NCT03562130

Characterization of the Long-term Safety, Efficacy, and Pharmacodynamics Revestive® in the Management of Short Bowel Syndrome Pediatric Patients

A Monocentric Single-arm Study to Characterize the Long-term Safety, Efficacy, and Pharmacodynamic of GLP-2 Analog (Revestive®) in the Management of Short Bowel Syndrome Pediatric Patients on Home-parenteral Nutrition (HPN)

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 25 (actual)
• Sponsor: Assistance Publique - Hôpitaux de Paris · Academic / Other
• Sex: All
• Age: 2 Years – 18 Years
• Healthy volunteers: Not accepted

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

Detailed description

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).

Conditions

• Short Bowel Syndrome

Interventions

Type	Name	Description
DRUG	Teduglutide	Daily sub cutaneous injection 0,05 mg/kg/day

Timeline

• Start date: 2018-07-02
• Primary completion: 2019-12-11
• Completion: 2020-07-13
• First posted: 2018-06-19
• Last updated: 2025-09-11

Locations

1 site across 1 country: France