Pediatric Teduglutide Registry

NCT04832087 | Completed FDA Drug

• 1 other identifier: P00033923
• Study Type: observational
• Target: 142
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Short Bowel Syndrome

Outcome Measures

Primary Outcomes (1)

• Percent change in calories from parenteral support (PS) (Kcal/kg/day)

  Maximum percent change in calories (energy intake in kcal/kg/day) from parenteral support relative to pre- treatment (baseline) assessment up to 6 months, 1 year, 2 years and 3 years from start of therapy among Cohort A.

  Up to 3 years

Secondary Outcomes (7)

• Percent change of (PS) volume (liters/week)

  Up to 3 years

• Percent change of PS infusions (number of days/week )

  Up to 3 years

• Proportion of subjects completely weaned from PS

  Up to 3 years

• Adverse Events

  Up to 3 years

• Weight-for-age Z-score (WAZ)

  Up to 3 years

Study Arms (2)

Cohort A

Participants with SBS who started Teduglutide after FDA approval (May 2019)

Drug: Teduglutide

Cohort B

Subjects who participated in any of the teduglutide pediatric clinical trial studies (TED-C13-003, TED-C14-006, SHP633-303, or SHP633-304) and subsequently started therapy with teduglutide

Drug: Teduglutide

Interventions

Teduglutide DRUG

Standard of care Teduglutide dosing

Also known as: Gattex

Cohort A; Cohort B

Eligibility Criteria

• Age: 1 Year+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Children with SBS on Teduglutide

You may qualify if:

• Diagnosis of SBS defined as intestinal resection for congenital or acquired intestinal disease leading to dependence on PS for 90 days
• Received teduglutide after FDA approval
• Age ≥ 1 years and ≤ 18 years at start of teduglutide initiation
• Weight ≥ 10 kg at start of teduglutide initiation
• Dependent on PS at the time of teduglutide initiation
• Subject data for primary outcome (PS energy intake in cal/kg/day) is available for medical chart abstraction at baseline (pretreatment) visit.
• 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.

You may not qualify if:

• Participation in the pediatric clinical trial studies of teduglutide (TED-C13-003, TED-C14-006 or SHP633-303, SHP633-304).
• In the opinion of the investigator, the subject or guardian is at high risk for non-compliance.
• Cohort B:
• Diagnosis of SBS defined as intestinal resection for congenital or acquired intestinal disease leading to dependence on PS for 90 days
• Participated in clinical trial study (TED-C13-003, TED-C14-006) and/or extension study (SHP633-303, SHP633-304)
• Currently receiving teduglutide
• 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.
• \. In the opinion of the investigator, the subject or guardian is at high risk for non-compliance.

Sponsors & Collaborators

• Boston Children's Hospital: lead
• Takeda: collaborator

Study Sites (1)

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Related Publications (2)

• 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.

  PMID: 31495952 RESULT

• 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.

  PMID: 27855998 RESULT

MeSH Terms

Conditions

Short Bowel Syndrome

Interventions

teduglutide; ALX-0600

Condition Hierarchy (Ancestors)

Malabsorption Syndromes; Intestinal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Postoperative Complications; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Lissette Jimenez

  Boston Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 3 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Co-Director Congenital Enteropathy Program; Attending Physician, Division of Gastroenterology, Hepatology and Nutrition

Study Record Dates

• First Submitted: March 29, 2021
• First Posted: April 5, 2021
• Study Start: May 18, 2021
• Primary Completion: August 31, 2023
• Study Completion: August 31, 2023
• Last Updated: December 11, 2023

Record last verified: 2023-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)