Safety and Dosing Study of Glucagon-like Peptide 2 (GLP-2) in Infants and Children With Intestinal Failure
NCT ID: NCT01573286
Last Updated: 2014-12-23
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
PHASE1/PHASE2
13 participants
INTERVENTIONAL
2012-01-31
2015-08-31
Brief Summary
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Detailed Description
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This proposal outlines a Phase 1 and 2 trial using subcutaneous administration, twice daily of GLP-2 in human infants and children with Intestinal Failure, typically from Short Bowel Syndrome, using varying doses, assigned in a prospective, randomized protocol, with open label monitoring.
The investigational plan is to begin with the Phase 1 trial, administering GLP 2 at varying doses (infants assigned to doses of 5,10, or 20 μg/kg/day, children greater than 1 year dosed at 20 μg/kg/day, given via twice daily subcutaneous injection).
Eligible subjects will be infants (less than 12 months corrected gestational age) with either major resection (remaining small intestine less than 40% of predicted length for gestational age), or demonstrated intestinal failure after intestinal resection/abdominal surgery/gastroschisis (Requirement for parenteral nutrition greater than 50% of total calories, more than 45 days after the last surgery).
Infants will be allocated sequentially to a group (n = 6 per group) treated with GLP-2 at 5,10, or 20 μg/kg/day.
Older children (greater than 1 year of age), requiring PN for \>30% calories\> one year post surgery will also be eligible; these patients will be dosed at 20 μg/kg/day (via twice daily subcutaneous injection) n= 7.
Patients will be followed on the principle of intention to treat after initial randomization. The endpoints will be monitoring for safety, and recording of adverse events and a pharmacokinetic profile at 3 days.
If the hormone is well tolerated these studies will be extended into a phase 2 study, for an additional 39 days; monitoring safety, patient tolerance of enteral nutrition, growth, citrulline levels, nutrient absorptive capacity, liver function and repeat pharmacokinetic studies.
Conditions
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Keywords
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Intestinal Failure in children (>1 year)
Children requiring parenteral nutrition for \>30% of calories more than 1 year (365) days post surgery will be eligible for treatment with Glucagon-like peptide 2 (20 ug/kg/day) for 6 weeks
Glucagon-Like Peptide 2
Patients will be treated with 20 ug/kg/day GLP-2, in two doses, given subcutaneously for 3 days (Phase 1). If the treatment is well tolerated, GLP-2 will be continued for a total of 42 days.
GLP-2 in Infants (<1 year of age)
Infants under one year of age with congenital anomalies, or intestinal resection, leaving them with anatomic short bowel syndrome (total remaining small intestine less than 40 % of predicted for gestational age) or with intestinal resection or repaired gastroschisis who have demonstrated dependence on parenteral nutrition at 45 days post operation with the requirement for \>50% of calories by PN (independent of the length of remnant small intestine) will be eligible for treatment with Glucagon-like peptide 2, at a dose of 5, 10 or 20 ug/kg/day.
Glucagon like peptide-2
Patients will treated with 5, 10 or 20 ug/kg/day of GLP-2, given twice daily by subcutaneous injection. The initial cohort of patients will be treated at 5 ug/kg (n=6), and if this dose is seen to be safe, and levels appropriate, the next group of 6 will be treated at 10 ug/kg/day. If this dose is seen to be safe, and levels appropriate, the final group of 6 will be treated at 20 ug/kg/day.
Patients will be given GLP-2 at the assigned dose, subcutaneously for 3 days (Phase 1). If the treatment is well tolerated, GLP-2 will be continued, at the same dose, for a total of 42 days.
Interventions
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Glucagon-Like Peptide 2
Patients will be treated with 20 ug/kg/day GLP-2, in two doses, given subcutaneously for 3 days (Phase 1). If the treatment is well tolerated, GLP-2 will be continued for a total of 42 days.
Glucagon like peptide-2
Patients will treated with 5, 10 or 20 ug/kg/day of GLP-2, given twice daily by subcutaneous injection. The initial cohort of patients will be treated at 5 ug/kg (n=6), and if this dose is seen to be safe, and levels appropriate, the next group of 6 will be treated at 10 ug/kg/day. If this dose is seen to be safe, and levels appropriate, the final group of 6 will be treated at 20 ug/kg/day.
Patients will be given GLP-2 at the assigned dose, subcutaneously for 3 days (Phase 1). If the treatment is well tolerated, GLP-2 will be continued, at the same dose, for a total of 42 days.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Infants with intestinal resection or repaired gastroschisis who have demonstrated dependence on parenteral nutrition at 45 days post operation with the requirement for \>50% of calories by PN (independent of the length of remnant small intestine).
* Children (\> 1 year corrected gestational age) Children with a requirement for \>30% of calories by PN more than 1 year (365) days post surgery will be eligible.
Exclusion Criteria
* Significant cardiovascular, hemodynamic or respiratory instability, as noted by 1) the requirement for dopamine \> 4 mcg/kg/min, 2) high frequency ventilatory support, 3) extracorporeal membrane oxygenation.
* Hepatic disease defined as direct bilirubin \> 100 umol/L (5.2 mg/dL)
* Renal disease defined as BUN \> 80 or creatinine \> 90 μmol/L (1.5 mg/dL)
* Inborn errors of metabolism necessitating protein restriction or other special diet;
* Ongoing sepsis syndrome, as noted by refractory hypotension, thrombocytopenia, acidosis, and/or bacteremia.
* Primary motility defect such as intestinal pseudo-obstruction.
* Absorptive defects (such as microvillus inclusion disease)
* Females who are post-pubertal must agree to comply with measures to prevent pregnancy during the study phase.
* Coagulopathy which precludes the use of subcutaneous injections.
* Allergy to GLP-2 or any of the constituent of the GLP-2 IC-115 preparation.
18 Years
ALL
No
Sponsors
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Stollery Children's Hospital
OTHER
The Hospital for Sick Children
OTHER
British Columbia Children's Hospital
OTHER
Alberta Children's Hospital
OTHER
Responsible Party
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David Sigalet MD PhD
Adjunct Professor, Division of Pediatric Surgery
Principal Investigators
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David Sigalet, MD PhD
Role: STUDY_DIRECTOR
Alberta Children's Hospital
Locations
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Alberta Children's Hospital
Calgary, Alberta, Canada
Stollery Children's Hospital
Edmonton, Alberta, Canada
British Columbia Children's Hospital
Vancouver, British Columbia, Canada
Hospital for Sick Children
Toronto, Ontario, Canada
Countries
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References
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Sigalet DL, de Heuvel E, Wallace L, Bulloch E, Turner J, Wales PW, Nation P, Wizzard PR, Hartmann B, Assad M, Holst JJ. Effects of chronic glucagon-like peptide-2 therapy during weaning in neonatal pigs. Regul Pept. 2014 Jan 10;188:70-80. doi: 10.1016/j.regpep.2013.12.006. Epub 2013 Dec 22.
Sigalet DL, Lam V, Karnik V, Brindle M, Boctor D, Casey LW, Dicken B, Butterworth S, Stoffman S,de Heuval E, Wright-wilson G, Wallace L. Safety and Dosing Study of Glucagon-like Peptide 2 (GLP-2) in Children With Intestinal Failure DDW Abstract presented at DDW 2014, Chicago IL
Other Identifiers
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150979
Identifier Type: OTHER
Identifier Source: secondary_id
GLP-2-01
Identifier Type: -
Identifier Source: org_study_id