Short Bowel Syndrom MedDRA version: 20.0 Level: PT Classification code 10049416 Term: Short-bowel syndrome System Organ Class: 10017947 - Gastrointestinal disorders
Conditions
Interventions
Trade Name: Revestive
Product Name: teduglutide
Pharmaceutical Form: Powder and solvent for solution for injection
Sponsors
Institut des Maladies Génétiques - Imagine
Eligibility
Sex/Gender
All
Inclusion criteria
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
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…
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 - To quantify the impact of Revestive® on the number of perfusions in a week - To evaluate the impact of Revestive® on diarrhea - To evaluate the impact of Revestive® on intestinal absorption using stool balance - To evaluate the long term safety of Revestive® - To evaluate the response rate to Revestive® ;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 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): To evaluate the impact of Revestive® on ostomy flow Ostomy flow will be assessed only for patient with a SBS type 1 by evaluations of: - Ostomy output/stool balance testing - Urine output (only during hospitalizations) from baseline to EOT - Changes in plasma citrulline To quantify the impact of Revestive® on the number of perfusions in a week - Change in days per week of PN as well as any patients who are able to completely wean off PN support - Increase in enteral nutritional tolerance (calories and volume) - Decrease in parenteral support (calories and volume) To evaluate the impact of Revestive® on diarrhea Diarrhea will be assessed only for patient with a SBS type 2 or 3 by evaluation of: - Changes in number of stools per day - Changes in stools consistency To evaluate the impact of Revestive® of intestinal absorption using stool balance Stool balance will be assessed by evaluations of: - Ingesta (calorimetric measure) - Stool weight/24h (reported to the patient’s weight) - Percentage of lipid absorption - Percentage of nitrogen absorption - Percentage of carbohydrate absorption - Percentage of total caloric absorption - Sodium in stool To evaluate the long term Safety of Revestive® Safety and tolerability will be assessed by evaluations of: - Adverse events, including GI symptoms - Physical examinations - Vital signs, including body weight, heart rate, and blood pressure - Electrocardiograms - Laboratory safety data, including electrolyte balance and glucose - Maintaining the growth; including weight, brachial perimeter, and length - Oral/enteral feeding tolerance - Nutritional intake and urinary/fecal output (intake/output) - Results of routine clinical evaluations including physical examination, and where appropriate, GI-specific testing including imaging (colonoscopy) abdominal ultrasound, fecal occult blood testing) To evaluate the response rate to Revestive® The response rate to Revestive® and the identificat | — |
Countries
France
Contacts
Public ContactElisabeth Hulier-Ammar
Institut des maladies Génétiques - Imagine
Outcome results
None listed