Quality of Life in Patients With Short Bowel Syndrome Treated Without and With Teduglutide - a Prospective Nested Matched Pair Analysis (MatchedQoL)

January 26, 2021 updated by: Elisabeth Blüthner, Charite University, Berlin, Germany
The aim of this prospective longitudinal study is to compare the quality of life of short bowel patients prior to and on teduglutide treatment with a non-treated patient group in a matched-pair design.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Detailed Description

Short bowel syndrome (SBS) is a malabsorptive disorder mostly caused by surgical interventions, which may result in chronic intestinal failure (cIF). The incidence is progressively increasing due to more aggressive surgical and medical approaches. Parenteral nutrition is still the mainstay of treatment for patients with irreversible cIF. New hormonal therapies hold promise by romoting mucosal growth and intestinal absorption and thereby leading to a consequent reduction in parenteral support and symptoms related to large stomal or faecal losses. Outside the typical primary endpoints of cost data, quality of life (QoL) is an important and yet underappreciated consideration of the effectiveness of targeted therapies. In the clinical STEPS trial, Jeppesen et al. have previously shown a significant improvement of the SBS-QoL total score after 24 week treatment with teduglutide longitudinally but could not identify a statistical significance to the placebo group. Accordingly, Chen and colleagues carried out a post hoc analysis of the afore mentioned clinical trial data based on 86 patients and could confirm the results auf Jeppesen et al. Taken together, these clinical trial data did not show a direct benefit of teduglutide treatment on QoL in these patients, while real life data on QoL changes are not available yet. During the release of teduglutide in Germany, the PNLiver trial (DRKS00010993) recruited chronic intestinal failure patients with parenteral nutrition from 2014 till 2019 to evaluate the capability of non-invasive liver function tests in cIF patients in a cross-sectional (n=90) and longitudinal study (n=20. All participants underwent study visits including clinical examination, dynamic liver function assessment, comprehensive blood tests, nutritional status assessment and quality of life assessment (Short Form 36 [SF-36] and SBS-QoL). Therefore, we have a comprehensive data set of a large monocentric cIF cohort prior to teduglutide treatment. Consequently, our unique QoL data from the PNLiver trail enables a followup assessment for an observation period above 24 weeks of patients who were in the meantime exposed to teduglutide in a real life setting. Nevertheless, QoL also tends to improve with longer duration on HPN. Therefore, we are aiming to extend the results with a pairwise matched control group from non teduglutide-treated PNLiver trail patients and collect follow-up data from this group accordingly.

Study Type

Observational

Enrollment (Anticipated)

40

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
      • Berlin, Germany, 10117
        • Recruiting
        • Charite University, Berlin, Germany

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

A comprehensive data set of a large monocentric cIF cohort prior to teduglutide treatment from the PNLiver trail already exists. Consequently, we aim to have a follow-up assessment of patients who are in the meantime treated with teduglutide as a clinical routine procedure.

We aim to extent the results with a pairwise matched control group from non teduglutide-treated PNLiver trail patients and collect follow-up data from this group accordingly.

Description

Inclusion Criteria:

  • SBS-associated adult chronic IF with parenteral nutritional support
  • previous inclusion in the PNLiver trial with existing SF-36 and SBSQoL data
  • signed informed consent

Exclusion Criteria:

  • teduglutide stop before study initiation
  • refusal or withdrawal of consent

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Non-treatment Group
  1. Quality of life assessment (SBS-QoL, SF- 36)
  2. Nutrition status assessment (BIA, BMI)
  3. Assessment of functional absorptive small bowel length (citrulline)
  4. Clinical data (nutritional program, stool characteristics)
Teduglutide-treated Group
  1. Quality of life assessment (SBS-QoL, SF- 36)
  2. Nutrition status assessment (BIA, BMI)
  3. Assessment of functional absorptive small bowel length (citrulline)
  4. Clinical data (nutritional program, stool characteristics)
Drug: Teduglutide
Intervention group consists of patients, who receive teduglutide within routine medical care

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
changes in QoL data during teduglutide therapy
Time Frame: through study completion, an average of 1 year
SBS-QoL, SF-36
through study completion, an average of 1 year
changes in QoL data of patients without teduglutide therapy
Time Frame: through study completion, an average of 1 year
SBS-QoL, SF-36
through study completion, an average of 1 year
changes in QoL data of teduglutide treated patients compared to changes in pairwise matched controls
Time Frame: through study completion, an average of 1 year
SBS-QoL, SF-36
through study completion, an average of 1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
BMI in kg/m^2
Time Frame: through study completion, an average of 1 year
weight and height will be combined to report BMI in kg/m^2
through study completion, an average of 1 year
Body cell mass in kg
Time Frame: through study completion, an average of 1 year
measured by bioelectrical impedance analysis
through study completion, an average of 1 year
citrulline levels
Time Frame: through study completion, an average of 1 year
through study completion, an average of 1 year
stool characteristics by bristol stool scale
Time Frame: through study completion, an average of 1 year
seven types of stool from 1 (hard lumps) till 7 (watery)
through study completion, an average of 1 year
parenteral support frequency
Time Frame: through study completion, an average of 1 year
through study completion, an average of 1 year
parenteral support volume
Time Frame: through study completion, an average of 1 year
through study completion, an average of 1 year
parenteral support calories
Time Frame: through study completion, an average of 1 year
through study completion, an average of 1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Charite University, Berlin, Germany

Collaborators

Takeda

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

September 1, 2020

Primary Completion (ANTICIPATED)

September 1, 2021

Study Completion (ANTICIPATED)

September 1, 2022

Study Registration Dates

First Submitted

August 29, 2020

First Submitted That Met QC Criteria

January 26, 2021

First Posted (ACTUAL)

February 1, 2021

Study Record Updates

Last Update Posted (ACTUAL)

February 1, 2021

Last Update Submitted That Met QC Criteria

January 26, 2021

Last Verified

January 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IIR-DE-002726

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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