Short Bowel SyndromeRESTORENCT04976075
ImpRovE underSTanding of Short bOwel syndRome in Latin-amErica
An observational study of Short Bowel Syndrome, run by La Fundacion Favaloro para la Investigacion y la Docencia Medica at 1 site in Argentina.
Trial summary
Background and justification: Short bowel syndrome (SBS) is a disabling and life-threatening condition that results from a partial or total bowel resection, and has become as the main cause of Type III, intestinal failure (IF). Read more about this study
- Who is it for?
- Ages 1 week to 100 years
- Conditions the study is for
- Short Bowel Syndrome
- How many people
- 150 participants
- Where
- 1 siteArgentina
- Study dates
- 1 Jun 2017to 30 Apr 2023
- 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 week to 100 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Based on the SIC prevalence calculations, we aim to include 150 patients. The inclusion of patients will be done at the national level, considering as reference centers those hospitals that have Nutritional Support and Intestinal Rehabilitation Units, both public and private. Researchers may consecutively include all patients who give their consent and meet the selection criteria.
Who may be able to join
- Patients of both sexes, diagnosed with Type III intestinal failure (FI) due to SIC
- Signature of informed consent and assent as appropriate
- 8 or more consecutive weeks of PN need
Who cannot join
- Less than 8 weeks of need for PN after diagnosis of SIC / FI
- Refusal to provide informed consent and assent as appropriate
- FI type I and II, and type III without SIC
Trial locations and contacts
1 site in 1 country.
Argentina1 site
University Hospital of the Favaloro Foundation
Buenos Aires, C1093AAS
About this study
Official title: Multicenter Prospective Observational and Epidemiological Registry for Patients With Intestinal Failure Due to Short Bowel Syndrome, in Latin America
Background and justification: Short bowel syndrome (SBS) is a disabling and life-threatening condition that results from a partial or total bowel resection, and has become as the main cause of Type III, intestinal failure (IF). Immediately after enterectomy there is an adaptation process consisting of structural, hormonal and metabolic changes to maximize intestinal function. These changes begin within days of resection and generally continue for several months. However, recent publications have reported even longer periods of time (up to 5 years) to achieve enteral autonomy.
It is possible to enhance the natural process of intestinal adaptation through medical or surgical treatments, called intestinal rehabilitation. During the process, complications related to intestinal failure or its treatment may arise, jeopardizing the result and even compromising survival. A better understanding of the medium and long-term results of patients under medical and / or surgical treatment with SIC is needed.
Read more
Despite the improvement recently achieved in managing IF, in most countries, pts are dispersed and seen by general health-care providers, with limited SBS or IF experience, causing increasing concern regarding the competence and equity of the care accessible to suffering pts.
The results obtained with the RESTORE project in adult patients highlight the relevance of having registries to better understand the natural history of this disease in adult patients, proving that a larger number of adult pts with SBS/III-IF can be identified and cared for than the numbers considered by recognized estimations. [Abstract sent to TTS, 2020].
To date, there are no data for pediatric patients with iIF secondary to SIC in Latin America, so its incidence, prevalence and evolution are unknown. Recent publications from middle-income countries, exposed the current inequality regarding the different types of therapies available within a given region.
Given the high morbidity and mortality associated with ICS-FI, there is an unmet need to create an adequate study that provides the information necessary to establish local and regional parameters and recommendations on its treatment.
Read the detailed description
RESTORE amedment 1project, obtained the study number DDI (1384) 1317 from the Institutional Research and Ethics committee after its approving the choices of participating centers by RESTORE's Scientific Steering Committee. For participation, those centers needed a full-time or part-time surgeon-though not necessarily offing rehabilitation surgery or transplantation or accessing enterohormone (EH) therapies-a therapist with nutritional experience, a dietitian, and specialized pharmaceutical resources. Participating teams, however, did not necessarily have to offer rehabilitation surgery or transplantation capabilities or have access to EH therapies to be elected for participation.
RESTORE's aim was assessing the long-term outcomes of SBS pts with IF, under the current standard of care in order to plumb IF's natural history, the indications for currently available therapies, and their means of modifying each course.
Study Designed: the first prospective, observational, epidemiological, multicenter study in Argentina of adult pts with SBS-caused type-III IF in Lat-America.
In adults, the inclusion of additional centers in Argentina, Mexico, Colombia, Peru, Uruguay and Chile will be required. In pediatric patients, it is proposed to add patients and centers in Argentina and, if necessary, in other Latin American countries (Mexico, Colombia, Peru, Chile and Uruguay).
Number of patients and centers:
The study aims to recruit at least 150 patients in 24 centers. The sample size is based on the expected number of subjects who may be eligible and willing to participate in the study.
Diagnosis and inclusion criteria
Inclusion criteria:
- Patients of both sexes, diagnosed with Type III-IF due to SBS
- Signature of informed consent and assent as appropriate
- 8 or more consecutive weeks of PN need
Exclusion criteria:
- Less than 8 weeks of need for PN after diagnosis of SBS / IF
- Refusal to provide informed consent and assent as appropriate
- FI type I and II, and type III without SBS
The data from this study will be entered into an electronic CRF and database; with visits being reinitiated at weeks 4, 8, 12, 20, and 24 and then yearly thereafter. Pts that missed 2 or more visits were considered lost from follow-up. Death, recovery of intestinal sufficiency, and intestinal transplant were considered the end points.
The results of this study will be summarized and evaluated using descriptive statistics.
- Conditions
- Short Bowel Syndrome
- Being tested
- no interventions
- 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.
no interventionsOther
no interventions
| Study group | Role | What they receive |
|---|---|---|
| Adults | Patients of 18 or more years old Receives: Other: no interventions | |
| Pediatrics | Patients less than 18 years old Receives: Other: no interventions |
What is being measured
The primary outcome is the main question the study is designed to answer.
Describe the long term outcome of Type III-IF patients with short bowel syndrome in Latinoamérica.
The number of patients with short bowel syndrome/year, the number of patients able to recover intestinal sufficiency under the current available therapy that includes a) PN, b) medical and / or surgical rehabilitation, under the routine clinical practice in Latin American countries, will be reported. Long term survival (in months), freedom from Home Parenteral Nutrition survival in months, as measure for PN independence or recovery of intestinal insufficiency will be measured. The existence on post-surgical complications will be reported by using the Dindo-Clavien classification score (from 0 to 5).
Measured: April 2023
Secondary outcomes (7)
Describe the demographic characteristics and distribution of the disease
Type III-IF due to Short Bowel Syndrome patients' location will be descried.
Measured: April 2023
Describe the nutritional progress
Nutritional progress (in Kgs and SGA) and growth (in cm, in children) in parallel with the assessment of the caloric intake (enteral and parenteral- in Kcal/week) and volume (l/week).
Measured: April 2023
Record the use of Enterohormones
Those pts who could not continue decreasing HPN for 6-months, but had remained clinically stable, were considered candidates for EH therapies with the semisynthetic glucagon-like peptides 1 and 2 (sGLP-1, sGLP-2) according to each individual's need and potential benefit.
In the sGLP-1 or sGLP-2 treatment, the initiation date, the clinical characteristics and course, and any adverse effects were recorded.
Measured: April 2023
Describe the use, management of HPN, and its related complications
The HPN starting date, volume, kcals, and number of days per week were registered. HPN-related complications-including liver disease, catheter-related infections, central-venous-access loss, volume and/or caloric-requirement (kcal) changes throughout the course of care, or modifications in number of infusion days and volume during the treatment-were also registered and the ESPEN clinical classification recorded. Describe the incidence of PN-related complications: liver disease / failure associated with intestinal failure and PN, catheter-related infection, thrombosis, and loss of venous access.
Measured: April 2023
Describe the surgical procedures, used, timing, outcomes and complications
Describe the surgical procedures used as part of intestinal rehabilitation, its complications and results CRATGI, or elongation surgery either with the Bianchi technique or STEP (Serial Transverse Enteroplasty). Postsurgical complications were registered after Dindo-Clavien's classification.
Measured: April 2023
Assess the quality of life
Assess the quality of life (QoL) of patients under treatment (for the adult cohort) by using a brief survey assessing consistent in 3 questions to observe 3 main areas or domain: 1) Profesional/work performance, 2) Social activity and 3 Sexual Activity, based on a 0 to 10 index. Will be apply only o adult patients. .
Measured: April 2023
Time to obtain therapy access
Time from from diagnoses to treatment access (in months) by age group and disease, will be asses.
Measured: April 2023
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jul 2021
- Study start1 Jun 2017
- Primary completion30 Apr 2023 (estimated)
- Study completion30 Apr 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Short Bowel SyndromeStudies of the same condition, open and under way.
- 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
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- La Fundacion Favaloro para la Investigacion y la Docencia Medica
- Sponsor type
- University, hospital or other
- Collaborators
- Fundación de Ciencias Exactas - UNLP
- Responsible party
- Principal investigator, Prof. Dr. Gabriel E. Gondolesi, La Fundacion Favaloro para la Investigacion y la Docencia Medica
- Investigators
- Gabriel Gondolesi, Doctor, Principal investigator, University Hospital of the Favaloro Foundation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04976075
- Sponsor's protocol ID
- DDI 1384 1317 CBE 666-17
- Acronym
- RESTORE
- Status last verified
- Aug 2021
- First submitted
- 21 May 2021
- Last updated
- 20 Aug 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter Prospective Observational and Epidemiological Registry for Patients With Intestinal Failure Due to Short Bowel Syndrome, in Latin America
- ClinicalTrials.gov ID
- NCT04976075
- Acronym / sponsor ID
- RESTORE / DDI 1384 1317 CBE 666-17
- Conditions
- Short Bowel Syndrome
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- La Fundacion Favaloro para la Investigacion y la Docencia MedicaUniversity, hospital or other
- Collaborators
- Fundación de Ciencias Exactas - UNLP
- Study start
- 1 Jun 2017 (actual)
- Primary completion
- 30 Apr 2023 (estimated)
- Study completion
- 30 Apr 2023 (estimated)
- First posted
- 26 Jul 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.
Background and justification: Short bowel syndrome (SBS) is a disabling and life-threatening condition that results from a partial or total bowel resection, and has become as the main cause of Type III, intestinal failure (IF). Immediately after enterectomy there is an adaptation process consisting of structural, hormonal and metabolic changes to maximize intestinal function. These changes begin within days of resection and generally continue for several months. However, recent publications have reported even longer periods of time (up to 5 years) to achieve enteral autonomy.
It is possible to enhance the natural process of intestinal adaptation through medical or surgical treatments, called intestinal rehabilitation. During the process, complications related to intestinal failure or its treatment may arise, jeopardizing the result and even compromising survival. A better understanding of the medium and long-term results of patients under medical and / or surgical treatment with SIC is needed.
Despite the improvement recently achieved in managing IF, in most countries, pts are dispersed and seen by general health-care providers, with limited SBS or IF experience, causing increasing concern regarding the competence and equity of the care accessible to suffering pts.
The results obtained with the RESTORE project in adult patients highlight the relevance of having registries to better understand the natural history of this disease in adult patients, proving that a larger number of adult pts with SBS/III-IF can be identified and cared for than the numbers considered by recognized estimations. [Abstract sent to TTS, 2020].
To date, there are no data for pediatric patients with iIF secondary to SIC in Latin America, so its incidence, prevalence and evolution are unknown. Recent publications from middle-income countries, exposed the current inequality regarding the different types of therapies available within a given region.
Given the high morbidity and mortality associated with ICS-FI, there is an unmet need to create an adequate study that provides the information necessary to establish local and regional parameters and recommendations on its treatment.
Detailed description
RESTORE amedment 1project, obtained the study number DDI (1384) 1317 from the Institutional Research and Ethics committee after its approving the choices of participating centers by RESTORE's Scientific Steering Committee. For participation, those centers needed a full-time or part-time surgeon-though not necessarily offing rehabilitation surgery or transplantation or accessing enterohormone (EH) therapies-a therapist with nutritional experience, a dietitian, and specialized pharmaceutical resources. Participating teams, however, did not necessarily have to offer rehabilitation surgery or transplantation capabilities or have access to EH therapies to be elected for participation.
RESTORE's aim was assessing the long-term outcomes of SBS pts with IF, under the current standard of care in order to plumb IF's natural history, the indications for currently available therapies, and their means of modifying each course.
Study Designed: the first prospective, observational, epidemiological, multicenter study in Argentina of adult pts with SBS-caused type-III IF in Lat-America.
In adults, the inclusion of additional centers in Argentina, Mexico, Colombia, Peru, Uruguay and Chile will be required. In pediatric patients, it is proposed to add patients and centers in Argentina and, if necessary, in other Latin American countries (Mexico, Colombia, Peru, Chile and Uruguay).
Number of patients and centers:
The study aims to recruit at least 150 patients in 24 centers. The sample size is based on the expected number of subjects who may be eligible and willing to participate in the study.
Diagnosis and inclusion criteria
Inclusion criteria:
- Patients of both sexes, diagnosed with Type III-IF due to SBS
- Signature of informed consent and assent as appropriate
- 8 or more consecutive weeks of PN need
Exclusion criteria:
- Less than 8 weeks of need for PN after diagnosis of SBS / IF
- Refusal to provide informed consent and assent as appropriate
- FI type I and II, and type III without SBS
The data from this study will be entered into an electronic CRF and database; with visits being reinitiated at weeks 4, 8, 12, 20, and 24 and then yearly thereafter. Pts that missed 2 or more visits were considered lost from follow-up. Death, recovery of intestinal sufficiency, and intestinal transplant were considered the end points.
The results of this study will be summarized and evaluated using descriptive statistics.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Adults | – | Other: no interventionsPatients of 18 or more years old |
| Pediatrics | – | Other: no interventionsPatients less than 18 years old |
Who may qualify
- Ages
- 1 week to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Based on the SIC prevalence calculations, we aim to include 150 patients. The inclusion of patients will be done at the national level, considering as reference centers those hospitals that have Nutritional Support and Intestinal Rehabilitation Units, both public and private. Researchers may consecutively include all patients who give their consent and meet the selection criteria.
Key inclusion criteria (3 listed)
- Patients of both sexes, diagnosed with Type III intestinal failure (FI) due to SIC
- Signature of informed consent and assent as appropriate
- 8 or more consecutive weeks of PN need
Key exclusion criteria (3 listed)
- Less than 8 weeks of need for PN after diagnosis of SIC / FI
- Refusal to provide informed consent and assent as appropriate
- FI type I and II, and type III without SIC
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 |
|---|---|---|
| no interventionsno interventions | Other | Adults, Pediatrics |
Primary outcome
Describe the long term outcome of Type III-IF patients with short bowel syndrome in Latinoamérica.
Time frame: April 2023
The number of patients with short bowel syndrome/year, the number of patients able to recover intestinal sufficiency under the current available therapy that includes a) PN, b) medical and / or surgical rehabilitation, under the routine clinical practice in Latin American countries, will be reported. Long term survival (in months), freedom from Home Parenteral Nutrition survival in months, as measure for PN independence or recovery of intestinal insufficiency will be measured. The existence on post-surgical complications will be reported by using the Dindo-Clavien classification score (from 0 to 5).
7 secondary outcomes
Describe the demographic characteristics and distribution of the disease
Time frame: April 2023
Describe the nutritional progress
Time frame: April 2023
Record the use of Enterohormones
Time frame: April 2023
Describe the use, management of HPN, and its related complications
Time frame: April 2023
Describe the surgical procedures, used, timing, outcomes and complications
Time frame: April 2023
Assess the quality of life
Time frame: April 2023
Time to obtain therapy access
Time frame: April 2023
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2021.
- 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.
Gabriel Gondolesi, Doctor principal investigator
University Hospital of the Favaloro Foundation
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 La Fundacion Favaloro para la Investigacion y la Docencia Medica at 1 site in Argentina.
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 3 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 20 Aug 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 Argentina. 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?
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?
Describe the long term outcome of Type III-IF patients with short bowel syndrome in Latinoamérica. (time frame: April 2023). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 20 Aug 2021; AzTrials last refreshed its copy on 8 Nov 2026.