Short Bowel Syndrome (SBS) MedDRA version: 20.1 Level: PT Classification code 10049416 Term: Short-bowel syndrome System Organ Class: 10017947 - Gastrointestinal disorders
Conditions
Interventions
Trade Name: Revestive
Product Code: A16AX08
Pharmaceutical Form: Powder and solution for solution for injection
INN or Proposed INN: Teduglutide
CAS Number: 287714-30-1
Current Sponsor code: SHP633 -
Sponsors
Shire Human Genetic Therapies, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Informed consent by the parent or legal guardian. 2. Male or female infant 4 to 12 months corrected gestational age at screening. 3. Weight at least 5 kg and weight-for-length Z-score greater than -2 at screening and baseline. 4. Short bowel syndrome with dependence on parenteral support to provide at least 50% of fluid or caloric needs. 5. Stable Parenteral Nutrition (PN) requirements for at least 1 month prior to screening, defined as a =10% change in the weight-normalized parenteral total fluid and caloric intake, despite attempts to wean PN, notwithstanding transient instability for events such as sepsis or interruption of central venous access. 6. This criteria was deleted. 7. Parent or legal guardian understands and is willing and able to fully adhere to study requirements as defined in this protocol. Are the trial subjects under 18? yes Number of subjects for this age range: 10 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Previous treatment with teduglutide. 2. Intestinal malabsorption due to a genetic condition, such as cystic fibrosis, microvillus inclusion disease, etc. 3. Severe, known dysmotility syndrome, such as pseudo-obstruction or persistent, severe, active gastroschisis-related dysmotility, that is the primary contributing factor to feeding intolerance and inability to reduce parenteral support, prior to screening. Dysmotility is defined as severe if it is expected to limit the advancement of enteral feeding. 4. Inability to advance oral or enteral feeding due to lack of access to the gut, such as oral aversion in the absence of a feeding tube. 5. Intestinal obstruction or clinically significant intestinal stenosis. 6. Major gastrointestinal surgical intervention, such as serial transverse enteroplasty or major intestinal resection or anastomosis, within 3 months prior to screening or planned during the study period. 7. Unstable cardiac disease. 8. Renal dysfunction, defined as estimated glomerular filtration rate 1.5 not corrected with parenteral vitamin K b. Platelet count 4 mg/dL (>68 µmol/L) over a 2 week period. 13. More than 3 serious complications of intestinal failure (e.g., catheter-associated bloodstream infections, interruption of nutrition due to feeding intolerance, catheter-associated thrombosis, severe fluid or electrolyte disturbances) within 1 month prior to or during screening. 14. A history of cancer or a known cancer predisposition syndrome, such as juvenile polyposis or Beckwith-Wiedemann syndrome, or first degree relative with early onset of gastrointestinal cancer (including hepatobiliary and pancreatic cancers). 15. Concurrent treatment with glucagon-like peptide-1 (GLP-1); glucagon-like peptide-2 (GLP-2); insulin-like growth factor-1 (IGF-1); growth hormone, somatostatin, or analogs of these hormones; or glutamine. 16. Participation in a clinical study using an experimental drug within 3 months or 5.5 half-lives of the experimental drug, whichever is longer. 17. Known or suspected intolerance or hypersensitivity to the investigational product, closely-related compounds, or any of the stated ingredients. 18. Any condition, disease, illness, or circumstance that, in the investigator’s opinion, puts the subject at any undue risk, prevents completion of the study, or interferes with analysis of the study results.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: the objectives of this trial are to evaluate the safety, efficacy/pharmacodynamics and pharmacokinetics (PK) of teduglutide treatment in infants with short bowel syndrome (SBS) dependent on parenteral support.;Secondary Objective: not applicable;Primary end point(s): Reduction in weight-normalized PN fluid volume by at least 20% from baseline at Week 24/EOT (end of treatment);Timepoint(s) of evaluation of this end point: week 24/EoT | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: each visit and week 24/EoT;Secondary end point(s): - Reduction in weight-normalized parenteral calories by at least 20% from baseline to Week 24/EOT - Achievement of enteral autonomy by week 24 - Time to achieve enteral autonomy - Change in weight-normalized parenteral fluid volume from baseline to each visit - Change in weight-normalized parenteral calories from baseline to each visit - Change in weight-normalized enteral fluid volume from baseline to each visit - Change in weight-normalized enteral caloric intake from baseline to each visit - Increase in weight-normalized enteral fluid intake by at least 20% from baseline to Week 24/EOT - Increase in weight-normalized enteral caloric intake by at least 20% from baseline to Week 24/EOT Pharmacokinetics The pharmacokinetic endpoint is plasma teduglutide concentration at nominal time point. Safety Safety endpoints consist of the following: - Adverse events (AEs) - Physical examinations - Vital signs - Weight, length, head circumference, and weight-for-length Z-scores (corrected for gestational age) - Laboratory safety data (biochemistry and haematology) - Urine output - Stool (including mixed) output - Antibodies to teduglutide Health Economics and Outcomes Research Health economics and outcomes research (HEOR) endpoints include the following: - Cumulative number of hospitalization days during the study | — |
Countries
Finland, France, Italy, United Kingdom
Contacts
Public ContactShire Medical Monitor
Shire
Outcome results
None listed