Pediatric cGVHD Symptom Scale
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ClinicalTrials.gov Identifier: NCT04044365 |
Recruitment Status :
Recruiting
First Posted : August 5, 2019
Last Update Posted : January 30, 2023
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Background:
Some children/adolescents who have had a stem cell transplant live with chronic graft-versus-host-disease (cGVHD). cGVHD is a side effect of the transplant that can cause multiple bothersome symptoms and negatively affect a child/adolescent squality of daily life. The questionnaires that measure thesymptoms caused by cGVHD are designed for adults. Children/adolescents may not describe their symptoms in the same way. The goal of this research is to improve the way we measure how bothersome these symptoms are for children/adolescents living with cGVHD.
Objective:
To develop a questionnaire (The Pediatric cGHVD Symptom Scale) for children/adolescents living with cGVHD to identify the symptoms they are experiencing and describe how bothersome those symptoms are to them. An additional goal is to design a parent/guardian companion questionnaire that can be used to capture the symptom experiences of very young children who may not be able to complete a questionnaire.
Eligibility:
Children/adolescents ages 5-17 who are receiving treatment for cGVHD after a stem cell transplant, and their parent/guardian..
Design:
This study consists of 2 projects.
Children/adolescents with cGVHD and their parent/guardianparticipants will be grouped by the child/adolescent s age: 5-7, 8-12, and 13-17.
In project 1, participants will complete an age-appropriate questionnaire about cGVHD symptoms. The questionnaire will ask about the child/adolescent s physical functioning and emotional well-being. The parent/guardian will out fill out a companion questionnaire online. The child/adolescent will then review their completed questionnaire during an interview with a researcher and will be asked whether the questions about their symptoms were difficult to understand. The parent/guardian and child/adolescent will then be interviewed together to further explore their responses to the questionnaires. Interviews will be done in person, by phone, and online. . Based on what is learned through these interviews, the wording of the questionnaire will be improved for better comprehension and ease of response.
In project 2, participants will complete this revised questionnairefor their age group along with some other questionnaires that ask about quality of life. Both the child/adolescent and parent/guardian will fill out the questionnaires online at three separate time points.
In both projects, children/adolescents with cGVHD and their parent/guardian participants will be grouped by the child/adolescent s age: 5-7, 8-12, and 13-17.
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Condition or disease |
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Graft vs Host Disease |

Study Type : | Observational |
Estimated Enrollment : | 390 participants |
Observational Model: | Cohort |
Time Perspective: | Prospective |
Official Title: | Development and Psychometric Testing of a Pediatric Chronic Graft- Versus-Host Disease (GVHD) Symptom Scale (PCSS) |
Actual Study Start Date : | October 2, 2019 |
Estimated Primary Completion Date : | April 1, 2024 |
Estimated Study Completion Date : | June 1, 2024 |

Group/Cohort |
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1/Project 1 Child/caregiver-proxy
Children age 5-7 with cGVHD and their caregiver, n=20 child/parent dyads
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2/Project 1 Child/caregiver-proxy
Children age 8-12 with cGVHD and their caregiver, n=20 child/parent pairs
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3/Project 1 Child/caregiver-proxy
Children age 13-17 with cGVHD and their caregiver, n=35 child/parent pairs
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4/Project 2 Child/caregiver-proxy
Children age 5-7 with cGVHD and their caregiver, n=40 child/parent pairs
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5/Project 2 Child/caregiver-proxy
Children age 8-12 with cGVHD and their caregiver, n=40 child/parent pairs
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6/Project 2 Child/caregiver-proxy
Children age 13-17 with cGVHD and their caregiver, n=40 child/parent pairs
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- Feasability [ Time Frame: 3 years ]To develop a psychometrically valid Pediatric cGVHD Symptom Scale (PCSS) and a companion parent-proxy measure as counterparts to the Lee cGVHD Symptom Scale.

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Ages Eligible for Study: | 5 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | Yes |
Sampling Method: | Non-Probability Sample |
- INCLUSION CRITERIA:
Pediatric Subject Inclusion Criteria:
- Children aged 5 to 17 years old, who have undergone prior allogeneic stem cell transplant
- Clinical diagnosis of cGVHD
- Currently receiving systemic treatment for cGVHD (including phototherapies), or has had systemic therapy for cGVHD tapered to discontinuation within the past 12 months
- No evidence of malignant disease relapse including molecular relapse and minimal residual disease. Patients with mixed chimerism are eligible to participate
- Subject must have an eligible caregiver proxy who is willing to participate in the study.
- Parent or guardian ability and willingness to sign a written informed consent document
- Subjects must be able to comprehend and speak the English language
- Subjects may participate in both Project 1 and Project 2 of the study. Participation in Project 1 is not required in order to be eligible to participate in Project 2.
Caregiver Proxy Inclusion Criteria
- Adult, >18 years of age, caregiver of participating subject
- Must be willing and able to provide informed consent.
- Must be able to comprehend and speak the English language
EXCLUSOIN CRITERIA:
Patients may be excluded from this study if in the judgment of the Principal or Associate Investigator, the subject is too ill, or subject s cognitive ability would compromise their ability to participate in study related procedures.

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): NCT04044365
Contact: Lori Wiener, Ph.D. | (240) 760-6419 | lori.wiener@nih.gov |
United States, Colorado | |
Children's Hospital Colorado (CHCO) | Recruiting |
Aurora, Colorado, United States, 80045 | |
Contact: Amy Keating, M.D. 720-777-0166 Amy.Keating@UCDenver.edu | |
United States, District of Columbia | |
Children s National Medical Center (CNMC) | Recruiting |
Washington, District of Columbia, United States, 20010 | |
Contact: Alexa Yarish 202-476-6250 ajyarish@childrensnational.org | |
United States, Georgia | |
Children's Healthcare of Atlanta - Egleston Hospital | Recruiting |
Atlanta, Georgia, United States, 30322 | |
Contact: Katherine K. Garrett, MPH, ELS 404-785-3644 Katherine.Garrett@choa.org | |
United States, Illinois | |
Lurie Children's Hospital of Chicago | Recruiting |
Chicago, Illinois, United States, 60611 | |
Contact: Anne Lakes 312-227-1876 alakes@luriechildrens.org | |
United States, Maryland | |
Johns Hopkins Hospital (JHH) | Recruiting |
Baltimore, Maryland, United States, 21287 | |
Contact: Kenneth R. Cook, M.D. 410-955-8751 kcooke5@jhmi.edu | |
National Institutes of Health Clinical Center | Recruiting |
Bethesda, Maryland, United States, 20892 | |
Contact: For more information at the NIH Clinical Center contact National Cancer Institute Referral Office 888-624-1937 | |
United States, Minnesota | |
University of Minnesota | Recruiting |
Minneapolis, Minnesota, United States, 55455 | |
Contact: Margaret MacMillan, M.D. 612-626-2961 macmi002@umn.edu | |
United States, Tennessee | |
St. Jude Children s Research Hospital | Recruiting |
Memphis, Tennessee, United States, 38105 | |
Contact: Marian Shaw 901-595-8149 Marian.Shaw@stjude.org | |
United States, Texas | |
Texas Children's Hospital (TCH)-Baylor | Recruiting |
Houston, Texas, United States, 77030 | |
Contact: Erin Doherty, M.D. 832-826-0860 eedohert@texaschildrens.org | |
The University of Texas MD Anderson Cancer Institute | Recruiting |
Houston, Texas, United States, 77030 | |
Contact: Alba Rubi Banegas 713-792-9857 aarubi@mdanderson.org | |
United States, Washington | |
Fred Hutchinson Cancer Center (FHCC) | Recruiting |
Seattle, Washington, United States, 98109 | |
Contact: Sheri Ballard 206-667-5160 sballard@fredhutch.org | |
Canada | |
Children's & Women's Health Centre of British Columbia (CWHC of BC) | Recruiting |
Vancouver BC, Canada | |
Contact: Colleen Fitzgerald (604) 875-2345 cfitzgerald@cw.bc.ca |
Principal Investigator: | Lori Wiener, Ph.D. | National Cancer Institute (NCI) |
Responsible Party: | National Cancer Institute (NCI) |
ClinicalTrials.gov Identifier: | NCT04044365 |
Other Study ID Numbers: |
190131 19-C-0131 |
First Posted: | August 5, 2019 Key Record Dates |
Last Update Posted: | January 30, 2023 |
Last Verified: | January 26, 2023 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Plan Description: | .All IPD recorded in the medical record will be shared with intramural investigators upon request. |
Supporting Materials: |
Study Protocol Statistical Analysis Plan (SAP) Informed Consent Form (ICF) |
Time Frame: | Clinical data available during the study and indefinitely. |
Access Criteria: | Clinical data will be made available via subscription to BTRIS and with the permission of the study PI. |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
Hematopoietic Stem Cell Transplantation (HSCT) Allogeneic Stem Cell Transplant Phototherapies Graft Vs Host Disease Natural History |
Graft vs Host Disease Immune System Diseases |