Pediatric Intestinal FailureNCT04629014
Prospective Multisite Study of Quality of Life in Pediatric Intestinal Failure
An observational study of Pediatric Intestinal Failure, run by Boston Children's Hospital at 14 sites in 2 countries.
Trial summary
This study proposes to quantify and describe the quality of life of children with intestinal failure, and to identify the medical and socio-economic factors that impact this quality of life, using data from multiple multidisciplinary intestinal failure centers across the United States and Canada specializing in the… Read more about this study
- Who is it for?
- Ages 6 months to 25 years
- Conditions the study is for
- Pediatric Intestinal Failure
- How many people
- 750 participants
- Where
- 14 sitesin 2 countries
- Study dates
- 27 Feb 2020to Dec 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 6 months to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: Participants will be children with a specific diagnosis of intestinal failure as determined by parenteral nutrition dependence at any point in their history for more than 60 out of 74 consecutive days who are managed at one of the intestinal rehabilitation programs located at the study participating sites.
Who may be able to join
- Participants will be followed in a participating institution's intestinal rehabilitation program
- Participants will have a diagnosis of intestinal failure due to functional or structural intestinal dysfunction with current or prior history of specialized nutritional support (parenteral nutrition requirement for 60 out of 74 consecutive days)
- Participants will be age 6 months to 25 years old.
- Parents/caregivers must be able to complete questionnaire without assistance.
- English or Spanish speaking
Who cannot join
- Participants aged less than 6 months or greater than 25 years will not be included in this study
- Participants will not be enrolled less than 3 months from index admission or initial outpatient evaluation
- Participants will not be enrolled less than 1 month from inpatient admission (any admission greater than 24 hours in duration)
- Participants will not be enrolled less than 1 month from any operative intervention requiring general anesthesia.
- Primary language other than English or Spanish.
- Participants who have a currently functional small bowel, liver/small bowel or multivisceral transplant
- While other major co-morbidities may be excluded at a later time during data analysis, or may be analyzed as a specific sub-group, they will not be excluded outright. For example, participants with global developmental delay may have parent surveys only, and therefore would need to be excluded from paired analysis of proxy vs. participant perception of HRQOL
Trial locations and contacts
14 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
14 sites in 2 countries
United States11 sites, 11 recruiting
- Recruiting
C.S. Mott Children's Hospital
Ann Arbor, Michigan, 48109
Meghan Arnold, MD, meghanar@med.umich.edu
Meghan Arnold, MD
- Recruiting
Children's Hospital Colorado
Aurora, Colorado, 80045
Lindsey Gumer, MD, MSc, 720-777-6669, lindsey.gumer@childrenscolorado.org
Lindsey Gumer, MD, MSc
- Not yet recruiting
Children's of Alabama
Birmingham, Alabama, 35233
David Galloway, MD, dgalloway@peds.uab.edu
David Galloway, MD
- Recruiting
Boston Children's Hospital
Boston, Massachusetts, 02115
Biren P Modi, MD MPH, 857-218-4651, biren.modi@childrens.harvard.edu
Biren P Modi, MD MPH
- Recruiting
Lurie Children's Hospital
Chicago, Illinois, 60611
Valeria Cohran, MD, vcohran@luriechildrens.org
Valeria Cohran, MD
- Recruiting
Dallas Children's Hospital
Dallas, Texas, 75390
Nandini Channabasappa, MD, nandini.channabasappa@utsouthwestern.edu
Nandini Channabasappa, MD
- Recruiting
Duke Children's Hospital
Durham, North Carolina, 27710
Debra Sudan, MD, debra.sudan@duke.edu
Debra Sudan, MD
- Recruiting
Connecticut Children's Medical Center
Hartford, Connecticut, 06106
Jonathan Salazar, MD, 860-545-9560, jsalazar02@connecticutchildrens.org
Jonathan Salazar, MD
- Recruiting
UT Health Houston
Houston, Texas, 77030
Essam Imseis, MD, 713-500-5663, essam.imseis@uth.tmc.edu
Essam Imseis, MD
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
Patrick J Javid, MD, patrick.javid@seattlechildrens.org
Patrick J Javid, MD
- Recruiting
St. Louis Children's Hospital
St Louis, Missouri, 63100
Brad Warner, MD, brad.warner@wustl.edu
Brad Warner, MD
Canada3 sites, 3 recruiting
- Recruiting
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8
Dana Boctor, MD, dana.boctor@albertahealthservices.ca
Dana Boctor, MD
- Recruiting
Hospital for Sick Children
Toronto, Ontario, M5G 1X8
Christina Belza, PhD, RN, 416-813-5347, christina.belza@sickkids.ca
Christina Belza, PhD, RN
- Recruiting
BC Children's Hospital
Vancouver, British Columbia, V6H 3N1
Hannah Piper, MD, hannah.piper@cw.bc.ca
Hannah Piper, MD
About this study
This study proposes to quantify and describe the quality of life of children with intestinal failure, and to identify the medical and socio-economic factors that impact this quality of life, using data from multiple multidisciplinary intestinal failure centers across the United States and Canada specializing in the care of these participants.
Read the detailed description
Health related quality of life is becoming an increasingly important topic as medical advancements continue to increase life expectancies in various illnesses. Quality of life (QoL) encompasses the physical, psychological, and social well-being of a person. Studies focusing on health related QoL show that providers have a limited understanding of the QoL of their patients, demonstrating a need for further research. Mortality rates in pediatric intestinal failure (PIF) have significantly dropped over the past 3-4 decades and long-term morbidity rather than survival has come into focus as the outcome of interest. However, there remains a significant knowledge gap regarding the specific factors that contribute to QoL among PIF patients and their families. Insight into the challenges these patients face can promote enhanced support services and psycho-education for caregivers of these children as well as help to tailor medical and surgical treatment with a focus on improved QoL. Due to the rare nature of PIF, and despite a recent bloom in multidisciplinary PIF centers, experience is limited in individual centers and a multi-site study with a robust and diverse patient population is optimal to study QoL in pediatric intestinal failure.
- Conditions
- Pediatric Intestinal Failure
- Keywords
- intestinal failure, short bowel syndrome, short gut syndrome, parenteral nutrition, specialized nutrition
- Medical subject headings
- Intestinal Failure, Short Bowel Syndrome, Hyperphagia
What is being measured
The primary outcome is the main question the study is designed to answer.
Health-related quality of life (hrQOL)
hrQOL as determined by PedsQL Generic Core at time of enrollment and corrected for time from onset of disease
Measured: Baseline
Health-related quality of life (hrQOL) Change over time
hrQOL as determined by PedsQL Generic Core, assessed annually, looking at trend in change over 5 years of assessment and corrected for time from onset of disease
Measured: Trend over 5 years
Disease-specific Health-related quality of life (hrQOL)
Disease-specific hrQOL as determined by PedsQL GI Symptoms Scale at time of enrollment and corrected for time from onset of disease
Measured: Baseline
Disease-specific Health-related quality of life (hrQOL)
Disease-specific hrQOL as determined by PedsQL GI Symptoms Scale, assessed annually, looking at trend in change over 5 years of assessment and corrected for time from onset of disease
Measured: Trend over 5 years
Secondary outcomes (2)
Family Impact
Family impact as determined by the PedsQL Family Impact Module
Measured: Baseline
Family Impact
Family impact as assessed by the PedsQL Family Impact Module, assessed annually, looking at trend in change over 5 years of assessment and corrected for time from onset of disease
Measured: Trend over 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Nov 2020
- Study start27 Feb 2020
- Primary completionDec 2029 (estimated)
- Study completionDec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for Pediatric Intestinal FailureStudies 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
- 750 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Seattle Children's Hospital, University of Alabama at Birmingham, Ann & Robert H Lurie Children's Hospital of Chicago, Washington University School of Medicine, University of Michigan, Duke Health, University of Texas Southwestern Medical Center, Alberta Children's Hospital, British Columbia Children's Hospital, The Hospital for Sick Children, The University of Texas Health Science Center, Houston, Children's Hospital Colorado, Connecticut Children's Medical Center
- Responsible party
- Principal investigator, Biren Modi, Boston Children's Hospital
- Investigators
- Patrick J Javid, MD, Principal investigator, Seattle Children's HospitalBiren P Modi, MD MPH, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04629014
- Sponsor's protocol ID
- P00032389
- Status last verified
- Mar 2026
- First submitted
- 29 Oct 2020
- Last updated
- 23 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Diamond IR, de Silva N, Pencharz PB, Kim JH, Wales PW; Group for the Improvement of Intestinal Function and Treatment. Neonatal short bowel syndrome outcomes after the establishment of the first Canadian multidisciplinary intestinal rehabilitation program: preliminary experience. J Pediatr Surg. 2007 May;42(5):806-11. doi: 10.1016/j.jpedsurg.2006.12.033.
PubMed 17502188Javid PJ, Malone FR, Reyes J, Healey PJ, Horslen SP. The experience of a regional pediatric intestinal failure program: Successful outcomes from intestinal rehabilitation. Am J Surg. 2010 May;199(5):676-9. doi: 10.1016/j.amjsurg.2010.01.013.
PubMed 20466115Modi BP, Langer M, Ching YA, Valim C, Waterford SD, Iglesias J, Duro D, Lo C, Jaksic T, Duggan C. Improved survival in a multidisciplinary short bowel syndrome program. J Pediatr Surg. 2008 Jan;43(1):20-4. doi: 10.1016/j.jpedsurg.2007.09.014.
PubMed 18206449Mutanen A, Kosola S, Merras-Salmio L, Kolho KL, Pakarinen MP. Long-term health-related quality of life of patients with pediatric onset intestinal failure. J Pediatr Surg. 2015 Nov;50(11):1854-8. doi: 10.1016/j.jpedsurg.2015.05.012. Epub 2015 Jun 3.
PubMed 26078213Norsa L, Artru S, Lambe C, Talbotec C, Pigneur B, Ruemmele F, Colomb V, Capito C, Chardot C, Lacaille F, Goulet O. Long term outcomes of intestinal rehabilitation in children with neonatal very short bowel syndrome: Parenteral nutrition or intestinal transplantation. Clin Nutr. 2019 Apr;38(2):926-933. doi: 10.1016/j.clnu.2018.02.004. Epub 2018 Feb 15.
PubMed 29478887Sanchez SE, McAteer JP, Goldin AB, Horslen S, Huebner CE, Javid PJ. Health-related quality of life in children with intestinal failure. J Pediatr Gastroenterol Nutr. 2013 Sep;57(3):330-4. doi: 10.1097/MPG.0b013e3182999961.
PubMed 23648789Varni JW, Limbers CA, Burwinkle TM. Impaired health-related quality of life in children and adolescents with chronic conditions: a comparative analysis of 10 disease clusters and 33 disease categories/severities utilizing the PedsQL 4.0 Generic Core Scales. Health Qual Life Outcomes. 2007 Jul 16;5:43. doi: 10.1186/1477-7525-5-43.
PubMed 17634123Modi BP, Piper HG, Belza C, Staffa S, Arnold MA, Boctor DL, Channabasappa N, Cohran VC, Galloway DP, Sudan D, Wales PW, Warner BW, Murtadi G, Javid PJ. Health-Related Quality of Life in Pediatric Intestinal Failure from Infancy to Adolescence: An International, Multi-Center Evaluation. J Pediatr. 2025 Jul;282:114566. doi: 10.1016/j.jpeds.2025.114566. Epub 2025 Mar 28.
PubMed 40158842
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Multisite Study of Quality of Life in Pediatric Intestinal Failure
- ClinicalTrials.gov ID
- NCT04629014
- Acronym / sponsor ID
- P00032389
- Conditions
- Pediatric Intestinal Failure
- Keywords
- intestinal failure, short bowel syndrome, short gut syndrome, parenteral nutrition, specialized nutrition
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 750 participants (estimated)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Collaborators
- Seattle Children's Hospital, University of Alabama at Birmingham, Ann & Robert H Lurie Children's Hospital of Chicago, Washington University School of Medicine, University of Michigan, Duke Health, University of Texas Southwestern Medical Center, Alberta Children's Hospital, British Columbia Children's Hospital, The Hospital for Sick Children, The University of Texas Health Science Center, Houston, Children's Hospital Colorado, Connecticut Children's Medical Center
- Study start
- 27 Feb 2020 (actual)
- Primary completion
- Dec 2029 (estimated)
- Study completion
- Dec 2029 (estimated)
- First posted
- 16 Nov 2020 (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.
This study proposes to quantify and describe the quality of life of children with intestinal failure, and to identify the medical and socio-economic factors that impact this quality of life, using data from multiple multidisciplinary intestinal failure centers across the United States and Canada specializing in the care of these participants.
Detailed description
Health related quality of life is becoming an increasingly important topic as medical advancements continue to increase life expectancies in various illnesses. Quality of life (QoL) encompasses the physical, psychological, and social well-being of a person. Studies focusing on health related QoL show that providers have a limited understanding of the QoL of their patients, demonstrating a need for further research. Mortality rates in pediatric intestinal failure (PIF) have significantly dropped over the past 3-4 decades and long-term morbidity rather than survival has come into focus as the outcome of interest. However, there remains a significant knowledge gap regarding the specific factors that contribute to QoL among PIF patients and their families. Insight into the challenges these patients face can promote enhanced support services and psycho-education for caregivers of these children as well as help to tailor medical and surgical treatment with a focus on improved QoL. Due to the rare nature of PIF, and despite a recent bloom in multidisciplinary PIF centers, experience is limited in individual centers and a multi-site study with a robust and diverse patient population is optimal to study QoL in pediatric intestinal failure.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 6 months to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Participants will be children with a specific diagnosis of intestinal failure as determined by parenteral nutrition dependence at any point in their history for more than 60 out of 74 consecutive days who are managed at one of the intestinal rehabilitation programs located at the study participating sites.
Key inclusion criteria (5 listed)
- Participants will be followed in a participating institution's intestinal rehabilitation program
- Participants will have a diagnosis of intestinal failure due to functional or structural intestinal dysfunction with current or prior history of specialized nutritional support (parenteral nutrition requirement for 60 out of 74 consecutive days)
- Participants will be age 6 months to 25 years old.
- Parents/caregivers must be able to complete questionnaire without assistance.
- English or Spanish speaking
Key exclusion criteria (7 listed)
- Participants aged less than 6 months or greater than 25 years will not be included in this study
- Participants will not be enrolled less than 3 months from index admission or initial outpatient evaluation
- Participants will not be enrolled less than 1 month from inpatient admission (any admission greater than 24 hours in duration)
- Participants will not be enrolled less than 1 month from any operative intervention requiring general anesthesia.
- Primary language other than English or Spanish.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Health-related quality of life (hrQOL)
Time frame: Baseline
hrQOL as determined by PedsQL Generic Core at time of enrollment and corrected for time from onset of disease
Health-related quality of life (hrQOL) Change over time
Time frame: Trend over 5 years
hrQOL as determined by PedsQL Generic Core, assessed annually, looking at trend in change over 5 years of assessment and corrected for time from onset of disease
Disease-specific Health-related quality of life (hrQOL)
Time frame: Baseline
Disease-specific hrQOL as determined by PedsQL GI Symptoms Scale at time of enrollment and corrected for time from onset of disease
Disease-specific Health-related quality of life (hrQOL)
Time frame: Trend over 5 years
Disease-specific hrQOL as determined by PedsQL GI Symptoms Scale, assessed annually, looking at trend in change over 5 years of assessment and corrected for time from onset of disease
2 secondary outcomes
Family Impact
Time frame: Baseline
Family Impact
Time frame: Trend over 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2026.
- Background: PubMed 17502188Diamond IR, de Silva N, Pencharz PB, Kim JH, Wales PW; Group for the Improvement of Intestinal Function and Treatment. Neonatal short bowel syndrome outcomes after the establishment of the first Canadian multidisciplinary intestinal rehabilitation program: preliminary experience. J Pediatr Surg. 2007 May;42(5):806-11. doi: 10.1016/j.jpedsurg.2006.12.033.
- Background: PubMed 20466115Javid PJ, Malone FR, Reyes J, Healey PJ, Horslen SP. The experience of a regional pediatric intestinal failure program: Successful outcomes from intestinal rehabilitation. Am J Surg. 2010 May;199(5):676-9. doi: 10.1016/j.amjsurg.2010.01.013.
- Background: PubMed 18206449Modi BP, Langer M, Ching YA, Valim C, Waterford SD, Iglesias J, Duro D, Lo C, Jaksic T, Duggan C. Improved survival in a multidisciplinary short bowel syndrome program. J Pediatr Surg. 2008 Jan;43(1):20-4. doi: 10.1016/j.jpedsurg.2007.09.014.
- Background: PubMed 26078213Mutanen A, Kosola S, Merras-Salmio L, Kolho KL, Pakarinen MP. Long-term health-related quality of life of patients with pediatric onset intestinal failure. J Pediatr Surg. 2015 Nov;50(11):1854-8. doi: 10.1016/j.jpedsurg.2015.05.012. Epub 2015 Jun 3.
- Background: PubMed 29478887Norsa L, Artru S, Lambe C, Talbotec C, Pigneur B, Ruemmele F, Colomb V, Capito C, Chardot C, Lacaille F, Goulet O. Long term outcomes of intestinal rehabilitation in children with neonatal very short bowel syndrome: Parenteral nutrition or intestinal transplantation. Clin Nutr. 2019 Apr;38(2):926-933. doi: 10.1016/j.clnu.2018.02.004. Epub 2018 Feb 15.
- Background: PubMed 23648789Sanchez SE, McAteer JP, Goldin AB, Horslen S, Huebner CE, Javid PJ. Health-related quality of life in children with intestinal failure. J Pediatr Gastroenterol Nutr. 2013 Sep;57(3):330-4. doi: 10.1097/MPG.0b013e3182999961.
- Background: PubMed 17634123Varni JW, Limbers CA, Burwinkle TM. Impaired health-related quality of life in children and adolescents with chronic conditions: a comparative analysis of 10 disease clusters and 33 disease categories/severities utilizing the PedsQL 4.0 Generic Core Scales. Health Qual Life Outcomes. 2007 Jul 16;5:43. doi: 10.1186/1477-7525-5-43.
- Derived from this study: PubMed 40158842Modi BP, Piper HG, Belza C, Staffa S, Arnold MA, Boctor DL, Channabasappa N, Cohran VC, Galloway DP, Sudan D, Wales PW, Warner BW, Murtadi G, Javid PJ. Health-Related Quality of Life in Pediatric Intestinal Failure from Infancy to Adolescence: An International, Multi-Center Evaluation. J Pediatr. 2025 Jul;282:114566. doi: 10.1016/j.jpeds.2025.114566. Epub 2025 Mar 28.
- 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.
Patrick J Javid, MD principal investigator
Seattle Children's Hospital
Biren P Modi, MD MPH principal investigator
Boston Children's Hospital
14 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pediatric Intestinal Failure, run by Boston Children's Hospital at 14 sites in 2 countries.
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 5 inclusion and 7 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: looking for participants now, as of the sponsor's last update on 23 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Canada. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Biren P Modi, MD MPH (857-218-4651, biren.modi@childrens.harvard.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Health-related quality of life (hrQOL) (time frame: Baseline). The study lists 4 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 NCT04629014, which also lists 8 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 23 Mar 2026; AzTrials last refreshed its copy on 7 Nov 2026.