Short Bowel Syndrome
Conditions
Keywords
Intestinal Failure, Pathologic Processes, Malabsorption Syndromes, Intestinal Diseases, Gastrointestinal Diseases, Digestive System Diseases, Postoperative Complications, Short Bowel Syndrome, Syndrome
Brief summary
The purpose of the present Phase 3 trial is to confirm the efficacy and safety of glepaglutide 10 mg twice weekly in a patient population with SBS-IF and generate additional long-term safety data. Glepaglutide is the International Nonproprietary Name and United States Adopted Name (USAN) for ZP1848.
Detailed description
The trial is a phase 3, double-blind, randomized, parallel-group, placebo-controlled, multicenter trial to confirm the efficacy and safety of glepaglutide 10 mg twice weekly, followed by a long-term, open-label safety evaluation in patients with short bowel syndrome-intestinal failure (SBS-IF).
Interventions
DRUGGlepaglutide
Subcutaneous (SC) injections twice weekly
OTHERPlacebo
SC injections twice weekly
Sponsors
Zealand Pharma
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Intervention model description
Participants are randomly assigned to one of two groups (arms) in parallel: one group receives glepaglutide, and the other group receives a placebo. After this initial phase, both groups receive open-label treatment with glepaglutide.
Eligibility
Sex/Gender
ALL
Age
18 Years to 90 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Signed informed consent; * Age of 18 to 90 years; * A diagnosis of SBS, defined as having a small bowel with an estimated length of less than 200 cm (equal to 79 inches) in continuity (latest intestinal resection ≥6 months before screening); * Stable PS need of ≥3 days per week; * No restorative surgery planned during the trial period; * Having a stoma or colon in continuity. Key
Exclusion criteria
* More than 2 SBS- or PS-related hospitalizations within 6 months before screening; * Poorly controlled Inflammatory Bowel Disease (IBD) that is moderately or severely active or a fistula that can interfere with the measurements or examinations required in the trial; * History of colorectal cancer or any other type of cancer (except for margin-free resected cutaneous basal, squamous cell carcinoma or adequately treated in situ cervical cancer) unless the patient has been disease-free for at least 5 years; ongoing bowel obstruction; * BMI \<18.5 kg/m\^2.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in weekly Parenteral Support (PS) volume | Baseline to Week 24 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of participants achieving clinical response | Baseline to Week 52 | Defined as achieving a reduction of at least 20% in weekly PS volume |
| Proportion of participants with a reduction in days on PS ≥1 day/week | Baseline to Week 52 | — |
| Proportion of participants with a reduction of 100% weekly PS volume (weaned-off) | Baseline to Week 52 | — |
| Proportion of participants achieving 'much better' Patient Global Impression of Change (PGIC) status | Week 24 | PGIC scoring categories are: 'Much better', 'A little better', 'No change', 'A little worse', and 'Much worse' |
| Change in weekly PS volume | Baseline to Week 12 | — |
| Incidence of Treatment Emergent Adverse Events (TEAEs) | Baseline to Week 52 | — |
Countries
Austria, Belgium, Denmark, Finland, France, Germany, Hungary, Italy, Netherlands, Norway, Poland, Spain, Sweden, United States
Outcome results
None listed