NCT00639717

Addition of Etanercept and Extracorporeal Photopheresis to Standard GVHD Prophylaxis in Patients Undergoing Reduced Intensity Unrelated Donor Hematopoietic Stem Cell Transplant

Completed Phase 2 Results posted Last updated 3 July 2017

What this trial tests

Phase 2 trial testing stem cell transplant in Graft Versus Host Disease in 48 participants. Completed in 1 April 2016.

Timeline

1 March 2009

Primary endpoint
1 January 2012

1 April 2016

Quick facts

Lead sponsor	University of Michigan Rogel Cancer Center
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	prevention
Enrollment	48
Start date	1 March 2009
Primary completion	1 January 2012
Estimated completion	1 April 2016
Sites	1 location across United States

Drugs / interventions tested

• stem cell transplant — full drug profile →
• tacrolimus (standard GVHD prophylaxis) — full drug profile →
• mycophenolate (standard GVHD prophylaxis)
• etanercept (etanercept) — full drug profile →
• methoxsalen (METHOXSALEN) — full drug profile →

Conditions studied

• Graft Versus Host Disease — all drugs for Graft Versus Host Disease →

Sponsor

University of Michigan Rogel Cancer Center

Who can join

Eligibility, any sex, with Graft Versus Host Disease. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Percentage of Patients Alive at 6 Months
  Time frame: 6 months
  Overall survival at 6 months
• Percentage of Patients Who Experienced Relapse by 6 Months
  Time frame: 6 months
  Relapse rate at 6 months. Relapse is defined as recurrence of disease.

Sponsor's own description

This research study investigates the benefits and possible risks of adding both etanercept (Enbrel) and ECP (extracorporeal photopheresis) to the conventional preventative (or prophylactic) treatments for graft-versus-host disease (GVHD). GVHD is a common, serious, and too often fatal, complication after matched unrelated donor stem cell transplantation, regardless of the pre-transplant conditioning regimen used (full or reduced intensity).

Reduced intensity transplants which employ lower doses of chemotherapy during the conditioning phase of the transplant, are less toxic than full intensity transplants. Reduced intensity transplants may extend the unrelated donor transplant option to older patients or to patients with existing medical conditions or illness, where a full intensity transplant is not possible. To be successful, reduced intensity transplants need to offset any lower effectiveness in killing cancer cells during the conditioning phase, with the establishment of a donor cell, graft-versus-leukemia effect (GVL). The GVL effect and GVHD are associated with each other and therefore, the goal of GVHD prophylaxis for this study is not so much to prevent all GVHD, but rather to prevent serious and fatal acute GVHD.

Most GVHD-related deaths are either the direct consequence of severe GVHD or from infections associated with intense immunosuppression, a consequence of the standard treatments for acute GVHD, which almost always include high-dose steroids. A more effective prophylaxis therapy that allows for the GVL effect to develop, while limiting the exposure to high-dose steroids may reduce transplant mortality and morbidity. We also will study how key chemical and cellular factors relate to clinical outcome.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Cytokines and costimulation in acute graft-versus-host disease.
   Hill GR, Koyama M. · · 2020 · cited 100× · PMID 32526028 · DOI 10.1182/blood.2019000952
2. Combination Therapy for Graft-versus-Host Disease Prophylaxis with Etanercept and Extracorporeal Photopheresis: Results of a Phase II Clinical Trial.
   Kitko CL, Braun T, Couriel DR, Choi SW, et al · · 2016 · cited 29× · PMID 26551636 · DOI 10.1016/j.bbmt.2015.11.002