NCT02322190

Biomarkers in Acute Graft-Versus-Host Disease and Extracorporeal Photopheresis Added to Investigator Chosen Therapies of Steroid Refractory Acute GVHD

Terminated Phase 2 Results posted Last updated 12 March 2020

What this trial tests

Phase 2 trial testing Extracorporeal Photopheresis (ECP) in Chronic Graft vs. Host Disease in 5 participants. Terminated before completion.

Timeline

20 December 2014

Primary endpoint
31 October 2019

31 October 2019

Quick facts

Lead sponsor	National Cancer Institute (NCI)
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	non randomized
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	5
Start date	20 December 2014
Primary completion	31 October 2019
Estimated completion	31 October 2019
Sites	1 location across United States

Drugs / interventions tested

• Extracorporeal Photopheresis (ECP) — full drug profile →
• Methoxsalen (METHOXSALEN) — full drug profile →

Conditions studied

• Chronic Graft vs. Host Disease — all drugs for Chronic Graft vs. Host Disease →
• Graft vs Host Disease — all drugs for Graft vs Host Disease →
• Graft-Versus-Host Disease — all drugs for Graft-Versus-Host Disease →

Sponsor

National Cancer Institute (NCI)

Who can join

Adults 18 to 99, any sex, with Chronic Graft vs. Host Disease or Graft vs Host Disease. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants With Steroid Refractory Disease Who Had Improvement and/or Resolution of Graft-versus-host Disease (GVHD) Associated Symptoms Primary · 7 months

GVHD associated symptoms was assessed by the American Society for Bone Marrow Transplantation consensus statement. Complete Response (CR) is no residual organ specific symptoms or findings. Very Good Partial Response (VGPR) is Skin: An active erythematous rash involving \< 25% of the body surface area; Liver: Persistent low-level hyperbilirubinemia, and/or Gut: Gastrointestinal function and water resorption in the colon are approaching normal. Partial Response (PR) is any improvement over baseline symptoms. Progressive disease (PD) is stable or worsening organ specific findings requiring chang

Group	Value	95% CI
Investigator Chosen Second Line Therapy	2

Days to Overall Survival Secondary · time from study entry to end of observations/off study, up to a year

Overall survival is defined as the time from study entry to end of observations/off study.

Group	Value	95% CI
Investigator Chosen Second Line Therapy	180	117 – 318

Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). Secondary · Date treatment consent signed to date off study, approximately 8 months and 6 days

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one

Group	Value	95% CI
Investigator Chosen Second Line Therapy	1

Adverse events — posted to ClinicalTrials.gov

Time frame: Date treatment consent signed to date off study, approximately 8 months and 6 days.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Investigator Chosen Second Line Therapy

Serious: 1/5 (20%)

Deaths: 1/5

Second Line Therapy + Extracorporeal Photopheresis

Serious: 0

Deaths: 0

Serious adverse events (1 terms)

Reaction	System	Investigator Chosen Second…	Second Line Therapy + Extr…
Death	General disorders	—	—

Most-reported serious reactions: Death.

Data from ClinicalTrials.gov NCT02322190 adverse events section.

Sponsor's own description

Background:

\- Some allogeneic stem cell transplant recipients get acute graft-versus-host disease (GVHD). They always get steroids as the first treatment, but this may not work. Those people where steroids are not enough may benefit from a treatment called extracorporeal photopheresis (ECP). ECP exposes white blood cells to ultraviolet light outside the body. Researchers want to study how certain markers in the blood predict the severity and outcome of acute GVHD and how ECP treatments work for people with acute GVHD. They will also study how these markers in the blood may help predict who should get ECP and its effects on the immune system.

Objectives:

\- To learn more about treatments for acute GVHD after allogeneic stem cell transplantation.

Eligibility:

\- Adults with acute GVHD enrolled in an National Cancer Institute (NCI) allogeneic transplantation protocol.

Design:

* Transplant physicians will confirm participant eligibility.
* Participants will receive treatment with steroids for their acute GVHD as prescribed by their transplant physician. This will continue while they are enrolled on this study.
* If steroids work in treating their acute GVHD, then every 28 days for 6 months, participants will have:
* a physical exam.
* blood tests.
* If steroids do not work, participants will get additional treatments as prescribed by their transplant physician who may choose to use ECP as a part of this additional treatment. For ECP, blood is removed through an intravenous (IV) catheter. A machine separates the white blood cells from the other blood parts. Those cells are treated with methoxsalen and exposed to ultraviolet light. Then they are returned to the participant through their IV.
* Participants who get ECP will over at least 6 months have:
* veins researched. They may have a catheter placed in a larger vein in the chest or groin.
* multiple blood tests.
* multiple pregnancy tests (if needed).
* multiple ECP procedures.
* At the end of ECP treatment and 6 months after ECP, participants will have additional physical exams and blood tests.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.