Short Bowel SyndromePTRNCT04832087
Pediatric Teduglutide Registry
An observational study of Short Bowel Syndrome, run by Boston Children's Hospital at 1 site in United States.
Trial summary
Pediatric specific post-marketing registry to evaluate the long-term safety and efficacy of teduglutide. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Short Bowel Syndrome
- What is being tested?
- Teduglutide
- How many people
- 142 participants
- Where
- 1 siteUnited States
- Study dates
- 18 May 2021to 31 Aug 2023
- How long will I take part?
- 3 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Children with SBS on Teduglutide
Who may be able to join
- 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.
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- 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.
- 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.
Who cannot join
- 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:
- 1. In the opinion of the investigator, the subject or guardian is at high risk for non-compliance.
Trial locations and contacts
1 site in 1 country.
United States1 site
Boston Children's Hospital
Boston, Massachusetts, 02115
About this study
Official title: Pediatric Teduglutide Registry: Clinical Use of Teduglutide in Children With Intestinal Failure, a Multicenter Post-marketing Evaluation
Pediatric specific post-marketing registry to evaluate the long-term safety and efficacy of teduglutide.
Read the 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.
- Conditions
- Short Bowel Syndrome
- Being tested
- Teduglutide
- Medical subject headings
- Short Bowel Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TeduglutideDrug
Also called Gattex
Standard of care Teduglutide dosing
| Study group | Role | What they receive |
|---|---|---|
| Cohort A | Participants with SBS who started Teduglutide after FDA approval (May 2019) Receives: 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 Receives: Drug: Teduglutide |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 3 years
Secondary outcomes (7)
Percent change of (PS) volume (liters/week)
Percent change of PS volume (liters/week) relative to pre-treatment (baseline) assessment up to 6 months, 1 year, 2 years and 3 years from start of therapy among Cohort A
Measured: Up to 3 years
Percent change of PS infusions (number of days/week )
Percent change of PS infusions (number of days/week relative to pre-treatment (baseline) assessment up to 6 months, 1 year, 2 years and 3 years from start of therapy among Cohort A
Measured: Up to 3 years
Proportion of subjects completely weaned from PS
Number (proportion) of children who wean completely from PS within 6 months, 1 year, 2 years and 3 years from start of therapy among Cohort A.
Measured: Up to 3 years
Adverse Events
Number (proportion) of subjects with adverse events among Cohort A. Number (proportion) of subjects with adverse events among Cohort B.
Measured: Up to 3 years
Weight-for-age Z-score (WAZ)
Mean change in Weight-for-age Z-score (WAZ), relative to pre-treatment (baseline) assessment up to 6 months, 1 year, 2 years and 3 years from start of therapy among Cohort A.
Measured: Up to 3 years
Height-for-age-Z-score (HAZ)
Mean change in Height-for-age-Z-score (HAZ), relative to pre-treatment (baseline) assessment up to 6 months, 1 year, 2 years and 3 years from start of therapy among Cohort A.
Measured: Up to 3 years
Body-Mass-Index Z-score (BMIZ)
Mean change in Body-Mass-Index Z-score (BMIZ), relative to pre-treatment (baseline) assessment up to 6 months, 1 year, 2 years and 3 years from start of therapy among Cohort A.
Measured: Up to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2021
- Study start18 May 2021
- Primary completion31 Aug 2023
- Study completion31 Aug 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Short Bowel SyndromeStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 142 participants (actual)
- Target duration
- 3 Years
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Takeda
- Responsible party
- Principal investigator, Lissette Jimenez, Boston Children's Hospital
- Investigators
- Lissette Jimenez, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04832087
- Sponsor's protocol ID
- P00033923
- Acronym
- PTR
- Status last verified
- Dec 2023
- First submitted
- 29 Mar 2021
- Last updated
- 11 Dec 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 31495952Carter 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.
PubMed 27855998Jimenez L, Ray A, Mercer D, Kocoshis S, Kinberg S, Martinez M, Gumer L, Merritt R, Corkins MR, Vanderpool C, Cohran V, Wendel D, Thompson J, Sudan D, Van Buren KW, Kaufman S, Carey AN, Sabrdaran S, Mitchell P, Dahlberg SE, Weller E, Duggan CP. Safety and long-term effectiveness of teduglutide in children with intestinal failure due to short bowel syndrome: a multicenter, post-marketing cohort study. Pediatr Res. 2026 Sep 17. doi: 10.1038/s41390-026-05489-0. Online ahead of print.
PubMed 42754721
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pediatric Teduglutide Registry: Clinical Use of Teduglutide in Children With Intestinal Failure, a Multicenter Post-marketing Evaluation
- ClinicalTrials.gov ID
- NCT04832087
- Acronym / sponsor ID
- PTR / P00033923
- Conditions
- Short Bowel Syndrome
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 142 participants (actual)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Collaborators
- Takeda
- Study start
- 18 May 2021 (actual)
- Primary completion
- 31 Aug 2023 (actual)
- Study completion
- 31 Aug 2023 (actual)
- First posted
- 5 Apr 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 3 Years
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort A | – | Drug: TeduglutideParticipants with SBS who started Teduglutide after FDA approval (May 2019) |
| Cohort B | – | Drug: TeduglutideSubjects 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 |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Children with SBS on Teduglutide
Key inclusion criteria (11 listed)
- 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
Key exclusion criteria (4 listed)
- 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.
- 1. In the opinion of the investigator, the subject or guardian is at high risk for non-compliance.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TeduglutideAlso: GattexStandard of care Teduglutide dosing | Drug | Cohort A, Cohort B |
Primary outcome
Percent change in calories from parenteral support (PS) (Kcal/kg/day)
Time frame: Up to 3 years
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.
7 secondary outcomes
Percent change of (PS) volume (liters/week)
Time frame: Up to 3 years
Percent change of PS infusions (number of days/week )
Time frame: Up to 3 years
Proportion of subjects completely weaned from PS
Time frame: Up to 3 years
Adverse Events
Time frame: Up to 3 years
Weight-for-age Z-score (WAZ)
Time frame: Up to 3 years
Height-for-age-Z-score (HAZ)
Time frame: Up to 3 years
Body-Mass-Index Z-score (BMIZ)
Time frame: Up to 3 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2023.
- Results publication: PubMed 31495952Kocoshis 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.
- Results publication: PubMed 27855998Carter 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.
- Derived from this study: PubMed 42754721Jimenez L, Ray A, Mercer D, Kocoshis S, Kinberg S, Martinez M, Gumer L, Merritt R, Corkins MR, Vanderpool C, Cohran V, Wendel D, Thompson J, Sudan D, Van Buren KW, Kaufman S, Carey AN, Sabrdaran S, Mitchell P, Dahlberg SE, Weller E, Duggan CP. Safety and long-term effectiveness of teduglutide in children with intestinal failure due to short bowel syndrome: a multicenter, post-marketing cohort study. Pediatr Res. 2026 Sep 17. doi: 10.1038/s41390-026-05489-0. Online ahead of print.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lissette Jimenez principal investigator
Boston Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Short Bowel Syndrome, run by Boston Children's Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent change in calories from parenteral support (PS) (Kcal/kg/day) (time frame: Up to 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04832087, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Dec 2023.