Clinical research may involve healthy volunteers or patients with specific illnesses. Sometimes it focuses on prevention, but other times it involves studying the effects of new therapies. Gastrointestinal Specialists of Georgia conducts various studies, or clinical trials, for diseases such as NASH, hepatitis C, Crohn’s disease, ulcerative colitis, and Clostridium difficule, or C. diff.
A clinical trial takes the research beyond a medical laboratory. Clinical studies observe volunteers in a normal setting and use data from medical exams and tests, as well as questionnaires, to measure changes over time. A clinical trial differs in that it evaluates how a drug, diet, surgical procedure, medical device, or behavioral intervention is more or less effective/harmful than standard treatments.
What Are Clinical Research Trials?
During clinical research trial, a medical treatment in development is tested on a pre-selected group of patients. Each one is defined by a series of protocols. These define:
- The types of patients accepted into the study
- Schedules for medical tests/procedures
- The kinds of drugs administered
- Dosages each patient receives
- The length of the study
- Procedures for measuring outcomes
The trial may evaluate how effective a new treatment is, or it might focus on early detection of a disease. Some studies are aimed at prevention of health problems. Others look at how to improve quality of life for patients diagnosed with a life-threatening or chronic illness.
Clinical trials must be approved by the U.S. Food and Drug Administration (FDA) before they can begin. However, the potential treatment or therapy is explored in advance. Scientists study its safety and efficacy on animals in a laboratory before human trials begin.
Phases of Clinical Trials
Once approved by the FDA, a clinical trial can advance through four phases. Drug trials are typically identified by their phase, to reveal how far along in the process a compound is, and if it will be approved for use among the general population. The trial phase is broken down into:
- Phase I: The safety and side effects of an experimental treatment are studied on a small group of healthy individuals. Researchers look for correct dosages as well. The group is usually somewhere between 20 and 80 people.
- Phase II: The effectiveness of a treatment is studied on a larger group, often 100 to 300 people, potentially over several years. Preliminary data show whether the drug is effective for treating a medical condition or disease. Side effects may also be measured.
- Phase III: Drugs are studied among different populations and at different dosages. Often involving thousands of people, a Phase III trial may examine the effects of a drug in combination with others. If the results are favorable, the FDA will conduct a review and decide whether to approve the experimental drug/device.
- Phase IV: The effectiveness and safety of a drug are monitored in large, diverse populations long after FDA approval. By studying larger populations over longer time periods, the long-term benefits and side-effects can be more accurately monitored.
Where to Find the Latest Disease Treatments at No Charge
To sign up for clinical research trials, you usually need to contact the respective department within the organization responsible for the study. For example, Gastrointestinal Specialists of Georgia has a Clinical Research Department. You can search clinical trials by body area, protocol, or keywords, or review the most current trials online to see requirements and details on the treatment. However, larger databases of clinical trials exist, such as the FDA, National Cancer Institute, and ClincialTrials.gov, where you can search for privately and publicly funded clinical studies.
With clinical trials, research is conducted on specific drugs, conditions, and diseases. The conditions currently being studied by GI Specialists of Georgia are explained below. Patients have access to studies if treatments for a particular disease aren’t available or a standard treatment has not been effective. If so, they may qualify to join a clinical trial and receive the latest treatment at no charge.
Diseases Studied by GI Specialists of Georgia
NASH- Non-alcoholic Steatohepatitis
Currently there is not a FDA approved medication for NASH, however researchers are working towards changing that by conducting clinical trials. Nonalcoholic Steatohepatitis (NASH) is a chronic condition in which the buildup of fat and inflammation in the liver may eventually lead to severe scarring called fibrosis, which may advance and require a liver transplant. It is currently the second leading cause of liver transplant in the U.S. Because people with NASH rarely experience symptoms, the disease can progress for years without a diagnosis.
To diagnose this condition, blood test can measure the liver enzymes to check for inflammation in the liver, an elastography (Fibroscan) can reveal the amount of fat and potential scarring in the liver, and a liver biopsy can give the physician a more precise measurement of the amount of fat and scarring in the liver.
NASH is associated with pre-diabetes, type 2 diabetes, being overweight or obese, high cholesterol and high blood pressure.
Lifestyle changes may help to reduce the amount of fat being stored in the liver. Even if you have nonalcoholic fatty liver disease, it can help to avoid drinking. If you are overweight or obese, do what you can to gradually lose weight — no more than 1 or 2 pounds a week.Eat a balanced and healthy diet and get regular exercise. Limit high-carb foods such as bread, grits, rice, potatoes, and corn. And eliminate drinks with lots of sugar like sports drinks and juice.
If left untreated, the additional fat stored in your liver can lead to cirrhosis or scarring of the liver. Scarring may take 20 years or more to form but can interfere with liver function. The additional fat can also increases the risk of liver cancer, while advanced cirrhosis can lead to liver failure.
Clinical research trials have helped make inroads with hepatitis C (HCV) treatment. The viral infection, which causes liver inflammation and can lead to major damage, had up until recently been treated with oral medication and weekly injections. The protocol has changed. Oral “direct-acting” anti-viral medications, taken daily from two to six months, can usually cure the virus. Treatment times are generally shorter, and the current medications have fewer side effects.
Still, the main challenge is diagnosing people who have it. About half of people infected don’t know it, as symptoms often aren’t noticeable for decades. People born between 1945 and 1965 are five times more likely to be infected.1
Getting diagnosed requires a blood test. The CDC recommends everyone at an increased risk of contracting HCV be screened at least once. Blood tests can measure the quantity and genotype of the virus, and tests for liver damage include liver biopsy, magnetic resonance elastography, and transient elastography.
Signs of hepatitis C include:
- Easy bleeding and bruising
- Dark-colored urine
- Poor appetite
- Itchy skin
- Weight loss
- Leg swelling
- Abdominal fluid buildup
- Spider-like blood vessels on skin
- Confusion/drowsiness/slurred speech
If not treated, an HCV infection can lead to cirrhosis or scarring of the liver. Scarring may take 20 years or more to form but can interfere with liver function. The virus also increases the risk of liver cancer, while advanced cirrhosis can lead to liver failure.
This inflammatory bowel disease can spread into deep tissue layers. Sometimes painful and debilitating, Crohn’s disease can cause inflammation in different parts of the digestive tract. There’s no known cure, but the disease is diagnosed using blood tests for anemia or infection, and by testing blood found in a stool sample.
A colonoscopy allows a doctor to view the colon and look for inflammatory cell clusters. Biopsies may be performed to further analyze the tissue in a laboratory. Medical imaging such as CT and MRI scans provide detailed views of organs and tissues. Additional procedures include capsule endoscopy, in which a small camera is swallowed and passes through the small intestine, and balloon-assisted enteroscopy, which reaches farther than a standard endoscope; it’s used when the capsule camera shows an abnormality.
Symptoms of Crohn’s disease include:
- Abdominal pain
- Blood in stool
- Mouth sores
- Reduced appetite
- Weight loss
- Pain or drainage near the anus
Complications include bowel obstruction, ulcers, fistulas (abnormal connections between body parts), anal fissure, malnutrition, and colon cancer.
Clinical trials research has helped doctors find treatments for Crohn’s, although there is still no cure. The disease is treated with anti-inflammatory drugs such as corticosteroids or oral 5-aminosalicylates. Immune system suppressors and antibiotics may also be used. Many patients are treated with anti-diarrheal medicines, vitamin B-12 shots, and iron, calcium, and vitamin D supplements. Nutrition therapy, intravenously or via a feeding tube, may be used to boost nutrition and rest the bowel to reduce inflammation.
Also an inflammatory bowel disease, ulcerative colitis causes long-lasting inflammation and ulcers in the inner lining of the large intestine and rectum. The condition may be debilitating, and complications can be life-threatening. It is diagnosed using blood tests, stool samples, a colonoscopy, flexible sigmoidoscopy in case of severe inflammation, and imaging such as x-rays and CT scans. Tests to examine the small intestine may also be performed.
There are several types of ulcerative colitis:
- Ulcerative proctitis: Affects only the area near the rectum.
- Proctosigmoiditis: Involves the rectum and sigmoid colon.
- Left-sided colitis: From the rectum to the sigmoid and descending colon.
- Pancolitis: Affects the entire colon.
- Acute severe ulcerative colitis: A rare form, affecting the entire colon and causing severe symptoms that prevent one from eating.
In general, symptoms of ulcerative colitis include:
- Diarrhea with blood or pus
- Abdominal pain/cramps
- Rectal pain/bleeding
- Urge to use the bathroom
- Weight loss
- Stunted growth in children
Treatments vary from anti-inflammatory drugs to immune system suppressors, to antibiotics, anti-diarrheal medications, pain relievers, and iron supplements. Surgical options include removing the colon and rectum, but dietary changes, stress management, and alternative medicines can help control flare-ups.
Most common in older adults in hospitals and long-term care facilities, C. difficile typically occurs after antibiotic regimens. Infections have become more common, even in younger individuals not considered high risk. The bacteria are found in the environment, food, and feces; its spores can survive for weeks or months.
C-diff is diagnosed through stool tests, colon examination, and imaging tests. A severe infection can cause:
- Frequent, watery diarrhea
- Abdominal pain/cramps
- Blood/pus in stool
- Rapid heart rate
- Loss of appetite
- Weight loss
- Abdominal swelling
- Kidney failure
C-diff is often treated with antibiotics, but an alternative treatment, yet to be FDA-approved, is a fecal microbiota transplant. Medical instruments are used to place a donor’s stool inside the patient’s colon to restore healthy intestinal bacteria. Donors are carefully screened for infections and medical conditions. Probiotics, including bacteria and yeast, may be administered to restore balance to the intestinal tract, and, therefore, the body’s natural ability to fight off infection.
Do I Qualify for a Clinical Trial?
Qualifying for a clinical trial means meeting specific requirements. Age group, duration of the disease, response to previous treatments, and medical test results factor in. Treatments often have a different focus than available options. For example, Gastrointestinal Specialists of Georgia is currently running a trial using filgotinib, a selective inhibitor of Janus kinase 1, to treat Crohn’s disease.2
A Phase III study is also evaluating the efficacy and safety of an induction and maintenance treatment for moderate to severely active Crohn’s disease,3 while another is evaluating the use of Combination RHB-104 among patients currently receiving other indicated treatments.4
An ulcerative colitis trial is studying the impact of a peptide dimmer (PTG-100) to inhibit the movement of leukocytes into the gut. The aim is to reduce inflammation and the signs/symptoms of the disease in subjects from 18 to 70 years old.5
Clinical research trials consist of new or experimental treatments. They’re often open to anyone with a specific condition or illness who hasn’t responded to conventional treatments. Each protocol has a unique set of rules, which a participant must agree to and abide by to remain in the study.
To find out if you qualify, researchers will review your medical history. For example, Gastrointestinal Specialists of Georgia’s Clinical Research Department can be reached at 678-819-4237 to see if you are eligible to take part in clinical trials for NASH, hepatitis C, Crohn’s disease, ulcerative colitis, or C-diff. Search for studies online or call today to discuss your eligibility.