of disease progression after completion of the 3-month modified FOLFIRINOX chemotherapy regimen in order to participate in the RCT. All radiologic assessments will be performed as consistent with the imaging protocol. All post induction and post IRE treatments are left to the discretion of the treating physician. The minimum period of follow-up will be for 24 months or until death.
Brief Summary:
This phase II trial studies the side effects and how well the combination of binimetinib and encorafenib work in treating patients with pancreatic cancer with a somatic BRAF V600E mutation. Binimetinib and encorafenib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving binimetinib and encorafenib may work better compared to the usual treatment in treating patients with pancreatic cancer and a somatic BRAF V600E mutation
Precision Promise is a multi-center, seamless Phase 2/3 platform trial designed to evaluate multiple regimens in metastatic pancreatic cancer. The goal of the platform is to find effective therapies for pancreatic cancer. The platform will rapidly and efficiently test multiple novel drugs and combinations compared to standard of care therapy in first and second metastatic patients. Bayesian response-adaptive randomization will be used to assign patients to arms based on their performance in subtypes of the disease. The primary endpoint is overall survival.
Primary Objectives
To compare each investigational arm versus standard of care (SOC) for superiority in overall survival in 1st and/or 2nd line metastatic pancreatic cancer patients and determine which, if any, patients benefit from each investigational arm.
Secondary Objectives
To determine short and long-term safety signals of each investigational arm in pancreatic cancer patients vs. SOC.
To determine progression-free survival (PFS) for each investigational arm vs. SOC.
Rates of overall response, CR, and PR; duration of overall response, CR or PR (whichever occurs first).
Rate of clinical benefit; duration of clinical benefit.
Comparing Two Methods to Follow Patients with Pancreatic Cysts
The purpose of this study is to compare the two approaches for monitoring pancreatic cysts. The study doctors want to compare more frequent monitoring vs less frequent monitoring in order to learn which monitoring method leads to better outcome for patients with pancreatic cysts.
Eligibility Criteria:
patient must be >= 50 years and =< 75 years of age
patients must have an Eastern Cooperative Oncology Group (ECOG) performance status 0-1
patient must not have acute pancreatitis or a history of chronic pancreatitis
patient must have received a CT, MRI, or EUS within 6 months prior to randomization that revealed one or more >= 1 cm pancreatic cyst (s)
patients of childbearing potential must not be known to be pregnant
patient must not have a prior diagnosis of pancreatic malignancy of any type
patient must not have a history of pancreatic resection
patients with only pancreatic lesions without malignant risk (pancreatic pseudocyst or classic serous cystic lesion) are not eligible
patient must not have a family history of pancreatic adenocarcinoma in one or more first-degree relatives (biological parents, full siblings or children)
patient must not have pancreatic cyst morphology that would prompt immediate surgical consideration (enhancing mural nodule, solid component in cyst, pancreatic duct >= 10 mm, cyst causing obstructive jaundice)
patient must not have a comorbid illness that precludes pancreatic cyst resection
patient must not be participating in an already established surveillance program
Inclusion Criteria: Histological or biopsy proven adenocarcinoma of the pancreas. Cytology is acceptable if histology cannot be obtained. Newly diagnosed non-metastatic PC judged by tumor board to be feasible for SBRT. Completed at least 6 weeks of chemotherapy consisting of FOLFIRINOX, mFOLFIRINOX, or a gemcitabine-based doublet regimen prior to start of SBRT. Remain non-metastatic as confirmed by a CT scan at screening. Female or male subjects ≥ 18 years of age. ECOG performance status of 0-2. Adequate end-organ function.
Exclusion Criteria: Subjects with documented metastatic disease. First-line chemotherapy other than FOLFIRINOX, mFOLFIRINOX, and/or a gemcitabine-based doublet regimen. Prior abdominal RT with substantial overlap in radiation fields. Subjects not recovered/controlled from treatment-related toxicities. Uncontrolled malignancy other than PC. Uncontrolled gastric or duodenal ulcer disease within 30 days of dosing. Visible invasion of bulky tumor into the lumen of the bowel or stomach on endoscopy.
The purpose of the study is to evaluate the efficacy of Olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
The BALANCE study is for AKCEA-APOCIII-LRX in patients with familial chylomicronemia syndrome. It will be “a global, multi-center, randomized, double-blind, placebo-controlled study enrolling up to 60 patients (age 18 and over) with FCS.” The study only recruits FCS patients and requires about 74 weeks to complete which includes an up to 8-week screening period, a 53-week treatment period, and a 13-week post-treatment evaluation period. Following the treatment period, eligible patients may have the option of enrolling in an open label extension study.
