Graft vs Host DiseasecGVHDNCT00632658
Development and Validation of a Symptom Scale for Children With Chronic Graft-versus-Host Disease
An observational study of Graft vs Host Disease, run by Ann & Robert H Lurie Children's Hospital of Chicago at 1 site in United States.
Trial summary
Chronic Graft-versus-Host Disease (cGVHD) is an important cause of morbidity and mortality in patients undergoing allogeneic bone marrow transplantation. cGVHD usually occurs after 100 days following transplantation and develops in 20-60% of transplant recipients. Read more about this study
- Who is it for?
- Ages 5 to 18 years
- Conditions the study is for
- Graft vs Host Disease
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2008to Jul 2011
- 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
Study population: Patients with cGVHD will be identified by their local physican and asked to participate in this research study. A total of 24 children will be asked to participate in this study. The parents of all of the 24 children will also be asked to participate and give us their feed-back on what it is like to cope with a child with cGVHD.
Who may be able to join
- 5-18 years of age
- Prior allogeneic Stem Cell Transplant, with any graft source, donor type, and GVHD prophylaxis allowed
- Clinical diagnosis of cGVHD
- Need for systemic treatment, defined as any medication or intervention delivered
- No evidence of primary disease relapse
- Signed, informed consent, and if applicable, adolescent assent
Who cannot join
- Inability to give signed informed consent
Trial locations and contacts
1 site in 1 country.
United States1 site
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
About this study
Official title: Development and Validation of a Symptoms Scale for Children With Chronic Graft-versus-Host Disease
Chronic Graft-versus-Host Disease (cGVHD) is an important cause of morbidity and mortality in patients undergoing allogeneic bone marrow transplantation. cGVHD usually occurs after 100 days following transplantation and develops in 20-60% of transplant recipients. The incidence of cGVHD varies depending on the age of the marrow recipient, the use of sibling or unrelated donor bone marrow, the use of unmanipulated T cell-depleted bone marrow, and perhaps other factors. Clinically, cGVHD is characterized by multi-system disease, which frequently mimics the clinical features of autoimmune diseases. The manifestations include skin changes (lichenoid and sclerodermatous changes, changes in pigmentation, loss of accessory structures such as hair, dystrophic nails, and rash), joint contractures, severe cramping, hepatic dysfunctions, sicca syndrome, obstructive lung disease, esophageal dysmotility, weight loss, polyserositis, immunodeficiency, and autoantibodies including anti-nuclear antibody, anti-erythrocyte antibodies, and anti-platelet antibodies.
Read the detailed description
A large number of children with cGVHD have to deal with many years of a disfiguring and painful chronic illness with the side effects of long term steroid use. The number of stem cell transplants done in children is only growing given that we are now transplanting patients with a variety of nonmalignant disorders and given the use of alternative donor sources. The broad categories of limited and extensive cGVHD are recognized by clinicians, but are not particularly useful in clinical practice. Since cGVHD may involve almost every organ system adn since cGVHD constitutes a waxing and waning nature, cGVHD makes clinical management very difficult and complicated. Currently, there is a symptoms scale used in the adult population for measuring symptom burden for adults with cGVHD. This scale is called the "Lee Symptoms Scale". The purpose of this project is to develop a scale that is similar in design to the Lee Scale, but it is specifically designed to measure the burden of cGVHD in the pediatric population
- Conditions
- Graft vs Host Disease
- Keywords
- cGVHD
- Medical subject headings
- Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Patients with cGVHD | Pediatric Patients with cGVHD will be asked to participate in an interview with their Physician. The interview will ask the pediatric patients questions about their cGVHD. The interview will be audio-recorded. |
What is being measured
The primary outcome is the main question the study is designed to answer.
To develop a Pediatric Chronic GVHD Symptoms Scale (PCSS) that reliably measures the disease specific burden of chronic GVHD in children
Measured: One Day Interview will be performed
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Mar 2008 (estimated)
- Study startJan 2008
- Primary completionJul 2011
- Study completionJul 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Graft vs Host DiseaseStudies of the same condition, open and under way.
- 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
- Prospective
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Reggie E Duerst, MD, Principal investigator, Ann & Robert H Lurie Children's Hospital of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00632658
- Sponsor's protocol ID
- SCT 0208B
- Acronym
- cGVHD
- Status last verified
- Oct 2019
- First submitted
- 4 Mar 2008
- Last updated
- 8 Oct 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Development and Validation of a Symptoms Scale for Children With Chronic Graft-versus-Host Disease
- ClinicalTrials.gov ID
- NCT00632658
- Acronym / sponsor ID
- cGVHD / SCT 0208B
- Conditions
- Graft vs Host Disease
- Keywords
- cGVHD
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of ChicagoUniversity, hospital or other
- Collaborators
- University of Minnesota, Dana-Farber Cancer Institute, Fred Hutchinson Cancer Center
- Study start
- Jan 2008
- Primary completion
- Jul 2011 (actual)
- Study completion
- Jul 2011 (actual)
- First posted
- 11 Mar 2008 (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.
Chronic Graft-versus-Host Disease (cGVHD) is an important cause of morbidity and mortality in patients undergoing allogeneic bone marrow transplantation. cGVHD usually occurs after 100 days following transplantation and develops in 20-60% of transplant recipients. The incidence of cGVHD varies depending on the age of the marrow recipient, the use of sibling or unrelated donor bone marrow, the use of unmanipulated T cell-depleted bone marrow, and perhaps other factors. Clinically, cGVHD is characterized by multi-system disease, which frequently mimics the clinical features of autoimmune diseases. The manifestations include skin changes (lichenoid and sclerodermatous changes, changes in pigmentation, loss of accessory structures such as hair, dystrophic nails, and rash), joint contractures, severe cramping, hepatic dysfunctions, sicca syndrome, obstructive lung disease, esophageal dysmotility, weight loss, polyserositis, immunodeficiency, and autoantibodies including anti-nuclear antibody, anti-erythrocyte antibodies, and anti-platelet antibodies.
Detailed description
A large number of children with cGVHD have to deal with many years of a disfiguring and painful chronic illness with the side effects of long term steroid use. The number of stem cell transplants done in children is only growing given that we are now transplanting patients with a variety of nonmalignant disorders and given the use of alternative donor sources. The broad categories of limited and extensive cGVHD are recognized by clinicians, but are not particularly useful in clinical practice. Since cGVHD may involve almost every organ system adn since cGVHD constitutes a waxing and waning nature, cGVHD makes clinical management very difficult and complicated. Currently, there is a symptoms scale used in the adult population for measuring symptom burden for adults with cGVHD. This scale is called the "Lee Symptoms Scale". The purpose of this project is to develop a scale that is similar in design to the Lee Scale, but it is specifically designed to measure the burden of cGVHD in the pediatric population
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with cGVHD | – | –Pediatric Patients with cGVHD will be asked to participate in an interview with their Physician. The interview will ask the pediatric patients questions about their cGVHD. The interview will be audio-recorded. |
Who may qualify
- Ages
- 5 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients with cGVHD will be identified by their local physican and asked to participate in this research study. A total of 24 children will be asked to participate in this study. The parents of all of the 24 children will also be asked to participate and give us their feed-back on what it is like to cope with a child with cGVHD.
Key inclusion criteria (6 listed)
- 5-18 years of age
- Prior allogeneic Stem Cell Transplant, with any graft source, donor type, and GVHD prophylaxis allowed
- Clinical diagnosis of cGVHD
- Need for systemic treatment, defined as any medication or intervention delivered
- No evidence of primary disease relapse
Key exclusion criteria (1 listed)
- Inability to give signed informed consent
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
To develop a Pediatric Chronic GVHD Symptoms Scale (PCSS) that reliably measures the disease specific burden of chronic GVHD in children
Time frame: One Day Interview will be performed
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 8 Oct 2019.
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Graft vs Host Disease
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.
Reggie E Duerst, MD principal investigator
Ann & Robert H Lurie Children's Hospital of Chicago
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 Graft vs Host Disease, run by Ann & Robert H Lurie Children's Hospital of Chicago 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 6 inclusion and 1 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 8 Oct 2019. 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?
To develop a Pediatric Chronic GVHD Symptoms Scale (PCSS) that reliably measures the disease specific burden of chronic GVHD in children (time frame: One Day Interview will be performed). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 8 Oct 2019.