Short Bowel Syndrome
Conditions
Interventions
All patients will receive the following interventions:
- ECG
- Vital signs and physical examination
- Colonoscopy or CT/MRI
- Urine collection
- Pregnancy test (for females of childbearing potent
Sponsors
Zealand Pharma A/S
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: Patients rolling over from the lead-in trial: The patient must meet all of the following inclusion criteria: 1) Signed informed consent 2) Either of the following: a) Completed the full Treatment Phase of the lead-in trial (ZP1848-17111), regardless of treatment adherence. OR b) Eligible based on the same inclusion/exclusion criteria as in the lead-in trial (patients may be directly screened into this trial) Patients screened directly to the Extension Trial: The patient must meet all of the following inclusion criteria: 1. Informed consent obtained before any trial-related activity. 2. Age >= 18 years and
Exclusion criteria
Exclusion criteria: Patients rolling over from the lead-in trial: The patient must be excluded from this trial if any of the following criteria are met: 1) Withdrew consent from lead-in trial. 2) Any condition, disease, or circumstance that in the Investigator's opinion would put the patient at any undue risk, prevent completion of the trial, or confounds planned assessments of the trial. 3) Use of GLP-1, GLP-2, human growth hormone (HGH), dipeptidyl peptidase-4 (DPP-4) inhibitors, citrulline, somatostatin, or analogs thereof within 3 months. Note: Prior glepaglutide trial drug is allowed. 4) Females of childbearing potential, who are pregnant, breast-feeding, intend to become pregnant, or are not using highly effective contraceptive methods. Highly effective contraception methods and definition of child-bearing potential are described in Section 11.4.4 of there Study Protocol. 5) Committed to an institution by virtue of an order issued either by the judicial or the administrative authorities. 6) An employee of the sponsor or Investigator or otherwise dependent on them. Patients screened directly to the Extension Trial: The patient must be excluded from the trial if he or she meets any of the following criteria: 1. More than 2 SBS-related or PS-related hospitalizations (e.g., catheter related bacteremia/sepsis, bowel obstruction, severe water-electrolytes disturbances, etc.) within 6 months prior to Screening. 2. Poorly controlled inflammatory bowel disease (IBD) that is moderately or severely active or fistula interfering with measurements or examinations required in the trial. 3. Bowel obstruction. 4. Known radiation enteritis or significant villous atrophy, e.g., due to active celiac disease. 5.Cardiac disease defined as: decompensated heart failure (New York Heart Association [NYHA] Class III-IV), unstable angina pectoris, and. 6. Clinically significant abnormal ECG as judged by the Investigator. 7. Repeated (2 or more consecutive measurements separated by at least 15 minutes) systolic blood pressure measurements > 180 mm Hg. 8. Human immunodeficiency virus (HIV) positive, acute liver disease, or unstable chronic liver disease. 9. Any history of colon cancer. History of any other cancers (except margin-free resected cutaneous basal or squamous cell carcinoma or adequately treated in situ cervical cancer) unless disease-free state for at least 5 years. 10. Estimated creatinine clearance (CrCL; by the Cockcroft-Gault formula) = 2 × the upper limit of normal (ULN), or b. Aspartate aminotransferase (AST) >= 5 × ULN, or c. Alanine aminotransferase (ALT) >= 5× ULN 12. Use of GLP-1, GLP-2, HGH, somatostatin, or analogs thereof, within 3 months prior to Screening. 13. Use of DPP-4 inhibitors within 3 months prior to Screening. 14. Systemic immunosuppressive therapy that has been introduced or has been unstable within 3 months prior to Screening. 15. Unstable biological therapy (e.g. anti-tumor necrosis factor alpha [TNF-a], natalizumab, etc.) within 6 months prior to Screening, including significant changes in doses or switch of drug. 16. Females of childbearing potential, who are pregnant, breastfeeding, intend to become pregnant or are not using highly effective contraceptive methods. Highly
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint will be analyzed using the incidence of AEs occurring in each 3-month period for the first year; subsequent years will have longer periods assigned for analysis. All efficacy endpoints will be analyzed using average weekly results over the 3-month period between visits. Safety Endpoints * Incidence and type of AEs (primary endpoint) * Incidence and type of SAEs and AESIs * Changes from baseline in: - Vital signs - Electrocardiogram (ECG) * Changes from baseline in: - Hematology - Biochemistry - Standard bone markers (for patients screened directly only) - Urinalysis * Immunogenicity Efficacy Endpoints The PS-related endpoints are based on actual PS, i.e. as reported by the patient. * Reduction in weekly PS volume from baseline * Reduction of at least 20% in weekly PS volume from baseline * Reduction in days on PS >= 1 day/week from baseline * Reduction in weekly PS volume of 100% (weaned off) | — |
Secondary
| Measure | Time frame |
|---|---|
| * Change in fluid composite effect (FCE) from baseline * Reduction in calculated energy content of parenteral macronutrients from baseline * Reduction in number of days on PS per week from baseline * Reduction of at least 40% in weekly PS volume from baseline * Change in weight from baseline | — |
Countries
Netherlands
Outcome results
None listed