Short Bowel Syndrome
Conditions
Brief summary
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
Interventions
DRUGNTRA-9620
Oral daily dose
DRUGPlacebo
Oral daily dose
Sponsors
Elgan Pharma Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
2 Weeks to 52 Weeks
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change in %PN/IV | baseline and end of treatment or 24 weeks, whichever occurs first | Percent change in %PN/IV from baseline based on caloric intake |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| NTRA-9620-A NTRA-9620 Dose 1 To be dosed orally for 24 weeks, 4 times/day
NTRA-9620: Oral daily dose | 1 |
| NTRA-9620-B NTRA-9620 Dose 2 To be dosed orally for 24 weeks, 4 times/day
NTRA-9620: Oral daily dose | 1 |
| Placebo Placebo To be dosed orally for 24 weeks, 4 times/day
Placebo: Oral daily dose | 0 |
| Total | 2 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Adverse Event | 1 | 0 | 0 |
Baseline characteristics
| Characteristic | NTRA-9620-A | NTRA-9620-B | Total | Placebo |
|---|---|---|---|---|
| Age, Categorical <=18 years | 1 Participants | 1 Participants | 2 Participants | — |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants | — |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants | — |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | — |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants | — |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | — |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | — |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | — |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 1 Participants | 1 Participants | — |
| Race (NIH/OMB) White | 1 Participants | 0 Participants | 1 Participants | — |
| Region of Enrollment United States | 1 participants | 1 participants | 2 participants | — |
| Sex: Female, Male Female | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Male | 1 Participants | 1 Participants | 2 Participants | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 1 | 0 / 1 | 0 / 0 |
| other Total, other adverse events | 0 / 1 | 0 / 1 | 0 / 0 |
| serious Total, serious adverse events | 1 / 1 | 0 / 1 | 0 / 0 |
Outcome results
Primary
Percent Change in %PN/IV
Percent change in %PN/IV from baseline based on caloric intake
Time frame: baseline and end of treatment or 24 weeks, whichever occurs first
Population: During the course of the study only two (2) subjects were enrolled prior to discontinuation. Thus, no conclusions regarding the efficacy or safety (risk/benefit) of NTRA-9620 in the short bowel syndrome (SBS) population can be determined from this study.