Short Bowel Syndrome (SBS) MedDRA version: 20.1 Level: PT Classification code 10049416 Term: Short-bowel syndrome System Organ Class: 10017947 - Gastrointestinal disorders
Conditions
Interventions
Product Code: FE 203799
Pharmaceutical Form: Powder and solution for solution for injection
INN or Proposed INN: none
CAS Number: 1295353-98-8
Current Sponsor code: FE 203799
Concentration unit: mg mi
Sponsors
GLyPharma Therapeutic, Inc. (a wholly owned subsidiary of VectivBio Holding AG)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Males and females with SBS secondary to surgical resection of the small intestine 2.18-80 years of age 3.Body Mass Index (BMI) between 16.0 and 32.0 (both inclusive) 4.Patients with a jejuno- or ileostomy and a faecal wet weight excretion of at least 1500 g/day, as recorded within the last 18 months according to the patient’s medical record 5.Parenteral support =3 times/week for =12 months according to the patient’s medical record 6.At least 6 months since last surgical bowel resection 7.Willing to adhere to a defined oral intake of fluids on certain days as required by the protocol (and based on the individual’s routine daily consumption) 8.Women of childbearing potential must agree to use an adequate method of contraception during the trial and for 60 days after the end-of-trial visit. Adequate methods of contraception include intrauterine device or hormonal contraception (oral contraceptive pill, depot injections or implant, transdermal depot patch or vaginal ring). To be considered sterilised or infertile, females must have undergone surgical sterilisation (bilateral tubectomy, hysterectomy or bilateral ovariectomy) or be post-menopausal (defined as at least 12 months amenorrhoea may be confirmed with follicle-stimulating hormone [FSH] test) if there is doubt) 9. Male subjects must commit to use barrier contraception (e.g. condom) during the trial and for 2 weeks after the end-of-trial visit. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 8 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: 1.Pregnancy or lactation 2.Positive results on the human immunodeficiency virus (HIV), hepatitis B and/or C tests 3.A history of clinically significant intestinal adhesions and/or chronic abdominal pain 4.Require chronic systemic narcotics for treatment of pain that exceeds an amount corresponding to 80 mg of morphine per day 5.History of cancer or clinically significant lymphoproliferative disease within =5 years, except for adequately treated basal cell skin cancer 6.History of gallstone within the past 3 years. Gallstones with subsequent cholecystectomy to resolve the issues is acceptable 7.Inflammatory bowel disease (IBD) patients who have not been on a stable drug treatment regimen for at least the past 4 weeks 8.Evidence of active IBD in the past 12 weeks 9.Visible blood in the stool within the last 3 months 10.Catheter sepsis experienced within the last 3 months 11.Decompensated heart failure (New York Heart Association [NYHA] class III-IV, see Appendix 12.2) and/or known coronary heart disease defined as unstable angina pectoris and/or myocardial infarction within the last 6 months prior to screening 12.Radiation enteritis, scleroderma or other condition of intestinal dysmotility, coeliac disease, refractory or tropical sprue 13.History of alcohol and/or drug abuse within the last 12 months 14.Inadequate hepatic function as defined by: bilirubin >upper limit of normal (ULN), alanine transaminase (ALT) or aspartate transaminase (AST) >2.0 × ULN; alkaline phosphatase (ALP) >2.5 × ULN; or international normalised ratio (INR) >1.5 × ULN 15.Inadequate renal function as defined by serum creatinine or blood urea nitrogen >2.5 × ULN 16.Unplanned hospitalisation of >24 hours duration within 1 month before the screening visit 17. Changes in systemic corticosteroids, methotrexate, cyclosporine, tacrolimus, sirolimus, infliximab or other biologic therapy/immune modifiers within 3 months of screening 18.Any use of growth hormone, glutamine or growth factors such as native GLP 2 or GLP 2 analogue within the last 3 months 19.Any use of antibiotics within the last 30 days 20.Participation in another clinical trial within the last 3 months and during this trial 21.Previously been randomised in this trial 22.Loss of blood or donation of blood or plasma >500 mL within 3 months prior to screening 23.Patient not capable of understanding or not willing to adhere to the trial visit schedules and other protocol requirements 24.For any other reason judged not eligible by the investigator
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the safety and tolerability of FE 203799 in patients with SBS with intestinal. ;Secondary Objective: To assess the pharmacodynamics (PD) of FE 203799 in patients with SBS with intestinal failure To assess the trough concentration and the post dose plasma concentration of FE 203799 in patients with SBS with intestinal failure To evaluate markers indicative of a pharmacological effect ;Primary end point(s): Adverse events (AEs; type, frequency and intensity), vital signs (systolic and diastolic blood pressure, heart rate), electrocardiogram (ECG; intervals, rhythm and morphology), clinical chemistry, haematology, haemostasis and urinalysis ;Timepoint(s) of evaluation of this end point: From baseline run-in to End of trail visit, in total during 42 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): •Change from baseline in urinary output over 48 hours at the end of the treatment period (Days 26-28) •Change from baseline in urinary output over 48 hours immediately after first dose in the treatment period (Days 1-3) •Change from baseline in urinary Na+ levels over 48 hours immediately after first dose (Days 1-3) •Change from baseline in urinary Na+ levels over 48 hours at the end of the treatment period (Days 26-28) •Change from baseline in PS volume during the treatment period (Days 21+22) •Change from baseline in oral fluid intake during the treatment period (Days 21+22) •Change from baseline in plasma citrulline levels on Day 8 and at the end of the treatment period (Day 29) •Change from baseline in lean body mass and other body composition parameters (bone mineral content and fat mass) using dual-energy X-ray absorptiometry (DEXA) scan at the end of the treatment period (Day 29) •Plasma concentration of FE 203799 at 72 hours (C72h) and trough concentration of FE 203799 (Ctrough) ;Timepoint(s) of evaluation of this end point: From baseline run-in to End of trail visit, in total during 42 weeks | — |
Countries
Denmark
Contacts
Public ContactChristian Meyer
GLyPharma Therapeutic, Inc. (a wholly owned subsidiary of VectivBio Holding AG)
Outcome results
None listed