Monocentric Single-arm study to characterize the long-term safety, efficacy, and pharmacodynamic of GLP-2 analog in the management of short bowel syndrome pediatric patients on home-parenteral nutritio

Phase 1

• Conditions: Short Bowel Syndrom; MedDRA version: 20.0Level: PTClassification code 10049416Term: Short-bowel syndromeSystem Organ Class: 10017947 - Gastrointestinal disorders; Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

• Registration Number: EUCTR2017-001405-32-FR
• Lead Sponsor: Institut des Maladies Génétiques - Imagine

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2017-09-19
• Last Update Posted: 17 March 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 25

Inclusion Criteria

- Being aged from 2 to 18 years old included ;
- Presenting less than 80 cm of residual small intestine with or without the terminal ileum, ileocecal valve and right colon or left with less than 120 cm in case of SBS caused by Hirschsprung disease;
- Being stable on PN support (inability to significantly reduce PN intake for the last six months before inclusion) ;
- Being dependent on PN for at least 2 year and enterally fed (oral or tube feeding) ;
- Having a normal colonoscopy in the 12 month before screening (for children with a SBS type 2 or 3 older than 12 years) ;

Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 5
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- Having a major gastrointestinal surgical intervention like serial transverse enteroplasty or any other bowel lengthening procedure performed within 6 months of screening ;
- Having a clinically significant untreated intestinal obstruction or active stenosis ;
- Having an unstable absorption due to cystic fibrosis or known DNA abnormalities ;
- Presenting a radiographic or manometric evidence of pseudo-obstruction or severe known dysmotility syndrome, including persistent, severe gastroschisis-related motility disorders ;
- Having an unstable cardiac disease, congenital heart disease or cyanotic disease, with the exception of patients who had undergone ventricular or atrial septal defect repair ;
- Having an history of cancer or clinically significant lymphoproliferative disease; excepted resected cutaneous basal or squamous cell carcinoma, or in situ non-aggressive and surgically resected cancer ;
- Having participated in a clinical study using an experimental drug within 1 month or an experimental antibody treatment within 3 months prior to screening, or concurrent participation in any clinical study using an experimental drug that would affect the safety of teduglutide ;
- Having already used native GLP-2 and glucagon-like peptide-1 analog or human growth hormone within 3 months prior to screening ;
- Having already used oral or IV glutamine, octreotide, or dipeptidyl peptidase IV (DPP-IV) inhibitors within 3 months prior to screening ;
- Having an active Crohn’s disease which has been treated with biological therapy within the 6 months prior to screening ;
- Having an intestinal polyposis;
- Being, for female patient, both lactating and breast-feeding or having a positive pregnancy test during the screening period;
- Refusing the follow the protocol requirements in terms of birth control ;
- Being unable to follow the study procedures for any reason: psychological, geographical…

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: - To evaluate the efficacy of Revestive® to decrease PN using PN/REE ratio in pediatric patients (aged 1 year through 18 years) with short bowel syndrome (SBS) who are dependent on parenteral support.;Secondary Objective: - To evaluate the impact of Revestive® on ostomy flow <br>- To quantify the impact of Revestive® on the number of perfusions in a week <br>- To evaluate the impact of Revestive® on diarrhea<br>- To evaluate the impact of Revestive® on intestinal absorption using stool balance<br>- To evaluate the long term safety of Revestive® <br>- To evaluate the response rate to Revestive® <br> <br><br>;Primary end point(s): - Proportion of patients who achieve a = 20% reduction of PN/REE from baseline to week 24. ;Timepoint(s) of evaluation of this end point: 48 weeks<br>