Safety and Efficacy Study in Infant With SBS (GIFT)

Elgan Pharma

Status and phase: Terminated; Phase 3; Phase 2

Conditions: Short Bowel Syndrome

Treatments: Drug: NTRA-9620; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02865122

GIFT-02

Details and patient eligibility

About

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

Enrollment: 2 patients

Sex: All

Ages: 2 to 52 weeks old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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2 participants in 3 patient groups, including a placebo group

NTRA-9620-A

Experimental group

Description:

NTRA-9620 Dose 1 To be dosed orally for 24 weeks, 4 times/day

Treatment:

Drug: NTRA-9620

NTRA-9620-B

Experimental group

Description:

NTRA-9620 Dose 2 To be dosed orally for 24 weeks, 4 times/day

Treatment:

Drug: NTRA-9620

Placebo

Placebo Comparator group

Description:

Placebo To be dosed orally for 24 weeks, 4 times/day

Treatment:

Drug: Placebo