Chronic Graft-Versus-Host-DiseaseNCT01036958
Development and Validation of a Symptom Scale for Children With Chronic Graft-versus-Host Disease
An observational study of Chronic Graft-Versus-Host-Disease, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
Background: Chronic graft-vs-host disease (GVHD) is an important cause of morbidity and mortality in patients undergoing allogeneic bone marrow transplantation. The symptoms of chronic GVHD are similar to those of other autoimmune diseases, and treatment for the pain often involves steroid use that can cause severe… Read more about this study
- Who is it for?
- Ages 5 to 18 years
- Conditions the study is for
- Chronic Graft-Versus-Host-Disease
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Nov 2009to 6 Feb 2018
- 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 5 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Children of 5 to 18 years of age, who have undergone prior allogeneic stem cell transplant.
- Clinical diagnosis of chronic GVHD with need for systemic treatment.
- No evidence of primary disease relapse.
- Must be willing to sign informed consent, or if applicable, child assent.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: Development and Validation of a Symptom Scale for Children With Chronic Graft-Versus-Host Disease
Background:
- Chronic graft-vs-host disease (GVHD) is an important cause of morbidity and mortality in patients undergoing allogeneic bone marrow transplantation. The symptoms of chronic GVHD are similar to those of other autoimmune diseases, and treatment for the pain often involves steroid use that can cause severe side effects over the long term.
- At present, there is no research instrument that measures symptoms in children with chronic GVHD. Treatment practitioners may use one of several pediatric quality of life questionnaires, but because none of these is specific for chronic GVHD each instrument has potential gaps in its ability to assess the full spectrum of problems experienced by children with chronic GVHD. Researchers are interested in developing a better understanding of the disease burden experienced by children and adolescents with chronic GVHD.
Read more
Objectives:
- To develop a Pediatric Chronic GVHD Symptom Scale (PCSS) that reliably measures the disease-specific burden of chronic GVHD in children.
Eligibility:
- Children and adolescents 5 to 18 years of age who have undergone prior allogeneic stem cell transplant and have been diagnosed with chronic GVHD that requires treatment.
Design:
- There are two phases to the study; participants will enroll in phase I (question generation) at this time.
- Researchers will interview participants and ask open-ended questions (requiring more than a one- or two-word response) about symptoms that adults with chronic GVHD have found problematic.
- Both parents and children will participate in the interviews, which will be audio-recorded. Depending on the child or adolescent s age, the interviews may be conducted together with the parents or separately.
- No treatment will be given as part of this study.
Read the detailed description
Background:
- Currently, a large number of children with chronic GVHD have to deal with many years of a disfiguring and painful chronic illness with the side effects of long term steroid use.
- The broad categories of limited and extensive chronic GVHD are recognized by clinicians, but are not particularly useful in clinical practice. Chronic GVHD may involve almost every organ although it most commonly affects skin, eyes, mouth, liver, intestines, lung and musculoskeletal system.
- Recently, there has been a large effort through the NIH Consensus for chronic GVHD to standardize response criteria for patients with chronic GVHD, on clinical trials. Because of the absence of evidence suggesting which response criteria truly correlates with improvement, the NIH Consensus Panel has recommended following not only physical signs of chronic GVHD, but also symptoms of GVHD.
Primary Objective:
- Develop a Pediatric Chronic GVHD Symptom Scale (PCSS) that reliably measures the disease specific burden of chronic GVHD in children.
Secondary Objectives:
- Correlate high and low scores on the PCSS with standard quality of life measures.
- Determine using the Rasch measurement model whether the PCSS has sufficient sensitivity to change for the scale to be useful in clinical intervention trials.
Eligibility:
- Children of 5 to 18 years of age, who have undergone prior allogeneic stem cell transplant
- Clinical diagnosis of chronic GVHD with need for systemic treatment
- No evidence of primary disease relapse
- Must be willing to sign informed consent, or if applicable, child assent
Design:
- With the final goal of developing a scale that is similar in design to the Lee Scale but which measures the symptom burden more specifically for children with chronic GVHD, the study will be conducted in two phases: item generation and psychometric validation.
- In phase I, the local team at each institution will interview participants using a script to identify symptom concerns for pediatric chronic GVHD patients. Based on interviews, we will decide whether to test different scales for the different age groups, or a single scale for all.
- Data from Phase II will be used to finalize and validate the pediatric symptom scale, through the assessment of test-retest characteristics, use of item reduction, examination of construct validity, internal consistency, convergent and divergent validity, and evaluation of sensitivity to change.
- Conditions
- Chronic Graft-Versus-Host-Disease
- Keywords
- Pediatric cGVHD, Response Criteria, Symptoms, Quality of Life, Graft-Versus-Host Disease, GVHD, Pediatric GVHD
- Medical subject headings
- Bronchiolitis Obliterans Syndrome, Graft vs Host Disease
What is being measured
The primary outcome is the main question the study is designed to answer.
Develop pediatric cGVHD symptom scale
Measured: 24 months
Secondary outcomes (2)
Rasch measurement model, determine sensitivity to use in clinicalintervention trials
Measured: 24 months
Correlate scores with QOL measures
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Dec 2009 (estimated)
- Study start25 Nov 2009
- Study completion6 Feb 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Chronic Graft-Versus-Host-DiseaseStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The 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
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Lori Wiener, Ph.D., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01036958
- Sponsor's protocol ID
- 100019
- Other IDs
- 10-C-0019
- Status last verified
- 6 Feb 2018
- First submitted
- 18 Dec 2009
- Last updated
- 26 Sep 2018
- Results posted
- No
Publications
Champlin RE, Gale RP. The early complications of bone marrow transplantation. Semin Hematol. 1984 Apr;21(2):101-8. No abstract available.
PubMed 6377500Thomas E, Storb R, Clift RA, Fefer A, Johnson FL, Neiman PE, Lerner KG, Glucksberg H, Buckner CD. Bone-marrow transplantation (first of two parts). N Engl J Med. 1975 Apr 17;292(16):832-43. doi: 10.1056/NEJM197504172921605. No abstract available.
PubMed 234595Sullivan KM, Parkman R. The pathophysiology and treatment of graft-versus-host disease. Clin Haematol. 1983 Oct;12(3):775-89. doi: 10.1016/s0308-2261(83)80010-1.
PubMed 6315285
Study at a glance
From the registry record, last updated by the sponsor on 26 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Development and Validation of a Symptom Scale for Children With Chronic Graft-Versus-Host Disease
- ClinicalTrials.gov ID
- NCT01036958
- Acronym / sponsor ID
- 100019
- Conditions
- Chronic Graft-Versus-Host-Disease
- Keywords
- Pediatric cGVHD, Response Criteria, Symptoms, Quality of Life, Graft-Versus-Host Disease, GVHD, Pediatric GVHD
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 25 Nov 2009
- Study completion
- 6 Feb 2018
- First posted
- 21 Dec 2009 (estimated)
- 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:
- Chronic graft-vs-host disease (GVHD) is an important cause of morbidity and mortality in patients undergoing allogeneic bone marrow transplantation. The symptoms of chronic GVHD are similar to those of other autoimmune diseases, and treatment for the pain often involves steroid use that can cause severe side effects over the long term.
- At present, there is no research instrument that measures symptoms in children with chronic GVHD. Treatment practitioners may use one of several pediatric quality of life questionnaires, but because none of these is specific for chronic GVHD each instrument has potential gaps in its ability to assess the full spectrum of problems experienced by children with chronic GVHD. Researchers are interested in developing a better understanding of the disease burden experienced by children and adolescents with chronic GVHD.
Objectives:
- To develop a Pediatric Chronic GVHD Symptom Scale (PCSS) that reliably measures the disease-specific burden of chronic GVHD in children.
Eligibility:
- Children and adolescents 5 to 18 years of age who have undergone prior allogeneic stem cell transplant and have been diagnosed with chronic GVHD that requires treatment.
Design:
- There are two phases to the study; participants will enroll in phase I (question generation) at this time.
- Researchers will interview participants and ask open-ended questions (requiring more than a one- or two-word response) about symptoms that adults with chronic GVHD have found problematic.
- Both parents and children will participate in the interviews, which will be audio-recorded. Depending on the child or adolescent s age, the interviews may be conducted together with the parents or separately.
- No treatment will be given as part of this study.
Detailed description
Background:
- Currently, a large number of children with chronic GVHD have to deal with many years of a disfiguring and painful chronic illness with the side effects of long term steroid use.
- The broad categories of limited and extensive chronic GVHD are recognized by clinicians, but are not particularly useful in clinical practice. Chronic GVHD may involve almost every organ although it most commonly affects skin, eyes, mouth, liver, intestines, lung and musculoskeletal system.
- Recently, there has been a large effort through the NIH Consensus for chronic GVHD to standardize response criteria for patients with chronic GVHD, on clinical trials. Because of the absence of evidence suggesting which response criteria truly correlates with improvement, the NIH Consensus Panel has recommended following not only physical signs of chronic GVHD, but also symptoms of GVHD.
Primary Objective:
- Develop a Pediatric Chronic GVHD Symptom Scale (PCSS) that reliably measures the disease specific burden of chronic GVHD in children.
Secondary Objectives:
- Correlate high and low scores on the PCSS with standard quality of life measures.
- Determine using the Rasch measurement model whether the PCSS has sufficient sensitivity to change for the scale to be useful in clinical intervention trials.
Eligibility:
- Children of 5 to 18 years of age, who have undergone prior allogeneic stem cell transplant
- Clinical diagnosis of chronic GVHD with need for systemic treatment
- No evidence of primary disease relapse
- Must be willing to sign informed consent, or if applicable, child assent
Design:
- With the final goal of developing a scale that is similar in design to the Lee Scale but which measures the symptom burden more specifically for children with chronic GVHD, the study will be conducted in two phases: item generation and psychometric validation.
- In phase I, the local team at each institution will interview participants using a script to identify symptom concerns for pediatric chronic GVHD patients. Based on interviews, we will decide whether to test different scales for the different age groups, or a single scale for all.
- Data from Phase II will be used to finalize and validate the pediatric symptom scale, through the assessment of test-retest characteristics, use of item reduction, examination of construct validity, internal consistency, convergent and divergent validity, and evaluation of sensitivity to change.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
Who may qualify
- Ages
- 5 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Children of 5 to 18 years of age, who have undergone prior allogeneic stem cell transplant.
- Clinical diagnosis of chronic GVHD with need for systemic treatment.
- No evidence of primary disease relapse.
- Must be willing to sign informed consent, or if applicable, child assent.
Key exclusion criteria (0 listed)
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
Develop pediatric cGVHD symptom scale
Time frame: 24 months
2 secondary outcomes
Rasch measurement model, determine sensitivity to use in clinicalintervention trials
Time frame: 24 months
Correlate scores with QOL measures
Time frame: 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Sep 2018.
- Background: PubMed 6377500Champlin RE, Gale RP. The early complications of bone marrow transplantation. Semin Hematol. 1984 Apr;21(2):101-8. No abstract available.
- Background: PubMed 234595Thomas E, Storb R, Clift RA, Fefer A, Johnson FL, Neiman PE, Lerner KG, Glucksberg H, Buckner CD. Bone-marrow transplantation (first of two parts). N Engl J Med. 1975 Apr 17;292(16):832-43. doi: 10.1056/NEJM197504172921605. No abstract available.
- Background: PubMed 6315285Sullivan KM, Parkman R. The pathophysiology and treatment of graft-versus-host disease. Clin Haematol. 1983 Oct;12(3):775-89. doi: 10.1016/s0308-2261(83)80010-1.
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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.
Lori Wiener, Ph.D. principal investigator
National Cancer Institute (NCI)
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 Chronic Graft-Versus-Host-Disease, run by National Cancer Institute (NCI) at 1 site in United States.
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 4 inclusion and 0 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: finished, as of the sponsor's last update on 26 Sep 2018. 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 United States. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Develop pediatric cGVHD symptom scale (time frame: 24 months). 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 NCT01036958, which also lists 3 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 26 Sep 2018.