Disease information
Ulcerative colitis is a chronic waxing and waning immune disease that is often diagnosed early in life (15-25 years of age) but may occur at any age and may require long term medical treatment. The immune system causes inflammation and ulcers in the lining of the large intestine and rectum. The most common symptoms are bloody diarrhoea, anaemia, tiredness, abdominal pain, faecal urgency as well as pain, cramps and strain when passing stool. The symptoms usually develop over time, rather than sudden onset. Symptoms and disease activity may get worse at times (flare or relapse) and then at other times symptoms may almost completely resolve (clinical remission). It is estimated that 30-60% of patients suffering from UC will have at least one relapse per year. The treatment goal for UC is to achieve and maintain clinical remission and healing in the lining of the large intestine. Since UC is a chronic condition, it may be associated with poor health, disability, reduced quality of life, health care expenditures and the potential for poor social and psychological outcomes. Improved therapies that enable patients to have fewer or no symptoms may improve their overall health and quality of life.
Therapeutic background
Guidelines are established to treat acute episodic UC. Medications for moderate to severe UC aim at modulating the immune response and reducing inflammation and improving symptoms (eg, faecal urgency, abdominal pain, rectal bleeding, etc.)
Treatment for UC may include:
- Topical or oral 5-ASA (mesalamine)
- Topical or systemic corticosteroids (e.g. prednisone or budesonide)
- Immunomodulators (e.g. azathioprine or 6-mercaptopurine)
- anti-TNF-alpha agents (e.g. infliximab, adalimumab, golimumab)
- anti-integrin therapies (e.g. vedolizumab or natalizumab)
- anti-IL12/23 (e.g. ustekinumab)
- Janus kinase inhibitors (e.g. tofacitinib)
- S1P1 modulators (e.g. ozanimod)
- Surgical removal of the colon (colectomy)
Ulcerative colitis is a chronic disease. While symptoms may improve for a period of time, relapse often occurs even among patients on treatment. Therefore, newer therapies that may have improved safety, efficacy, and/or convenience may be important. No one therapy may be right for every patient, so providing patients with better options is a goal for next generation treatment.
If you suffer from moderate to severe UC, you may qualify to participate in this research study. We are testing whether the study drug will improve ulcerative colitis disease activity (disease symptoms and rectal/colon inflammation). If you choose to participate and are eligible, you may be randomly allocated to receive either study drug or placebo (no active treatment), and you will be monitored for improvement in your disease. Approximately 75% of participants will be on study drug in this study during the initial treatment period (ie 12 weeks). After initial treatment period (ie, first 12 weeks), all participants may be eligible to receive study drug.
If you participate in this study, you have the right to withdraw from the research study without providing any reason.
Who can participate?
Candidates who fulfill the following criteria may be eligible for the study:
- Male and female between 18–65 years old
- Have a diagnosis of ulcerative colitis
- You have moderately or severely active ulcerative colitis despite being on medications now or in the past
Other criteria are also required before you may pre-qualify for participation in the study. To find out, please click on the link below and answer the digital questionnaire.
Location(s)
USA
- Chapel Hill
- San Diego
- New York
- Shreveport
- Oklahoma City
- Garland
- Southlake
Reimbursement
There will be no additional costs to you if you choose to participate in this research study. The investigational medication and any tests required by the study are provided by the study team, and you will receive reimbursement for travel expenses incurred by taking part in the research study until your participation is complete.
Please note that taxes may need to be paid on travel expense reimbursement; it might also need to be reported to authorities.
Study description
The research study will assess the safety, tolerability, and the effect of the study drug in moderate to severe ulcerative colitis (UC). You will be provided with a further detailed overview of the study procedures by the study team before participation in the research study. There will be opportunity and time to discuss any questions before starting any study procedures.
The duration of study participation is a maximum of 60 weeks in the four study periods described below:
Screening period
During the screening visit, at the study site, you will be told about the study and get answers to questions about the study. If you agree to participate, you will be asked to sign the informed consent document. If you agree to participate, you will undergo clinical and laboratory assessments that will determine if you are eligible for the study. This will include collection of blood, stool and urine for laboratory analyses, vital signs (heart rate and blood pressure), and height and weight, chest x-ray, ECG, and health questionnaires. You will be given an electronic diary to record your daily ulcerative colitis symptoms. You will also undergo an endoscopy (flexible sigmoidoscopy or colonoscopy) where the study doctor uses a light with a camera to look at the wall of your rectum and colon. Biopsies of the intestinal lining will also be collected during the endoscopy procedure.
Washout period
To participate in the study, some drugs used to treat ulcerative colitis or other diseases will have to be stopped for a period of time before you can be eligible to receive the study drug. This process is called a washout period. The period can vary according to the type of drug you are currently using. The study team will provide you with detailed information about the length of your possible washout period.
Treatment period
If you are eligible to participate in the study, you will be randomly assigned to either study drug or placebo for 12 weeks. Neither you nor the study doctor will know whether you are receiving the study drug or placebo. After 12 weeks, all participants may receive the study drug treatment. The maximum length of exposure to the study drug in this study is up to 52 weeks. If you or other participants do not respond to the study drug, you may be discontinued from treatment. During this period, you will also be filling out an electronic diary daily, and you will undergo endoscopy at Week 12, at Week 52, and/or at the time you stop the study drug. During this period, you will be in contact with the study team and have regular clinic visits to monitor your health.
Follow-up period
After termination of the study treatment, you will enter a 4-week post-treatment follow-up period. Like in the treatment period, you will be in contact with the study team to monitor your health.
Timeframe
In the figure below, you can see the maximum duration of the different periods of the research study.
