Short Bowel Syndrome
Conditions
Brief summary
A Study of Vurolenatide in Adult Patients with Short Bowel Syndrome.
Detailed description
A Phase 2 study of Vurolenatide in adult patients with SBS. Patients were planned to be dosed in four treatment groups with placebo and/or active Vurolenatide. The study is 13 weeks (3 weeks screening, 4 weeks SC study drug administration, and 6 weeks of follow up). Safety and efficacy will be analyzed.
Interventions
DRUGPlacebo
PBO - weekly SC administration
DRUGVurolenatide 50mg/PBO
Vurolenatide - 50 mg biweekly SC administration, PBO alternate weeks
DRUGVurolenatide 100mg/PBO
Vurolenatide - 100 mg biweekly SC administration, PBO alternate weeks
DRUGVurolenatide 50/50 mg
Vurolenatide - 50 mg weekly SC administration
Sponsors
9 Meters Biopharma, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
Double-Blind, Double-Dummy, Placebo-Controlled.
Intervention model description
Double-Blind, Double-Dummy, Placebo-Controlled.
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: Criteria for patient inclusion in this study are as follows: 1. Male and female adults with SBS secondary to surgical resection of small intestine 2. 18-75 years of age at the time of screening. 3. Female patients must be postmenopausal (at least 2 years prior to dosing) or surgically sterile or agree to use an acceptable form of birth control from screening until 30 days after last dose. If oral contraceptives are used, patients must have been on a stable dose for ≥6 months. 4. Male patients must agree to use an acceptable form of birth control during the study and for 30 days after the last dose. Male patients should not donate sperm for 90 days after last dose. 5. At least 6 months since last surgical bowel resection. 6. Patients may be on Parenteral Support \[PS\] (nutrition and/or fluid and electrolytes for at least some of their nutritional needs). 7. If on PS, stable administration of PS volume for 1 month (±20% vol) prior to enrollment \[stable administration of PS volume confirmed by Medical Monitor\]. 8. Able to ingest solid or semi-solid foods and drink. Key
Exclusion criteria
Criteria for exclusion from participation in this study are presented below. 1. Pregnancy or lactation 2. Body mass index at screening \<18 or \>30 kg/m2 3. Clinically significant intestinal adhesions and/or chronic abdominal pain that can interfere with the conduct of the study 4. Active Crohn's disease or IBD (as evaluated by standard procedures employed by the investigator/institution). If in remission, must be ≥12 weeks of remission prior to screening 5. Inflammatory bowel disease patients who have NOT been on a stable drug treatment regimen for at least the past 3 months prior to screening 6. Visible blood in the stool within the last 3 months 7. Known heart failure or active coronary disease 8. Alcohol or drug abuse within the last 12 months by history, or a disqualifying positive urine drug test at screening. 9. Inadequate renal function as defined by serum creatinine \<0.7 or \>1.3 mg/dL (in men) and \<0.6 or \>1.1 mg/dL in women. 10. Personal or family history of medullary thyroid cancer. 11. History of pancreatitis 12. Any use of growth hormone, or growth factors such as native GLP-2 or GLP-2 analog (teduglutide) within the last 3 months 13. Use of antibiotics within the last 30 days 14. Patient not capable of understanding or not willing to adhere to the study visit schedules and other protocol requirements
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 24hr total stool output volume | 10 weeks (including 6 weeks follow up) | The primary outcome measure is 24hr total stool output volume over the double-blind treatment period compared to baseline. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Assess the safety and tolerability of vurolenatide | 10 weeks (including 6 weeks follow up) | Assessment of Adverse events - The frequency of spontaneously reported AEs will be assessed for each treatment group separately by System Organ Class and Preferred Term; and vital signs, physical examination results, clinical laboratory tests results will be evaluated. |
| To assess change from Baseline in Quality of Life | 10 weeks (including 6 weeks follow up) | Change from baseline in Quality of Life as measured by the SF-36 instrument over the double-blind treatment period |
| To assess the change from baseline in Patient Global Impression | 10 weeks (including 6 weeks follow up) | Change from baseline in patient reported global SBS improvement over the double-blind treatment period |
Countries
United States
Outcome results
None listed