Clinical trial NCT01380535: Extracorporeal Photopheresis (ECP) With Methoxsalen for Chronic Graft Versus Host Disease (cGVHD)
Official title: 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: EARLY_PHASE1
Conditions: Chronic Graft Versus Host Disease
Keywords: Extracorporeal photopheresis therapy
Methoxsalen
cGVHD
hematopoietic stem cell transplantation
Lead sponsor: Mallinckrodt
Sponsor class: INDUSTRY
Collaborators: name: Parexel
First posted: 2011-06-27
Enrollment: 60
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Pilot Study for the 2015 NIH Consensus Criteria to inform the design of subsequent studies
Primary purpose: TREATMENT

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).

Eligibility criteria: 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

Primary outcomes:
measure: Number of Participants With an Overall Response at Week 28
timeFrame: Week 28
Secondary outcomes:
Other outcomes:
Locations:
city: Miami
country: United States
facility: University of Miami Hospital
state: Florida
city: Indianapolis
country: United States
facility: Indiana Blood and Marrow Transplantation Research
state: Indiana
city: New Orleans
country: United States
facility: Tulane University Health Sciences Center, 1430 Tulane Avenue, Rm 7551,
state: Louisiana
city: Detroit
country: United States
facility: Karmanos Cancer Institute
state: Michigan
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: New York
country: United States
facility: New York Presbyterian Hospital
state: New York
city: Stony Brook
country: United States
facility: Stony brook university medical Center
state: New York
city: Nashville
country: United States
facility: Vanderbilt Ingram Cancer Center
state: Tennessee
city: Dallas
country: United States
facility: Medical City Dallas Hospital,Transplant center
state: Texas
city: Dallas
country: United States
facility: Parkland Memorial Hospital
state: Texas
city: San Antonio
country: United States
facility: Methodist healthcare system of san Antonio
state: Texas
city: Vienna
country: Austria
facility: General Hospital of Vienna (Medical University of Vienna)
city: Saint Antoine
country: France
facility: Hopital Saint Antoine
state: Paris
city: Nantes
country: France
facility: CHU de Nantes
city: Paris
country: France
facility: Hospital Saint Louis
city: Cologne
country: Germany
facility: Universitätsklinik Köln
city: Dresden
country: Germany
facility: Universitäts-Klinikum Carl Gustav Carus
city: Erlangen
country: Germany
facility: Universitatsklinikum Erlangen
city: Essen
country: Germany
facility: Universitätsklinikum Essen
city: Hamburg
country: Germany
facility: Universitatskrankenhaus Hamburg-Eppendorf
city: Leipzig
country: Germany
facility: Universitätsklinikum Leipzig AöR
city: Mannheim
country: Germany
facility: Universitätklinikum Mannheim
city: Munchen
country: Germany
facility: Medizinische Universitatsklinik
city: Tubingen
country: Germany
facility: Uniklinik für Kinder und Jugendmedizin
city: Budapest
country: Hungary
facility: Egyesített Szent István és Szent László Kórház, Gyáli út 5-7,
city: Catania
country: Italy
facility: A.O.U. Policlinico- Vittorio Emanuele
city: Genova
country: Italy
facility: Universita de Genova - Ospedale S. Martino
city: Santander
country: Spain
facility: Hospital Marques de Valdecilla
state: Cantabria
city: Barcelona
country: Spain
facility: Santa Creu I Sant Pau
city: London
country: United Kingdom
facility: Kings College Hospital
state: Greater London
city: Birmingham
country: United Kingdom
facility: University of Birmingham: Queen Elizabeth Hospital
city: London
country: United Kingdom
facility: Royal Marsden Hospital
Study officials:
affiliation: Mallinckrodt
name: Global Clinical Leader
role: STUDY_DIRECTOR
References:
citation: 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.