DRUG
Teduglutide
Daily sub cutaneous injection 0,05 mg/kg/day
Status
Completed
Phase
Phase 4
Enrollment
25
Locations
1
Results
Not posted
Publications
1
Study summary
The purpose of this study is to evaluate if the treatment could maximize intestinal absorption, minimize the inconvenience of diarrhea, and avoid, reduce or eliminate the need for parenteral support (PS) to achieve normal growth, to avoid parenteral nutrition complications and to achieve the best possible quality of life for the patient
The short bowel syndrome (SBS) may be defined as a severe malabsorption caused by reduction of intestinal absorptive surface following massive resection of the small intestine. Teduglutide (Revestive®) is an analog of glucagon-like peptide 2 (GLP-2), a naturally occurring hormone that regulates the functional and structural integrity of the cells lining the gastrointestinal tract. The aim of the treatment is to maximize intestinal absorption, minimize the inconvenience of diarrhea, and avoid, reduce or eliminate the need for parenteral support (PS) to achieve the best possible quality of life for the patient. The rationale for the use of Revestive® is based on data obtained, especially in the trial in SBS patients. Treatment with 0.05 mg/kg/day was safe and well tolerated (no recorded side effects). Patients remained stable despite substantial reduction in parenteral nutrition (PN) supply as evidenced by stable body weight and height, serum electrolytes, pancreatic enzymes and renal function tests. Treatment was associated with: * Reduced PN volume and calories delivered by 25 and 45% respectively with 20% of patients weaned off PN during the study period * Increased Enteral Nutrition (EN) supply in volume and calories by 40 and 62% respectively * Increased in plasma citrulline during the treatment period, but decreased after Teduglutide discontinuation The recommended dose of Revestive® in children and adolescents (aged 1 to 17 years) is the same as for adults (0.05 mg/kg body weight once daily).
Interventions
DRUG
Daily sub cutaneous injection 0,05 mg/kg/day
Timeline
First posted
Jun 19, 2018
Study start
Jul 2, 2018
Primary completion
Dec 11, 2019
Study completion
Jul 13, 2020
Results posted
Not reported
Registry updated
Sep 11, 2025
Outcomes
Decrease in parenteral nutrition: Parenteral Nutrition/Resting Energy Expenditure (PN/REE)
Time frame · At week 24
Evaluate the efficacy of Revestive® treatment
Ostomy output defined as stool balance testing, urine output and plasma citrulline
Time frame · up to week 48
Evaluate the impact of Revestive on ostomy flow
Change in days per week of Parenteral Nutrition (PN)
Time frame · up to week 48
Quantify the impact of Revestive on the number of perfusion in a week
Change in number of stool per day
Time frame · up to week 48
to evaluate the impact of Revestive on diarrhea
Change in stools consistency (Bristol stool chart)
Time frame · up to week 48
to evaluate the impact of Revestive on diarrhea
Ingesta (calorimetric measure)
Time frame · Every 4 weeks up to week 48
to evaluate the impact of Revestive on Intestinal absorption
Stool weight/24h
Time frame · Every 4 weeks up to week 48
to evaluate the impact of Revestive on Intestinal absorption
Percentage of lipid in stool
Time frame · Every 4 weeks up to week 48
to evaluate the impact of Revestive on Intestinal absorption
Percentage of nitrogen in stool
Time frame · Every 4 weeks up to week 48
to evaluate the impact of Revestive on Intestinal absorption
Percentage of carbohydrate in stool
Time frame · Every 4 weeks up to week 48
to evaluate the impact of Revestive on Intestinal absorption
Percentage of sodium in stool
Time frame · Every 4 weeks up to week 48
to evaluate the impact of Revestive on Intestinal absorption
Eligibility
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 having less than 120 cm in case of Short Bowel Syndrome (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 years and enterally fed (oral or tube feeding) ; * Having a normal colonoscopy in the 12 months before screening for children with maintained colon (=SBS type 2 or 3) older than 12 years ; * Having signed the Informed consent form (or parents or legal representative for minor patients). 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 a 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… * Hypersensitivity to the active substance or to any of the excipients listed in section 6.1 of Summary of Product Characteristics (SPC), or trace residues of tetracycline. * Active or suspected malignancy. * Patients with a history of malignancies in the gastrointestinal tract including the hepatobiliary system within the last five years.
Study locations
France. Showing up to 24 locations stored in the fast local snapshot.
Hôpital Necker - Enfants malades
Paris, France
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