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Prospective Multisite Study of Quality of Life in Pediatric Intestinal Failure

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ClinicalTrials.gov Identifier: NCT04629014
Recruitment Status : Recruiting
First Posted : November 16, 2020
Last Update Posted : November 16, 2020
Sponsor:
Collaborators:
Seattle Children's Hospital
University of Alabama at Birmingham
Lurie Children's Hospital of Chicago
Washington University School of Medicine
University of Michigan
Duke Health
University of California, Los Angeles
University of Texas Southwestern Medical Center
Alberta Children's Hospital
British Columbia Children's Hospital
The Hospital for Sick Children
Information provided by (Responsible Party):
Biren Modi, Boston Children's Hospital

Brief 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 care of these participants.

Condition or disease
Pediatric Intestinal Failure

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.

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Study Type : Observational
Estimated Enrollment : 400 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Prospective Multisite Study of Quality of Life in Pediatric Intestinal Failure
Actual Study Start Date : February 27, 2020
Estimated Primary Completion Date : December 2025
Estimated Study Completion Date : December 2025

Resource links provided by the National Library of Medicine





Primary Outcome Measures :
  1. 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

  2. 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

  3. 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

  4. 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


Secondary Outcome Measures :
  1. Family Impact [ Time Frame: Baseline ]
    Family impact as determined by the PedsQL Family Impact Module

  2. Family Impact [ Time Frame: Trend over 5 years ]
    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



Information from the National Library of Medicine

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Ages Eligible for Study:   6 Months to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
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.
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04629014


Contacts
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Contact: Biren P Modi, MD MPH 857-218-4651 biren.modi@childrens.harvard.edu

Locations
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United States, Alabama
Children's of Alabama Not yet recruiting
Birmingham, Alabama, United States, 35233
Contact: David Galloway, MD       dgalloway@peds.uab.edu   
Principal Investigator: David Galloway, MD         
United States, California
UCLA Mattel Children's Hospital Recruiting
Los Angeles, California, United States, 90095
Contact: Robert Venick, MD       rvenick@mednet.ucla.edu   
Principal Investigator: Robert Venick, MD         
United States, Illinois
Lurie Children's Hospital Recruiting
Chicago, Illinois, United States, 60611
Contact: Valeria Cohran, MD       vcohran@luriechildrens.org   
Principal Investigator: Valeria Cohran, MD         
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Biren P Modi, MD MPH    857-218-4651    biren.modi@childrens.harvard.edu   
Principal Investigator: Biren P Modi, MD MPH         
United States, Michigan
C.S. Mott Children's Hospital Recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Meghan Arnold, MD       meghanar@med.umich.edu   
Principal Investigator: Meghan Arnold, MD         
United States, Missouri
St. Louis Children's Hospital Recruiting
Saint Louis, Missouri, United States, 63100
Contact: Brad Warner, MD       brad.warner@wustl.edu   
Principal Investigator: Brad Warner, MD         
United States, North Carolina
Duke Children's Hospital Recruiting
Durham, North Carolina, United States, 27710
Contact: Debra Sudan, MD       debra.sudan@duke.edu   
Principal Investigator: Debra Sudan, MD         
United States, Texas
Dallas Children's Hospital Recruiting
Dallas, Texas, United States, 75390
Contact: Nandini Channabasappa, MD       nandini.channabasappa@utsouthwestern.edu   
Principal Investigator: Nandini Channabasappa, MD         
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Patrick J Javid, MD       patrick.javid@seattlechildrens.org   
Principal Investigator: Patrick J Javid, MD         
Canada, Alberta
Alberta Children's Hospital Recruiting
Calgary, Alberta, Canada, T3B 6A8
Contact: Dana Boctor, MD       dana.boctor@albertahealthservices.ca   
Principal Investigator: Dana Boctor, MD         
Canada, British Columbia
BC Children's Hospital Recruiting
Vancouver, British Columbia, Canada, V6H 3N1
Contact: Hannah Piper, MD       hannah.piper@cw.bc.ca   
Principal Investigator: Hannah Piper, MD         
Canada, Ontario
Hospital for Sick Children Recruiting
Toronto, Ontario, Canada, M5G 1X8
Contact: Paul Wales, MD       paul.wales@sickkids.ca   
Principal Investigator: Paul Wales, MD         
Sponsors and Collaborators
Boston Children's Hospital
Seattle Children's Hospital
University of Alabama at Birmingham
Lurie Children's Hospital of Chicago
Washington University School of Medicine
University of Michigan
Duke Health
University of California, Los Angeles
University of Texas Southwestern Medical Center
Alberta Children's Hospital
British Columbia Children's Hospital
The Hospital for Sick Children
Investigators
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Principal Investigator: Patrick J Javid, MD Seattle Children's Hospital
Principal Investigator: Biren P Modi, MD MPH Boston Children's Hospital
Publications:

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Responsible Party: Biren Modi, Associate Surgical Director, Center for Advanced Intestinal Rehabilitation, Boston Children's Hospital
ClinicalTrials.gov Identifier: NCT04629014    
Other Study ID Numbers: P00032389
First Posted: November 16, 2020    Key Record Dates
Last Update Posted: November 16, 2020
Last Verified: November 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Biren Modi, Boston Children's Hospital:
intestinal failure
short bowel syndrome
short gut syndrome
parenteral nutrition
specialized nutrition