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

Early Phase 1

Completed

• Conditions: Chronic Graft Versus Host Disease
• Interventions: Drug: ECP Methoxsalen; Drug: Standard of Care

• Registration Number: NCT01380535
• Lead Sponsor: Mallinckrodt

Brief Summary

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

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).

Study Timeline

• Study First Submitted: 2011-06-22
• Study First Submitted QC: 2011-06-23
• Study First Posted: 2011-06-27
• Study Start: 2011-11
• Primary Completion: 2015-03
• Study Completion: 2015-03
• Results First Submitted: 2018-03-13
• Results First Submitted QC: 2019-10-28
• Results First Posted: 2019-10-29
• Status Verified: 2020-02
• Last Update Submitted: 2020-02-07
• Last Update Posted: 2020-02-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 60

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:

  1. safety and well-being of participant or offspring
  2. safety of study staff
  3. analysis of results

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
ECP Methoxsalen + Standard of Care	ECP Methoxsalen	Participants receive methoxsalen administered via ECP in addition to standard of care
ECP Methoxsalen + Standard of Care	Standard of Care	Participants receive methoxsalen administered via ECP in addition to standard of care
Standard of Care	Standard of Care	Participants receive standard of care only

Primary Outcome Measures

Name	Time	Method
Number of Participants With an Overall Response at Week 28	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).

Trial Locations

Locations (32)

Medical City Dallas Hospital,Transplant center; 🇺🇸 Dallas, Texas, United States

Parkland Memorial Hospital; 🇺🇸 Dallas, Texas, United States

A.O.U. Policlinico- Vittorio Emanuele; 🇮🇹 Catania, Italy

Uniklinik für Kinder und Jugendmedizin; 🇩🇪 Tubingen, Germany

Egyesített Szent István és Szent László Kórház, Gyáli út 5-7,; 🇭🇺 Budapest, Hungary

Universitätklinikum Mannheim; 🇩🇪 Mannheim, Germany

CHU de Nantes; 🇫🇷 Nantes, France

Universitätsklinik Köln; 🇩🇪 Cologne, Germany

Universitäts-Klinikum Carl Gustav Carus; 🇩🇪 Dresden, Germany

Universitatsklinikum Erlangen; 🇩🇪 Erlangen, Germany

Royal Marsden Hospital; 🇬🇧 London, United Kingdom

Kings College Hospital; 🇬🇧 London, Greater London, United Kingdom

Universitatskrankenhaus Hamburg-Eppendorf; 🇩🇪 Hamburg, Germany

Universita de Genova - Ospedale S. Martino; 🇮🇹 Genova, Italy

Vanderbilt Ingram Cancer Center; 🇺🇸 Nashville, Tennessee, United States

Hopital Saint Antoine; 🇫🇷 Saint Antoine, Paris, France

General Hospital of Vienna (Medical University of Vienna); 🇦🇹 Vienna, Austria

Hospital Saint Louis; 🇫🇷 Paris, France

Hospital Marques de Valdecilla; 🇪🇸 Santander, Cantabria, Spain

Universitätsklinikum Essen; 🇩🇪 Essen, Germany

University of Miami Hospital; 🇺🇸 Miami, Florida, United States

New York Presbyterian Hospital; 🇺🇸 New York, New York, United States

Tulane University Health Sciences Center, 1430 Tulane Avenue, Rm 7551,; 🇺🇸 New Orleans, Louisiana, United States

Karmanos Cancer Institute; 🇺🇸 Detroit, Michigan, United States

Indiana Blood and Marrow Transplantation Research; 🇺🇸 Indianapolis, Indiana, United States

Hackensack University Medical Center; 🇺🇸 Hackensack, New Jersey, United States

Methodist healthcare system of san Antonio; 🇺🇸 San Antonio, Texas, United States

Universitätsklinikum Leipzig AöR; 🇩🇪 Leipzig, Germany

Medizinische Universitatsklinik; 🇩🇪 Munchen, Germany

Santa Creu I Sant Pau; 🇪🇸 Barcelona, Spain

University of Birmingham: Queen Elizabeth Hospital; 🇬🇧 Birmingham, United Kingdom

Stony brook university medical Center; 🇺🇸 Stony Brook, New York, United States