Open-label Extension Trial to Evaluate the Long-term Safety of Apraglutide in Short Bowel Syndrome.

Study Overview
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Brief Summary
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The primary objective of the trial is to assess long-term safety and tolerability of apraglutide in subjects with SBS-IF.

Detailed Description
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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.

Official Title
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An Open-label Extension Trial to Evaluate the Long-term Safety of Apraglutide in Short Bowel Syndrome.

Conditions
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Short Bowel Syndrome

Intervention / Treatment
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* Drug: Apraglutide


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 Able to give informed consent and agree to follow the details of participation as outlined in the protocol. Exclusion Criteria: Subject not capable of understanding or not willing to adhere to the trial visit schedules and other protocol requirements. Any other reason judged not eligible by the Investigator. Pregnancy or lactation

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Apraglutide subcutaneous (SC) injections, once weekly<br>Peptide analogue of Glucagon-like Peptide 2 (GLP-2) | Drug: Apraglutide<br>* 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<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse events (AE) | System organ class, frequency and severity | From baseline to week 208 | 
| Clinical chemistry | Clinical Chemistry panel of analytes will be examined for clinically significant changes. | From baseline to week 208 | 
| Hematology | Hematology panel of analytes will be examined for clinically significant changes. | From baseline to week 208 | 
| Hemostasis | Hemostasis INR will be examined for clinically significant changes. | From baseline to week 104 | 
| Urinalysis | Urinanalysis panel of analytes will be examined for clinically significant changes. | From baseline to week 208 | 
| Occurrence of clinically relevant changes in vital signs | 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. | From baseline to week 208 | 
| Occurrence of clinically relevant changes in electrocardiogram | ECG; intervals and rhythm | From baseline to week 208 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in PS volume |  | From baseline to week 208 | 
| Change from baseline in PS frequency | Change in PS days/week from baseline to week 104 | From baseline to week 208 | 
| Clinically significant changes in PS total energy | Units: kcal | From baseline to week 208 | 
| 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 | Units: kg | From baseline to week 104 | 
| Change from baseline on the Patient Global Impression of Severity (PGIS) |  | From baseline to week 104 | 
| 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 | 
| Changes from baseline on Patient Global Impression of Satisfaction with Parenteral Support (PGI-SPS) |  | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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SBS, Short Bowel Syndrome, Intestinal Failure, SBS-IF