Short Bowel Syndrome
Conditions
Keywords
Short Bowel Syndrome, SBS, Intestinal Failure, SBS-IF
Brief summary
The primary objective of the trial is to assess long-term safety and tolerability of apraglutide in subjects with SBS-IF.
Detailed description
This is an international, multicenter, open label extension trial to collect further safety, tolerability, efficacy, durability, and clinical outcomes of apraglutide once weekly adminstration, for up to 208 weeks or until apraglutide is commercially available in the country, whichever comes first. Eligible subjects would be those who were trial subjects of TA799-007 or TA799-013 trials.
Interventions
DRUGApraglutide
Apraglutide is a synthetic peptide analogue of GLP-2 under development for treatment of SBS-IF, which acts as a full agonist at the GLP-2 receptor with in vitro potency and selectivity comparable with native GLP-2
Sponsors
VectivBio AG
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Males and females with a diagnosis of SBS-IF secondary to surgical resection of the small intestine, with Colon-in-Continuity (CIC) or stoma, who were trial subjects of parent trials TA799-007 or TA799-013 2. Able to give informed consent and agree to follow the details of participation as outlined in the protocol.
Exclusion criteria
1. Subject not capable of understanding or not willing to adhere to the trial visit schedules and other protocol requirements. 2. Any other reason judged not eligible by the Investigator. 3. Pregnancy or lactation
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Adverse events (AE) | From baseline to week 208 | System organ class, frequency and severity |
| Clinical chemistry | From baseline to week 208 | Clinical Chemistry panel of analytes will be examined for clinically significant changes. |
| Hematology | From baseline to week 208 | Hematology panel of analytes will be examined for clinically significant changes. |
| Hemostasis | From baseline to week 104 | Hemostasis INR will be examined for clinically significant changes. |
| Urinalysis | From baseline to week 208 | Urinanalysis panel of analytes will be examined for clinically significant changes. |
| Occurrence of clinically relevant changes in vital signs | From baseline to week 208 | * Systolic and diastolic blood pressure in mmHg will be examined for clinically significant changes. * Heart rate in Beats per Minute (BPM) will be examined for clinically significant changes. |
| Occurrence of clinically relevant changes in electrocardiogram | From baseline to week 208 | ECG; intervals and rhythm |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline on the Pittsburgh Sleep Quality Inventory (PSQI) | From baseline to week 208 | — |
| Change from baseline on the Patient Global Impression of Change (PGIC) | From baseline to week 208 | — |
| Changes from baseline on Patient Global Impression of Treatment Satisfaction (PGI-TS) | From baseline to week 208 | — |
| Change from baseline in PS volume | From baseline to week 208 | — |
| Changes from baseline on Patient Global Impression of Parenteral Support Impact (PGI-PSI) | From baseline to week 208 | — |
| Change from baseline on the Short Form (36) Health Survey (SF-36) | From baseline to week 208 | — |
| Change from baseline on the EuroQoL-5 dimension -5 level survey (EQ-5D-5L) | From baseline to week 208 | — |
| Changes from baseline on Patient Global Impression of Satisfaction with Parenteral Support (PGI-SPS) | From baseline to week 208 | — |
| Change from baseline in PS frequency | From baseline to week 208 | Change in PS days/week from baseline to week 104 |
| Clinically significant changes in PS total energy | From baseline to week 208 | Units: kcal |
| Change from baseline in PS infusion time | From baseline to week 208 | — |
| Percentage of subjects reaching enteral autonomy | From baseline to week 104 | — |
| Change from baseline in body weight | From baseline to week 104 | Units: kg |
| Change from baseline on the Patient Global Impression of Severity (PGIS) | From baseline to week 104 | — |
Countries
Argentina, Belgium, Czechia, Denmark, France, Germany, Hungary, Israel, Italy, Japan, Norway, Poland, South Korea, Spain, Sweden, Taiwan, United Kingdom, United States
Outcome results
None listed