Safety and Efficacy Study in Infant With SBS (GIFT)

A Multi-Center, Randomized, Double-Blind, Three-Arm, Parallel-Group Trial to Assess the Efficacy and Safety of NTRA-9620 in Infants With Short Bowel Syndrome (SBS) Following Surgical Resection

The objective of this clinical study is to evaluate the efficacy and safety of NTRA-9620 compared with placebo in pediatric subjects (aged 28 weeks postmenstrual age to 52 weeks old) with SBS following surgical resection

Study Overview

• Status: Terminated
• Conditions: Short Bowel Syndrome
• Intervention / Treatment: Drug: NTRA-9620; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 2
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• United States
  • California
    • Palo Alto, California, United States, 94304
      • Lucile Packard Children's Hospital Stanford
  • Connecticut
    • Hartford, Connecticut, United States, 06106
      • Connecticut Children's Hospital
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • Children's Mercy Hospital
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital
    • Columbus, Ohio, United States, 43205
      • Nationwide Children's Hospital
  • Texas
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 weeks to 11 months (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria

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. 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 nutrient absorption.

Exclusion Criteria

1. Subject has undergone any bowel lengthening procedure.

2. Subject has a malabsorption disorder due to:

   • congenital etiology (such as microvilli inclusion disease, tufting enteropathy)
   • Untreated Hirchsprung's disease
3. 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 first 12 weeks of the study.
4. Subjects with hyperinsulinemia.
5. Subjects with unexplained or recurrent hypoglycemia with blood glucose ≤ 50 mg/dL within 48 hours of treatment initiation.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NTRA-9620-A; NTRA-9620 Dose 1 To be dosed orally for 24 weeks, 4 times/day	Drug: NTRA-9620; Oral daily dose
Experimental: NTRA-9620-B; NTRA-9620 Dose 2 To be dosed orally for 24 weeks, 4 times/day	Drug: NTRA-9620; Oral daily dose
Placebo Comparator: Placebo; Placebo To be dosed orally for 24 weeks, 4 times/day	Drug: Placebo; Oral daily dose

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent Change in %PN/IV	Percent change in %PN/IV from baseline based on caloric intake	baseline and end of treatment or 24 weeks, whichever occurs first

Collaborators and Investigators

• Sponsor: Elgan Pharma Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): March 20, 2017
• Primary Completion (Actual): March 22, 2018
• Study Completion (Actual): March 22, 2018

Study Registration Dates

• First Submitted: August 9, 2016
• First Submitted That Met QC Criteria: August 9, 2016
• First Posted (Estimate): August 12, 2016

Study Record Updates

• Last Update Posted (Actual): October 12, 2021
• Last Update Submitted That Met QC Criteria: October 11, 2021
• Last Verified: October 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Postoperative Complications; Disease; Gastrointestinal Diseases; Intestinal Diseases; Malabsorption Syndromes; Syndrome; Short Bowel Syndrome
• Other Study ID Numbers: GIFT-02

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No