Prospective Multisite Study of Quality of Life in Pediatric Intestinal Failure

NCT ID: NCT04629014

Last Updated: 2024-12-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Total Enrollment

750 participants

Study Classification

OBSERVATIONAL

Study Start Date

2020-02-27

Study Completion Date

2027-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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

See the medical conditions and disease areas that this research is targeting or investigating.

Pediatric Intestinal Failure

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

intestinal failure short bowel syndrome short gut syndrome parenteral nutrition specialized nutrition

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* 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

Exclusion Criteria

* 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
Minimum Eligible Age

6 Months

Maximum Eligible Age

25 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Seattle Children's Hospital

OTHER

Sponsor Role collaborator

University of Alabama at Birmingham

OTHER

Sponsor Role collaborator

Ann & Robert H Lurie Children's Hospital of Chicago

OTHER

Sponsor Role collaborator

Washington University School of Medicine

OTHER

Sponsor Role collaborator

University of Michigan

OTHER

Sponsor Role collaborator

Duke Health

OTHER

Sponsor Role collaborator

University of Texas Southwestern Medical Center

OTHER

Sponsor Role collaborator

Alberta Children's Hospital

OTHER

Sponsor Role collaborator

British Columbia Children's Hospital

OTHER

Sponsor Role collaborator

The Hospital for Sick Children

OTHER

Sponsor Role collaborator

Boston Children's Hospital

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Biren Modi

Surgical Director, Center for Advanced Intestinal Rehabilitation

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Patrick J Javid, MD

Role: PRINCIPAL_INVESTIGATOR

Seattle Children's Hospital

Biren P Modi, MD MPH

Role: PRINCIPAL_INVESTIGATOR

Boston Children's Hospital

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Children's of Alabama

Birmingham, Alabama, United States

Site Status NOT_YET_RECRUITING

Lurie Children's Hospital

Chicago, Illinois, United States

Site Status RECRUITING

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

C.S. Mott Children's Hospital

Ann Arbor, Michigan, United States

Site Status RECRUITING

St. Louis Children's Hospital

St Louis, Missouri, United States

Site Status RECRUITING

Duke Children's Hospital

Durham, North Carolina, United States

Site Status RECRUITING

Dallas Children's Hospital

Dallas, Texas, United States

Site Status RECRUITING

Seattle Children's Hospital

Seattle, Washington, United States

Site Status RECRUITING

Alberta Children's Hospital

Calgary, Alberta, Canada

Site Status RECRUITING

BC Children's Hospital

Vancouver, British Columbia, Canada

Site Status RECRUITING

Hospital for Sick Children

Toronto, Ontario, Canada

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

United States Canada

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Biren P Modi, MD MPH

Role: CONTACT

Phone: 857-218-4651

Email: [email protected]

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

David Galloway, MD

Role: primary

Valeria Cohran, MD

Role: primary

Biren P Modi, MD MPH

Role: primary

Meghan Arnold, MD

Role: primary

Brad Warner, MD

Role: primary

Debra Sudan, MD

Role: primary

Nandini Channabasappa, MD

Role: primary

Patrick J Javid, MD

Role: primary

Dana Boctor, MD

Role: primary

Hannah Piper, MD

Role: primary

Paul Wales, MD

Role: primary

References

Explore related publications, articles, or registry entries linked to this study.

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.

Reference Type BACKGROUND
PMID: 17502188 (View on PubMed)

Javid 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.

Reference Type BACKGROUND
PMID: 20466115 (View on PubMed)

Modi 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.

Reference Type BACKGROUND
PMID: 18206449 (View on PubMed)

Mutanen 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.

Reference Type BACKGROUND
PMID: 26078213 (View on PubMed)

Norsa 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.

Reference Type BACKGROUND
PMID: 29478887 (View on PubMed)

Sanchez 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.

Reference Type BACKGROUND
PMID: 23648789 (View on PubMed)

Varni 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.

Reference Type BACKGROUND
PMID: 17634123 (View on PubMed)

Modi 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.

Reference Type DERIVED
PMID: 40158842 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

P00032389

Identifier Type: -

Identifier Source: org_study_id