A Study to Investigate the Efficacy and Safety of Apraglutide Compared With Placebo in Adult Participants With Short Bowel Syndrome Associated With Intestinal Failure (SBS-IF)
This study is investigating a new medication called apraglutide for adults who have a condition called Short Bowel Syndrome with Intestinal Failure (SBS-IF). This means their small intestine isn't long enough or doesn't work properly to absorb all the nutrients they need, so they rely on feeding through a drip (parenteral support). The main aim is to see how well apraglutide works and if it's safe compared to a placebo, which is a dummy medicine with no active ingredients. Around 124 people worldwide will take part, receiving either apraglutide or the placebo once a week for 24 weeks. The study wants to help people with SBS-IF live healthier lives.
At a glance
What is this study about?
This research study is about a condition called Short Bowel Syndrome with Intestinal Failure, often shortened to SBS-IF. This happens when a part of your small intestine is missing or doesn't work well, usually after surgery. Because of this, your body can't absorb enough nutrients from food and drink, and you might need extra feeding through a drip into your bloodstream, known as parenteral support.
The study wants to find out if a new medicine called apraglutide can help people with SBS-IF. It's designed to see if apraglutide is both effective in improving the condition and safe to use. To do this, some people will receive the new medicine, while others will receive a placebo – a dummy medicine that looks the same but has no active ingredients. This helps researchers understand the true effects of apraglutide.
By taking part, you would be helping doctors and scientists learn more about SBS-IF and how best to treat it. The goal is to find new ways to improve the health and quality of life for people living with this challenging condition.
Key takeaways
- The study is testing a new medicine, apraglutide, for adults with Short Bowel Syndrome (SBS-IF).
- It aims to see if apraglutide is safe and effective compared to a dummy medicine (placebo).
- Participants will receive weekly injections for 24 weeks.
- Regular clinic visits and health checks will be part of the study.
- If you complete the study, you may have the option to continue with apraglutide long-term.
- Your decision to join or leave the study won't affect your regular medical care.
Who may be eligible?
This study is looking for adults aged 18 or over who have Short Bowel Syndrome with Intestinal Failure (SBS-IF) and are currently receiving feeding through a drip (parenteral support) at least three days a week. Your small intestine should be between 10 cm and 200 cm long.
There are also some requirements based on your recent surgery. If you still have a connection between your small and large bowel, your last surgery for SBS-IF must have been at least 12 months ago. If you have a stoma (an opening on your belly, like a jejunostomy or ileostomy), your last surgery for SBS-IF must have been at least 6 months ago.
Your body mass index (BMI) needs to be between 18.5 and 30. If you are able to have children, you or your partner would need to agree to use reliable birth control during and for a short time after the study.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 18 years old or older?
- Do you have Short Bowel Syndrome with Intestinal Failure (SBS-IF)?
- Are you receiving feeding through a drip (parenteral support) at least 3 days a week?
- Was your last SBS-IF surgery at least 6 months ago (or 12 months if you still have a continuous bowel)?
- Is your BMI between 18.5 and 30?
- Are you willing to use effective birth control if you or your partner can have children?
What does participation involve?
If you join this study, it will be divided into several stages. First, there's a screening period to check if you're a good fit, which includes optimising your current treatment and making sure your condition is stable. If you qualify, you'll then be randomly assigned to receive either apraglutide or the placebo. You'll receive this medicine once a week, usually as an injection under the skin, for 24 weeks.
Throughout the 24 weeks, you'll have regular visits to the clinic for assessments. These will help doctors monitor your health, how you're reacting to the medicine, and whether it's helping your condition. After you've completed the 24 weeks of treatment, there will be a follow-up period to check on your safety and to look for any remaining traces of the medicine in your body.
Importantly, if you complete the main 24-week treatment period, you might also have the option to join another long-term study where everyone receives apraglutide. If you choose not to join the long-term study, you'll have a final safety check-up about 4 weeks after your last dose of the study medicine.
Potential risks and benefits
Locations (94)
- Ronald Reagan UCLA Medical Center, Center for the Health SciencesVerified postcodeLos Angeles, United States· Not yet recruiting
- Denver Health Medical CenterVerified postcodeDenver, United States· Not yet recruiting
- MedStar Georgetown University HospitalVerified postcodeWashington D.C., United States· Not yet recruiting
- University of Florida Health (UF Health)Verified postcodeGainesville, United States· Not yet recruiting
- GI ProsVerified postcodeNaples, United States· Recruiting
- Cleveland Clinic FloridaVerified postcodeWeston, United States· Not yet recruiting
- Loyola University Medical CenterVerified postcodeMaywood, United States· Not yet recruiting
- UofL Physicians - Colon & Rectal SurgeryVerified postcodeLouisville, United States· Not yet recruiting
- Henry Ford Medical Center - ColumbusVerified postcodeNovi, United States· Not yet recruiting
- Albany Medical CenterVerified postcodeAlbany, United States· Not yet recruiting
- Mount Sinai Medical Center, RMTI - Intestinal Transplantation & RehabilitationVerified postcodeNew York, United States· Not yet recruiting
- Duke University Medical CenterVerified postcodeDurham, United States· Not yet recruiting
Common questions
What is Short Bowel Syndrome with Intestinal Failure (SBS-IF)?
It's a condition where your small intestine can't absorb enough nutrients, often after surgery, meaning you might need extra feeding through a drip.
What is a placebo?
A placebo is a dummy medicine that looks just like the real medicine but has no active ingredients. It helps researchers see the true effect of the new medicine.
How long will I be in the study?
The main treatment part of the study lasts 24 weeks, plus screening and follow-up periods. There might also be an option to join a longer study afterwards.
Will I know if I'm getting the real medicine or the placebo?
No, this is a 'double-blind' study, meaning neither you nor your study doctor will know whether you're receiving apraglutide or the placebo until the study is over.
What is parenteral support (PS)?
Parenteral support means getting nutrition and fluids directly into your bloodstream through a drip, rather than through your digestive system.
How to find out more
Emie Liu, MD
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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