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

Mallinckrodt

Status and phase: Completed; Early Phase 1

Conditions: Chronic Graft Versus Host Disease

Treatments: Drug: Standard of Care; Drug: ECP Methoxsalen

Study type: Interventional

Funder types: Industry

Identifiers

NCT01380535

10-005

2010-022780-35 (EudraCT Number)

Details and patient eligibility

About

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.

Full 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).

Enrollment: 60 patients

Sex: All

Ages: 18 to 99 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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60 participants in 2 patient groups

ECP Methoxsalen + Standard of Care

Experimental group

Description:

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

Treatment:

Drug: ECP Methoxsalen

Drug: Standard of Care

Standard of Care

Active Comparator group

Description:

Participants receive standard of care only

Treatment:

Drug: Standard of Care