NCT01573286

Brief Summary

This protocol outlines a randomized,open label trial examining the safety, pharmacology and efficacy of Glucagon like peptide 2 (GLP-2) in infants and children with intestinal failure. The investigators hypothesize that GLP-2 given subcutaneously in these patients will be well tolerated, and have similar metabolism to what has been shown in adults. The investigators also expect to show an improvement in the tolerance of enteral nutrition, and a decreased requirement for intravenous feeding.

Trial Health

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Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
13

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jan 2012

Typical duration for phase_1

Geographic Reach
1 country

4 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2012

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

April 5, 2012

Completed
4 days until next milestone

First Posted

Study publicly available on registry

April 9, 2012

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2014

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2015

Completed
Last Updated

December 23, 2014

Status Verified

December 1, 2014

Enrollment Period

2.6 years

First QC Date

April 5, 2012

Last Update Submit

December 19, 2014

Conditions

Keywords

Infant Nutrition DisordersChildhood nutrition disordersmalnutritionpost surgical syndromesnecrotising enterocolitisintestinal atresiagastroschisis

Outcome Measures

Primary Outcomes (2)

  • Frequency of Adverse events

    During active drug administration, patients will be monitored daily for serious adverse events. Patients will also be monitored (daily, if inpatients, bi weekly if outpatients) for clinically significant changes in safety data, vital signs, physical examination,and injection site reactions. Laboratory values of liver function and renal function will be monitored weekly for inpatients and bi weekly for outpatients. Following discontinuation of the treatment, patients will be monitored at 1 ,6 and 12 months post completion of the therapy.

    One year

  • Pharmacokinetics (Peak serum level. Area under the curve

    On days 3 and 42 of the trial, GLP-2 levels will be drawn at time 0 (before injection), 45,90 and 180 minutes post injection. Results will be analyzed for peak levels, and AUC.

    Done on Day 3 and 42

Secondary Outcomes (6)

  • Changes in the Enteral Caloric intake

    one year

  • Nutritional Parameters

    one year

  • Mucosal Morphology

    6 weeks

  • Intrinsic GLP-2 Production

    One year

  • Septic Episodes

    6 weeks

  • +1 more secondary outcomes

Study Arms (2)

Intestinal Failure in children (>1 year)

EXPERIMENTAL

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

Drug: Glucagon-Like Peptide 2

GLP-2 in Infants (<1 year of age)

EXPERIMENTAL

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.

Drug: Glucagon like peptide-2

Interventions

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.

Also known as: Glucagon-like peptide-2 (Lot: IC-115) Mfg. University of Calgary
Intestinal Failure in children (>1 year)

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.

Also known as: Glucagon-like peptide-2 (Lot: IC-115) Mfg. University of Calgary
GLP-2 in Infants (<1 year of age)

Eligibility Criteria

AgeUp to 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Infants (\< 1 year corrected gestational age) Infants 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) will be eligible for treatment in the immediate post-operative period.
  • 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.

You may not qualify if:

  • Significant extra-intestinal disease (e.g., grade IV intraventricular hemorrhage, severe hypoxic encephalopathy);
  • 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.
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Alberta Children's Hospital

Calgary, Alberta, T3B 6A8, Canada

Location

Stollery Children's Hospital

Edmonton, Alberta, T6G 2C8, Canada

Location

British Columbia Children's Hospital

Vancouver, British Columbia, V6H 3V4, Canada

Location

Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

Related Publications (2)

  • 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.

    PMID: 24368164BACKGROUND
  • 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

    RESULT

MeSH Terms

Conditions

Intestinal FailureShort Bowel SyndromeInfant Nutrition DisordersMalnutritionEnterocolitis, NecrotizingIntestinal AtresiaGastroschisis

Interventions

Glucagon-Like Peptide 2

Condition Hierarchy (Ancestors)

Intestinal DiseasesGastrointestinal DiseasesDigestive System DiseasesMalabsorption SyndromesPostoperative ComplicationsPathologic ProcessesPathological Conditions, Signs and SymptomsNutrition DisordersNutritional and Metabolic DiseasesEnterocolitisGastroenteritisDigestive System AbnormalitiesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMusculoskeletal AbnormalitiesMusculoskeletal DiseasesHernia, AbdominalHerniaPathological Conditions, Anatomical

Intervention Hierarchy (Ancestors)

Glucagon-Like PeptidesProglucagonGastrointestinal HormonesHormonesHormones, Hormone Substitutes, and Hormone Antagonists

Study Officials

  • David Sigalet, MD PhD

    Alberta Children's Hospital

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Adjunct Professor, Division of Pediatric Surgery

Study Record Dates

First Submitted

April 5, 2012

First Posted

April 9, 2012

Study Start

January 1, 2012

Primary Completion

August 1, 2014

Study Completion

August 1, 2015

Last Updated

December 23, 2014

Record last verified: 2014-12

Locations