An Open-Label, Single-Arm Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Etrasimod in Adolescent Participants with Moderately to Severely Active Ulcerative Colitis
Published: 11/10/2022
General Information:
The purpose of this study is to determine whether oral etrasimod is a safe and effective treatment for moderately to severely active ulcerative colitis in adolescents (≥ 12 years up to < 18 years of age).
Eligibility:
Key Inclusion Criteria:
- Male or female adolescents ≥ 12 years up to < 18 years of age (at the time of enrollment)
- Weight at Screening ≥ 30 kg
- Diagnosed with UC ≥ 4 weeks prior to screening
- Active UC confirmed by endoscopy and histopathology, disease extent involving the distal sigmoid colon
- Moderately to severely active UC defined as MMS of 5 to 9, including an ES ≥ 2 and RB score ≥
- Inadequate response to, loss of response to, or intolerance to at least 1 of the following
- Conventional therapy
- Biologic or JAK inhibitor therapy
Key Exclusion Criteria:
- Severe extensive colitis
- Diagnosis of Crohn’s disease or indeterminate colitis
- Positive assay or stool culture for pathogens or positive test for C. difficile (can re-screen after treatment)
- Severe infections, active or latent tuberculosis
- Certain cardiovascular conditions or treatments
- Decreased pulmonary function
- History of macular edema or retinopathy
- Treatment with ≥ 3 biologic agents or ≥ 2 biologics plus a JAK inhibitor approved for treatment of UC
- Treatment with rectal 5 ASA or CS ≤ 2 Weeks prior to or during screening
Additional criteria will be reviewed by the study team at the first visit.
Study Details:
Study description/explanation of participation:
Adolescent patients diagnosed with moderately to severely active UC may be eligible to participate in this study. UC is a chronic condition that is characterized by inflammation (pain, swelling, and redness) of the colon (part of the bowel or large intestine) caused by an over-reaction of the immune system (the body’s defense system, which fights infection). The purpose of this study is to assess an oral once-daily investigational drug called etrasimod for its safety and effectiveness in treating moderately to severely active UC in adolescents (≥ 12 to < 18 years of age). This is a Phase 2 study which consists of 3 periods: a screening period, a 52-week treatment period, and a follow-up period. Participants have the option to continue in a Long-Term Extension (LTE) Period of up to 4 years (5 years after study enrollment) or until marketing authorization is obtained in their country. Participants who discontinue or do not enter the LTE Period will have 2-Week and 4-Week Follow-Up Visits after Week 52 or Early Termination.
Description of treatment or intervention (mechanism of action):
Participants who qualify for the APD334-207 study, will be assigned to either 1 mg or 2 mg study drug according to their weight. Participants weighing 30 kg to < 40 kg will receive a 1 mg etrasimod and participants weighing ≥ 40 kg will receive 2 mg etrasimod.
In people with UC, some kinds of cells in the body (part of your immune system called “lymphocytes”) leave lymph nodes and travel to the colon where they may contribute to inflammation and tissue damage. Etrasimod, the study drug, is a substance designed to block some lymphocytes from leaving lymph nodes. It is hoped that by doing so, the study drug may reduce inflammation in the colon, leading to an improvement in UC.
Patient participation requirements:
- This is a once-daily oral drug
- Over the course of the study, participation will require approximately 12 clinic visits.
- A variety of assessments such as lab tests, vital signs, physical examination, colonoscopies, pulmonary function tests, eye examination, stool collection, electrocardiogram etc. will be performed.
- Participants will need to complete some questionnaires during clinic visits and to keep an e-diary daily
Possible risks and side effects:
Based on experience with other medicines that are believed to work in a way that is similar to etrasimod, the following are potential risks that may be associated with the study drug, including an increased risk of infection (due to a reduction in the white blood cells that are part of the immune system), an increase in blood pressure, macular edema (swelling in part of the retina, in the eye), decreased lung function test results, changes in blood test results that look at liver function, and risks to the unborn child if a pregnant woman receives the treatment during pregnancy.
This is not a complete list of the potential risks and side effects of etrasimod and the study investigator will go over this with you in more detail.