Clinical trial NCT04832087: Pediatric Teduglutide Registry
Official title: Pediatric Teduglutide Registry: Clinical Use of Teduglutide in Children With Intestinal Failure, a Multicenter Post-marketing Evaluation
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Short Bowel Syndrome
Lead sponsor: Boston Children's Hospital
Sponsor class: OTHER
Collaborators: name: Takeda
First posted: 2021-04-05
Enrollment: 142
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Children with SBS on Teduglutide

Brief summary: Pediatric specific post-marketing registry to evaluate the long-term safety and efficacy of teduglutide.

Detailed description: This will be a multi-center post-marketing pediatric registry study evaluating the efficacy and safety of teduglutide. This is an observational longitudinal registry of pediatric SBS patients who are using FDA approved teduglutide as per standard of care.

Eligibility criteria: Cohort A:

Inclusion Criteria:

1. Diagnosis of SBS defined as intestinal resection for congenital or acquired intestinal disease leading to dependence on PS for 90 days
2. Received teduglutide after FDA approval
3. Age ≥ 1 years and ≤ 18 years at start of teduglutide initiation
4. Weight ≥ 10 kg at start of teduglutide initiation
5. Dependent on PS at the time of teduglutide initiation
6. Subject data for primary outcome (PS energy intake in cal/kg/day) is available for medical chart abstraction at baseline (pretreatment) visit.
7. The subject or guardian signs and dates a written informed consent form and any required privacy authorization and minor provides any assent required prior to enrolment into the registry.

Exclusion Criteria:

1. Participation in the pediatric clinical trial studies of teduglutide (TED-C13-003, TED-C14-006 or SHP633-303, SHP633-304).
2. In the opinion of the investigator, the subject or guardian is at high risk for non-compliance.

Cohort B:

Inclusion Criteria:

1. Diagnosis of SBS defined as intestinal resection for congenital or acquired intestinal disease leading to dependence on PS for 90 days
2. Participated in clinical trial study (TED-C13-003, TED-C14-006) and/or extension study (SHP633-303, SHP633-304)
3. Currently receiving teduglutide
4. The subject or guardian signs and dates a written informed consent form and any required privacy authorization and minor provides any assent required prior to enrolment into the registry.

Exclusion Criteria:

1. In the opinion of the investigator, the subject or guardian is at high risk for non-compliance.

Primary outcomes:
measure: Percent change in calories from parenteral support (PS) (Kcal/kg/day)
timeFrame: Up to 3 years
Secondary outcomes:
measure: Percent change of (PS) volume (liters/week)
timeFrame: Up to 3 years
measure: Percent change of PS infusions (number of days/week )
timeFrame: Up to 3 years
measure: Proportion of subjects completely weaned from PS
timeFrame: Up to 3 years
measure: Adverse Events
timeFrame: Up to 3 years
measure: Weight-for-age Z-score (WAZ)
timeFrame: Up to 3 years
measure: Height-for-age-Z-score (HAZ)
timeFrame: Up to 3 years
measure: Body-Mass-Index Z-score (BMIZ)
timeFrame: Up to 3 years
Other outcomes:
Locations:
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
Study officials:
affiliation: Boston Children's Hospital
name: Lissette Jimenez
role: PRINCIPAL_INVESTIGATOR
References:
citation: Kocoshis SA, Merritt RJ, Hill S, Protheroe S, Carter BA, Horslen S, Hu S, Kaufman SS, Mercer DF, Pakarinen MP, Venick RS, Wales PW, Grimm AA. Safety and Efficacy of Teduglutide in Pediatric Patients With Intestinal Failure due to Short Bowel Syndrome: A 24-Week, Phase III Study. JPEN J Parenter Enteral Nutr. 2020 May;44(4):621-631. doi: 10.1002/jpen.1690. Epub 2019 Sep 8.
citation: Carter BA, Cohran VC, Cole CR, Corkins MR, Dimmitt RA, Duggan C, Hill S, Horslen S, Lim JD, Mercer DF, Merritt RJ, Nichol PF, Sigurdsson L, Teitelbaum DH, Thompson J, Vanderpool C, Vaughan JF, Li B, Youssef NN, Venick RS, Kocoshis SA. Outcomes from a 12-Week, Open-Label, Multicenter Clinical Trial of Teduglutide in Pediatric Short Bowel Syndrome. J Pediatr. 2017 Feb;181:102-111.e5. doi: 10.1016/j.jpeds.2016.10.027. Epub 2016 Nov 15.