Extracorporeal Photopheresis (ECP) With Methoxsalen for Chronic Graft Versus Host Disease (cGVHD)

Study Overview
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Brief Summary
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Chronic graft-versus-host disease (cGVHD) is a long-lasting complication that can occur after transplants. The transplanted cells seem to fight with the patient's own cells. Extracorporeal photopheresis (ECP) is a fairly new procedure for cGVHD. The participant gets a port to hook up to a machine. The machine removes the white blood cells, mixes them with a light-sensitive drug, shines light on it, and puts all the blood back in. This study will find out if patients respond better if they get ECP with methoxsalen, in addition to the pills normally used to treat cGVHD.

Detailed Description
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The investigation of extracorporeal photopheresis (ECP) plus standard of care (SoC) (SoC+ECP) in chronic graft-versus-host disease (cGVHD) within prospective, randomized clinical studies is limited, despite its frequent clinical use. This phase 1/pilot study was the first randomized, prospective study to investigate ECP use as first-line therapy in cGVHD, based on the 2015 National Institutes of Health (NIH) consensus criteria for diagnosis and response assessment. Adult patients with new-onset (≤3 years of hematopoietic stem cell transplantation) moderate or severe cGVHD were randomized 1:1 to 26 weeks of SoC+ECP vs SoC (corticosteroids and cyclosporine A/tacrolimus) between 2011 and 2015. The primary endpoint was overall response rate (ORR), defined as complete or partial response, at week 28 in the intention-to-treat population (ITT).

Official Title
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A Randomized Controlled Study of Extracorporeal Photopheresis (ECP) Therapy With UVADEX for the Treatment of Patients With Moderate to Severe Chronic Graft-versus-Host Disease (cGvHD)

Conditions
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Chronic Graft Versus Host Disease

Intervention / Treatment
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* Drug: ECP Methoxsalen
* Drug: Standard of Care


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: To be eligible for inclusion in this trial, the patient must have moderate to severe chronic graft-versus-host disease [defined by the National Institutes of Health (NIH) 2015 Consensus Criteria] that started within 3 years after hematopoietic stem cell transplantation (HSCT). Exclusion Criteria: Is intolerant to corticosteroids or hypersensitive to methoxsalen Received certain treatments during time periods disallowed by protocol Has infections and/or requires treatment that (per protocol or in the opinion of the investigator) might compromise: safety and well-being of participant or offspring safety of study staff analysis of results

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 99 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Pilot Study for the 2015 NIH Consensus Criteria to inform the design of subsequent studies
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ECP Methoxsalen + Standard of Care<br>Participants receive methoxsalen administered via ECP in addition to standard of care | Drug: ECP Methoxsalen<br>* Methoxsalen delivered by ECP<br>* Other names: Extracorporeal photopheresis (ECP);Drug: Standard of Care<br>* Tapered prednisone with cyclosporine or tacrolimus via oral administration, consistent with local institutional practice (corticosteroids and cyclosporine A/tacrolimus)<br>* Other names: Reference therapy;|
| Active Comparator: Standard of Care<br>Participants receive standard of care only | Drug: Standard of Care<br>* Tapered prednisone with cyclosporine or tacrolimus via oral administration, consistent with local institutional practice (corticosteroids and cyclosporine A/tacrolimus)<br>* Other names: Reference therapy;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With an Overall Response at Week 28 | Participants with overall response included those with partial response or complete response, according to study staff who did not know which treatment they received (blinded assessment). | Week 28 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Extracorporeal photopheresis therapy, Methoxsalen, cGVHD, hematopoietic stem cell transplantation