Short Bowel SyndromeMatchedQoLNCT04733066
Quality of Life in Patients With Short Bowel Syndrome Treated Without and With Teduglutide - a Prospective Nested Matched Pair Analysis
An observational study of Short Bowel Syndrome and Intestinal Failure, run by Charite University, Berlin, Germany at 1 site in Germany.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Short Bowel Syndrome, Intestinal Failure
- What is being tested?
- Teduglutide
- How many people
- 40 participants
- Where
- 1 siteGermany
- Study dates
- 1 Sep 2020to Sep 2022
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- teduglutide stop before study initiation
- refusal or withdrawal of consent
Trial locations and contacts
1 site in 1 country.
Sophie Pevny, Dr. contact
Germany1 site, 1 recruiting
- Recruiting
Charite University, Berlin, Germany
Berlin, 10117
About this study
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.
Read the 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.
- Conditions
- Short Bowel Syndrome, Intestinal Failure
- Being tested
- Teduglutide
- Keywords
- Quality of Life, Teduglutide, GLP-2 Analogue
- Medical subject headings
- Short Bowel Syndrome, Intestinal Failure
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TeduglutideDrug
Intervention group consists of patients, who receive teduglutide within routine medical care
| Study group | Role | What they receive |
|---|---|---|
| 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) Receives: Drug: Teduglutide |
What is being measured
The primary outcome is the main question the study is designed to answer.
changes in QoL data during teduglutide therapy
SBS-QoL, SF-36
Measured: through study completion, an average of 1 year
changes in QoL data of patients without teduglutide therapy
SBS-QoL, SF-36
Measured: through study completion, an average of 1 year
changes in QoL data of teduglutide treated patients compared to changes in pairwise matched controls
SBS-QoL, SF-36
Measured: through study completion, an average of 1 year
Secondary outcomes (7)
BMI in kg/m^2
weight and height will be combined to report BMI in kg/m^2
Measured: through study completion, an average of 1 year
Body cell mass in kg
measured by bioelectrical impedance analysis
Measured: through study completion, an average of 1 year
citrulline levels
Measured: through study completion, an average of 1 year
stool characteristics by bristol stool scale
seven types of stool from 1 (hard lumps) till 7 (watery)
Measured: through study completion, an average of 1 year
parenteral support frequency
Measured: through study completion, an average of 1 year
parenteral support volume
Measured: through study completion, an average of 1 year
parenteral support calories
Measured: through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Feb 2021
- Study start1 Sep 2020
- Primary completionSep 2021 (estimated)
- Study completionSep 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)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 Charite University, Berlin, GermanyThe 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
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Charite University, Berlin, Germany
- Sponsor type
- University, hospital or other
- Collaborators
- Takeda
- Responsible party
- Principal investigator, Elisabeth Blüthner, Charite University, Berlin, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04733066
- Sponsor's protocol ID
- IIR-DE-002726
- Acronym
- MatchedQoL
- Status last verified
- Jan 2021
- First submitted
- 29 Aug 2020
- Last updated
- 1 Feb 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Jeppesen PB, Pertkiewicz M, Forbes A, Pironi L, Gabe SM, Joly F, Messing B, Loth S, Youssef NN, Heinze H, Berghofer P. Quality of life in patients with short bowel syndrome treated with the new glucagon-like peptide-2 analogue teduglutide--analyses from a randomised, placebo-controlled study. Clin Nutr. 2013 Oct;32(5):713-21. doi: 10.1016/j.clnu.2013.03.016. Epub 2013 Mar 28.
PubMed 23587733Chen K, Mu F, Xie J, Kelkar SS, Olivier C, Signorovitch J, Jeppesen PB. Impact of Teduglutide on Quality of Life Among Patients With Short Bowel Syndrome and Intestinal Failure. JPEN J Parenter Enteral Nutr. 2020 Jan;44(1):119-128. doi: 10.1002/jpen.1588. Epub 2019 Apr 21.
PubMed 31006876Bluthner E, Bednarsch J, Pape UF, Karber M, Maasberg S, Gerlach UA, Pascher A, Wiedenmann B, Pratschke J, Stockmann M. Advanced liver function assessment in patients with intestinal failure on long-term parenteral nutrition. Clin Nutr. 2020 Feb;39(2):540-547. doi: 10.1016/j.clnu.2019.02.039. Epub 2019 Mar 5.
PubMed 30885502Bluthner E, Bednarsch J, Stockmann M, Karber M, Pevny S, Maasberg S, Gerlach UA, Pascher A, Wiedenmann B, Pratschke J, Pape UF. Determinants of Quality of Life in Patients With Intestinal Failure Receiving Long-Term Parenteral Nutrition Using the SF-36 Questionnaire: A German Single-Center Prospective Observational Study. JPEN J Parenter Enteral Nutr. 2020 Feb;44(2):291-300. doi: 10.1002/jpen.1531. Epub 2019 Mar 13.
PubMed 30864177
Study at a glance
From the registry record, last updated by the sponsor on 1 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Quality of Life in Patients With Short Bowel Syndrome Treated Without and With Teduglutide - a Prospective Nested Matched Pair Analysis
- ClinicalTrials.gov ID
- NCT04733066
- Acronym / sponsor ID
- MatchedQoL / IIR-DE-002726
- Conditions
- Short Bowel Syndrome, Intestinal Failure
- Keywords
- Quality of Life, Teduglutide, GLP-2 Analogue
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Charite University, Berlin, GermanyUniversity, hospital or other
- Collaborators
- Takeda
- Study start
- 1 Sep 2020 (actual)
- Primary completion
- Sep 2021 (estimated)
- Study completion
- Sep 2022 (estimated)
- First posted
- 1 Feb 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.
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.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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 | – | Drug: Teduglutide1. 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) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (2 listed)
- teduglutide stop before study initiation
- refusal or withdrawal of consent
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 |
|---|---|---|
| TeduglutideIntervention group consists of patients, who receive teduglutide within routine medical care | Drug | Teduglutide-treated Group |
Primary outcome
changes in QoL data during teduglutide therapy
Time frame: through study completion, an average of 1 year
SBS-QoL, SF-36
changes in QoL data of patients without teduglutide therapy
Time frame: through study completion, an average of 1 year
SBS-QoL, SF-36
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
7 secondary outcomes
BMI in kg/m^2
Time frame: through study completion, an average of 1 year
Body cell mass in kg
Time frame: through study completion, an average of 1 year
citrulline levels
Time frame: through study completion, an average of 1 year
stool characteristics by bristol stool scale
Time frame: through study completion, an average of 1 year
parenteral support frequency
Time frame: through study completion, an average of 1 year
parenteral support volume
Time frame: through study completion, an average of 1 year
parenteral support calories
Time frame: through study completion, an average of 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Feb 2021.
- Background: PubMed 23587733Jeppesen PB, Pertkiewicz M, Forbes A, Pironi L, Gabe SM, Joly F, Messing B, Loth S, Youssef NN, Heinze H, Berghofer P. Quality of life in patients with short bowel syndrome treated with the new glucagon-like peptide-2 analogue teduglutide--analyses from a randomised, placebo-controlled study. Clin Nutr. 2013 Oct;32(5):713-21. doi: 10.1016/j.clnu.2013.03.016. Epub 2013 Mar 28.
- Background: PubMed 31006876Chen K, Mu F, Xie J, Kelkar SS, Olivier C, Signorovitch J, Jeppesen PB. Impact of Teduglutide on Quality of Life Among Patients With Short Bowel Syndrome and Intestinal Failure. JPEN J Parenter Enteral Nutr. 2020 Jan;44(1):119-128. doi: 10.1002/jpen.1588. Epub 2019 Apr 21.
- Background: PubMed 30885502Bluthner E, Bednarsch J, Pape UF, Karber M, Maasberg S, Gerlach UA, Pascher A, Wiedenmann B, Pratschke J, Stockmann M. Advanced liver function assessment in patients with intestinal failure on long-term parenteral nutrition. Clin Nutr. 2020 Feb;39(2):540-547. doi: 10.1016/j.clnu.2019.02.039. Epub 2019 Mar 5.
- Background: PubMed 30864177Bluthner E, Bednarsch J, Stockmann M, Karber M, Pevny S, Maasberg S, Gerlach UA, Pascher A, Wiedenmann B, Pratschke J, Pape UF. Determinants of Quality of Life in Patients With Intestinal Failure Receiving Long-Term Parenteral Nutrition Using the SF-36 Questionnaire: A German Single-Center Prospective Observational Study. JPEN J Parenter Enteral Nutr. 2020 Feb;44(2):291-300. doi: 10.1002/jpen.1531. Epub 2019 Mar 13.
- 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.
Sophie Pevny, Dr. contact
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 and Intestinal Failure, run by Charite University, Berlin, Germany at 1 site in Germany.
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 3 inclusion and 2 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: status not confirmed, as of the sponsor's last update on 1 Feb 2021. 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 Germany. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Elisabeth Blüthner, MD (004930450665209, elisabeth.bluethner@charite.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
changes in QoL data during teduglutide therapy (time frame: through study completion, an average of 1 year). The study lists 3 primary outcomes in all. 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 NCT04733066, which also lists 4 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 1 Feb 2021.