Safety and Efficacy Study in Infants With SBS

Phase 1

• Conditions: Short Bowel syndrome; MedDRA version: 19.1Level: LLTClassification code 10025479Term: Malabsorption syndromeSystem Organ Class: 10017947 - Gastrointestinal disorders; MedDRA version: 19.1Level: PTClassification code 10025476Term: MalabsorptionSystem Organ Class: 10017947 - Gastrointestinal disorders; MedDRA version: 19.1Level: SOCClassification code 10017947Term: Gastrointestinal disordersSystem Organ Class: 10017947 - Gastrointestinal disorders; Therapeutic area: Body processes [G] - Digestive System and Oral Physiological Phenomena [G10]

• Registration Number: EUCTR2015-000181-60-NL
• Lead Sponsor: utrinia Ltd.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-02-14
• Last Update Posted: 24 July 2017

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 150

Inclusion Criteria

Subjects must meet all of the following criteria to be included:
1)Subject must be at least 28 weeks post-menstrual age and up to 52 weeks chronological age at enrollment.
2)Subject weight must be at least 500 grams (17.6 ounces) at time of enrollment.
3)Clinically diagnosed with SBS requiring PN/IV secondary to surgical resection of the intestine.
4)After major surgical resection leading to SBS, the subject has maximally 70% of expected bowel length preserved or an ostomy in place such that = 70% of the small bowel is available for absorption .
5)Subject can tolerate at least 10 mL/kg/day of enteral nutrition (EN) for at least 7 days at time of enrollment.
6)At time of enrollment subject is on at least 70% of prescribed PN/IV and no more than 30% of EN based on total caloric intake for at least 7 days prior to study entry.
7)Subject is randomized into the trial within 4 months from the surgical resection that has led to the diagnosis of SBS.
8)Subject’s parent(s) or legal guardian(s) provide written informed consent.
9)Subject’s parent(s) or legal guardian(s) understand and are willing to comply with all study procedures and requirements.

Are the trial subjects under 18? yes
Number of subjects for this age range: 150
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 0
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

Subjects meeting any of the following criteria at study entry will be excluded:
1)Subject has undergone any bowel lengthening procedure.
2)Subject has a malabsorption disorder such as:
•Congenital etiology (such as microvilli inclusion disease, tufting enteropathy)
•Untreated Hirchsprung’s disease
3)Significant motility disorder such as:
•Pseudo obstruction
•Severe gastroschisis defined as: primary reason for PN support is due to persistent feeding intolerance of less than 20 ml/kg/day EN intake or signs and symptoms (i.e., abdominal distention, vomiting) requiring prokinetic agents.
4)Any known inherited abnormality (e.g., Fanconi syndrome,) that is not related to SBS.
5)Prior bowel resection due to isolated spontaneous intestinal perforation.
6)< 10 cm of remaining small bowel left with no colon.
7)Uncontrolled systemic infection, acute gastroenteritis, pneumonia, cardiovascular or other abnormality including EKG findings that in the opinion of the investigator makes the infant unstable and at significant risk of not completing the first 12 weeks of the study.
8)Subjects with known hyperinsulinemia .
9)Subjects with unexplained or recurrent hypoglycemia with blood glucose = 50 mg/dL within 48 hours of treatment initiation.
10)Subjects with severe anemia of Hemoglobin less than 60 g/L and requiring transfusion within 48 hours of treatment initiation to avoid a life threatening event.
11)Subjects who require pancreatic enzyme replacement therapy.
12)Subject is currently receiving oral or injectable insulin for any reason.
13)Participation in another interventional clinical study within the past 30 days that may interfere with the results of this study.
14)History or current use of growth factors or glutamine.
15)In the opinion of the investigator, the subject has any other medical condition that would make participation in this study either unsafe or would compromise the potential benefit of insulin treatment

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective is to evaluate the efficacy and safety of NTRA-9620 compared with placebo when added to standard of care (SOC) in pediatric subjects (aged 28 weeks post-menstrual age to 52 weeks chronological age) with SBS within 4 months from surgical resection who are on parenteral nutrition (PN) support.;Secondary Objective: N/A;Primary end point(s): The primary endpoints are the percent changes in %PN (PC_PN0-t) from baseline based on caloric intake to 12 weeks and again to 24 weeks. <br><br>;Timepoint(s) of evaluation of this end point: 12 and 24 weeks treatment