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

NCT ID: NCT01380535

Last Updated: 2020-02-19

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

EARLY_PHASE1

Total Enrollment

60 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-11-30

Study Completion Date

2015-03-31

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).

Conditions

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Chronic Graft Versus Host Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Pilot Study for the 2015 NIH Consensus Criteria to inform the design of subsequent studies
Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors

Study Groups

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ECP Methoxsalen + Standard of Care

Participants receive methoxsalen administered via ECP in addition to standard of care

Group Type EXPERIMENTAL

ECP Methoxsalen

Intervention Type DRUG

Methoxsalen delivered by ECP

Standard of Care

Intervention Type DRUG

Tapered prednisone with cyclosporine or tacrolimus via oral administration, consistent with local institutional practice (corticosteroids and cyclosporine A/tacrolimus)

Standard of Care

Participants receive standard of care only

Group Type ACTIVE_COMPARATOR

Standard of Care

Intervention Type DRUG

Tapered prednisone with cyclosporine or tacrolimus via oral administration, consistent with local institutional practice (corticosteroids and cyclosporine A/tacrolimus)

Interventions

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ECP Methoxsalen

Methoxsalen delivered by ECP

Intervention Type DRUG

Standard of Care

Tapered prednisone with cyclosporine or tacrolimus via oral administration, consistent with local institutional practice (corticosteroids and cyclosporine A/tacrolimus)

Intervention Type DRUG

Other Intervention Names

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Uvadex Extracorporeal photopheresis (ECP) Reference therapy

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:

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

18 Years

Maximum Eligible Age

99 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Parexel

INDUSTRY

Sponsor Role collaborator

Mallinckrodt

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Global Clinical Leader

Role: STUDY_DIRECTOR

Mallinckrodt

Locations

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University of Miami Hospital

Miami, Florida, United States

Site Status

Indiana Blood and Marrow Transplantation Research

Indianapolis, Indiana, United States

Site Status

Tulane University Health Sciences Center, 1430 Tulane Avenue, Rm 7551,

New Orleans, Louisiana, United States

Site Status

Karmanos Cancer Institute

Detroit, Michigan, United States

Site Status

Hackensack University Medical Center

Hackensack, New Jersey, United States

Site Status

New York Presbyterian Hospital

New York, New York, United States

Site Status

Stony brook university medical Center

Stony Brook, New York, United States

Site Status

Vanderbilt Ingram Cancer Center

Nashville, Tennessee, United States

Site Status

Medical City Dallas Hospital,Transplant center

Dallas, Texas, United States

Site Status

Parkland Memorial Hospital

Dallas, Texas, United States

Site Status

Methodist healthcare system of san Antonio

San Antonio, Texas, United States

Site Status

General Hospital of Vienna (Medical University of Vienna)

Vienna, , Austria

Site Status

Hopital Saint Antoine

Saint-Antoine, Paris, France

Site Status

CHU de Nantes

Nantes, , France

Site Status

Hospital Saint Louis

Paris, , France

Site Status

Universitätsklinik Köln

Cologne, , Germany

Site Status

Universitäts-Klinikum Carl Gustav Carus

Dresden, , Germany

Site Status

Universitatsklinikum Erlangen

Erlangen, , Germany

Site Status

Universitätsklinikum Essen

Essen, , Germany

Site Status

Universitatskrankenhaus Hamburg-Eppendorf

Hamburg, , Germany

Site Status

Universitätsklinikum Leipzig AöR

Leipzig, , Germany

Site Status

Universitätklinikum Mannheim

Mannheim, , Germany

Site Status

Medizinische Universitatsklinik

München, , Germany

Site Status

Uniklinik für Kinder und Jugendmedizin

Tübingen, , Germany

Site Status

Egyesített Szent István és Szent László Kórház, Gyáli út 5-7,

Budapest, , Hungary

Site Status

A.O.U. Policlinico- Vittorio Emanuele

Catania, , Italy

Site Status

Universita de Genova - Ospedale S. Martino

Genova, , Italy

Site Status

Hospital Marques de Valdecilla

Santander, Cantabria, Spain

Site Status

Santa Creu I Sant Pau

Barcelona, , Spain

Site Status

Kings College Hospital

London, Greater London, United Kingdom

Site Status

University of Birmingham: Queen Elizabeth Hospital

Birmingham, , United Kingdom

Site Status

Royal Marsden Hospital

London, , United Kingdom

Site Status

Countries

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United States Austria France Germany Hungary Italy Spain United Kingdom

References

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Jagasia M, Scheid C, Socie G, Ayuk FA, Tischer J, Donato ML, Batai A, Chen H, Chen SC, Chin T, Boodee H, Mitri G, Greinix HT. Randomized controlled study of ECP with methoxsalen as first-line treatment of patients with moderate to severe cGVHD. Blood Adv. 2019 Jul 23;3(14):2218-2229. doi: 10.1182/bloodadvances.2019000145.

Reference Type RESULT
PMID: 31332045 (View on PubMed)

Related Links

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https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6650730/

Cited article made available by the US National Library of Medicine, National Institutes of Health

Other Identifiers

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2010-022780-35

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

10-005

Identifier Type: -

Identifier Source: org_study_id

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