Predicting the Quality of Response to Specific Treatments in Patients With cGVHD, PQRST Study

Chronic Graft Versus Host Disease Clinical Trial

Official title:

Predicting the Quality of Response to Specific Treatments (PQRST)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04431479
• Other study ID #: RG1006823
• Secondary ID: NCI-2020-0124210
• Status: Recruiting
• Start date: June 29, 2020
• Est. completion date: August 2, 2025

Study information

• Verified date: March 2024
• Source: Fred Hutchinson Cancer Center
• Contact: Stephanie J Lee
• Phone: 206-667-6190
• Email: sjlee[@]fredhutch.org
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This trial collects clinical data and blood samples to predict the quality of response to specific treatments in patients with chronic graft-versus-host disease (cGVHD) who are about to start initial or second-line therapy. Collecting and analyzing clinical data and blood samples from patients with cGVHD before and after treatment initiation may help doctors identify changes that may predict treatment response.

Description:

OUTLINE: Patients complete questionnaires over 10 minutes about physical symptoms, activity level, and emotional well-being and have their medical records reviewed at baseline, 1, 3, and 6 months after starting index treatment, and at start of a new systemic treatment. Patients also undergo collection of blood samples over 1-2 minutes at baseline and at 1 month after starting index treatment, or at a treatment change visit if new therapy has not started.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: August 2, 2025
• Est. primary completion date: August 2, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Prior allogeneic stem cell transplant, with any graft source, donor type, and GVHD prophylaxis
• No evidence of persistent or progressive malignancy at the time of enrollment
• Agrees to be evaluated at the transplant center before initial or second-line treatment is started (may be concurrent with the enrollment visit), and later between 2-6 weeks, 3 months and 6 months after treatment is started or if a new therapy is started before 6 months
• Signed, informed consent

Exclusion Criteria:

• Inability to comply with study procedures
• Uncontrolled psychiatric disorder
• Anticipated survival < 6 months

Related Conditions & MeSH terms

• Bronchiolitis Obliterans Syndrome
• Chronic Graft Versus Host Disease
• Graft vs Host Disease

Intervention

Procedure:
• Biospecimen Collection
Undergo collection of blood sample

Other:
• Medical Chart Review
Review of medical chart
• Quality-of-Life Assessment
Ancillary studies
• Questionnaire Administration
Complete questionnaire

Locations

Country	Name	City	State
Canada	Vancouver General Hospital/BC Cancer	Vancouver	British Columbia
United States	Dana-Farber Harvard Cancer Center	Boston	Massachusetts
United States	Roswell Park Cancer Institute	Buffalo	New York
United States	Cleveland Clinic Foundation	Cleveland	Ohio
United States	University of Florida	Gainesville	Florida
United States	University of Minnesota/Masonic Cancer Center	Minneapolis	Minnesota
United States	Vanderbilt University/Ingram Cancer Center	Nashville	Tennessee
United States	UPMC Hillman Cancer Center	Pittsburgh	Pennsylvania
United States	Fred Hutch/University of Washington Cancer Consortium	Seattle	Washington
United States	Moffitt Cancer Center	Tampa	Florida

Sponsors (2)

Lead Sponsor	Collaborator
Fred Hutchinson Cancer Center	National Cancer Institute (NCI)

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Clinical Response according to the 2014 criteria	At the assessments, the 9 provider-reported National Institute of Health (NIH) organ severity scores (skin, eye, mouth, esophagus, upper gastrointestinal [GI], lower GI, liver, lung, and joint manifestations) will be collected reflecting disease activity in the past week. All scored items are single 4-7 point Likert scales. Based on past work, we anticipate it will take approximately 8 minutes to conduct the physical exam and record the relevant results. Pulmonary function testing results will be collected if available otherwise portable spirometry will be performed. Response will be assessed according to the recommendations of the 2014 NIH response measures publication or any applicable updates.	Up to 6 months
Primary	Time to next systemic treatment	Any addition of another systemic cGVHD treatment for medical reasons will be considered a failure, whether added because of a new or worsening manifestation of cGVHD, used as a "steroid sparing agent," or substituted due to toxicity.	From the start of the index medication until the addition of another systemic chronic graft versus host disease (cGVHD) treatment with death and treated recurrent malignancy considered competing events, assessed up to 3 years
Primary	Duration of treatment	Duration of treatment is defined as the time until discontinuation of therapeutic systemic immunosuppression (adrenal replacement and topical/local therapies are allowed) without resumption for at least 3 months.	Up to 3 months
Primary	Survival		From the start of the index medication to death with patients lost to follow up or alive at the conclusion of the study censored, assessed up to 3 years
Primary	Non-relapse mortality	Non-relapse mortality is defined as death in remission, and relapse is considered a competing risk.	Up to 3 years
Primary	Patient-reported outcomes	Will be assessed using Lee symptom scale and Patient Reported Outcomes Measurement Information System (PROMIS). The summary score of the Lee Symptom Scale and the PROMIS Global will be calculated according to the instructions of the developers. For analyses assessing change in quality of life, improvement or worsening of the Summary symptom score by 6 points or more or the PROMIS Physical or Mental Functioning scales by 5 points or more compared to baseline will be considered a clinically significant change.	Up to 3 years