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

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 18 years and older
• All sexes
• No healthy volunteers
• Adult, Older adult

Study population: Patients with chronic graft-versus-host disease (GVHD) who are starting a new therapy for GVHD

Trial locations and contacts

10 sites in 2 countries.

About this study

Official title: Predicting the Quality of Response to Specific Treatments (PQRST)

Conditions

Chronic Graft Versus Host Disease

Being tested

Biospecimen Collection, Medical Chart Review, Quality-of-Life Assessment, Questionnaire Administration

Medical subject headings

Bronchiolitis Obliterans Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

• Biospecimen CollectionProcedure or surgery

  Undergo collection of blood sample

• Medical Chart ReviewOther

  Also called Chart Review

  Review of medical chart

• Quality-of-Life AssessmentOther

  Also called Quality of Life Assessment

  Ancillary studies

• Questionnaire AdministrationOther

  Complete questionnaire

What is being measured

The primary outcome is the main question the study is designed to answer.

1. 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. Response will be assessed according to the recommendations of the 2014 NIH response measures publication or any applicable updates.

   Measured: Up to 6 months

2. 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.

   Measured: 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

3. 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.

   Measured: Up to 3 months

4. Survival

   Measured: 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

5. Non-relapse mortality

   Non-relapse mortality is defined as death in remission, and relapse is considered a competing risk.

   Measured: Up to 3 years

6. 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.

   Measured: Up to 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

1. First posted16 Jun 2020
2. Study start29 Jun 2020
3. Primary completion1 Jul 2027 (estimated)
4. Study completion1 Jul 2027 (estimated)

Resources

• Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
• Publications (2)Papers the sponsor has linked to this study.
• Other trials for Chronic Graft Versus 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.