Trial Outcomes & Findings for Biomarkers in Acute Graft-Versus-Host Disease and Extracorporeal Photopheresis Added to Investigator Chosen Therapies of Steroid Refractory Acute GVHD (NCT NCT02322190)

Last Updated: 2020-03-12

Results Overview

In an effort to determine steroid refractoriness after Extracorporeal Photopheresis (ECP) treatment, tissue and blood obtained from participants was examined to see if biomarkers (i.e., Serum T cell immunoglobulin and mucin domain-containing protein 3 (TIM3), ST2, Nitrate, Interleukin-6 (IL-6), regenerating family member 3 alpha (Reg-3-a), lipopolysaccharide binging protein (LPS-BP), tumor necrosis factor receptor 1 (TNFR1), interleukin-2 receptor alpha chain (IL-2Ra), CXC motif chemokine 10 (CXCL10), hepatocyte growth factor (HGF)) were present.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

5 participants

Primary outcome timeframe

14 Days

Results posted on

2020-03-12

Participant Flow

No participants were entered in to the Second Line Therapy + Extracorporeal Photopheresis group.

Participant milestones

Participant milestones
Measure	Investigator Chosen Second Line Therapy Investigator chosen second line therapy is determined on an individual basis based on standard care and actual site of Graft -versus-Host Disease (GVHD). The frequency or duration of therapy is based on Principal Investigator (PI) discretion.	Second Line Therapy + Extracorporeal Photopheresis Second line therapy in addition to Extracorporeal Photopheresis (ECP) Extracorporeal Photopheresis (ECP): Twice weekly for 1 month or Twice every other week for 2 months or Twice during one week per month for 4 months for a total of up to 7 months of treatment Methoxsalen: Used in conjunction with photopheresis procedure
Overall Study STARTED	5	0
Overall Study COMPLETED	4	0
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Investigator Chosen Second Line Therapy Investigator chosen second line therapy is determined on an individual basis based on standard care and actual site of Graft -versus-Host Disease (GVHD). The frequency or duration of therapy is based on Principal Investigator (PI) discretion.	Second Line Therapy + Extracorporeal Photopheresis Second line therapy in addition to Extracorporeal Photopheresis (ECP) Extracorporeal Photopheresis (ECP): Twice weekly for 1 month or Twice every other week for 2 months or Twice during one week per month for 4 months for a total of up to 7 months of treatment Methoxsalen: Used in conjunction with photopheresis procedure
Overall Study Death on study	1	0

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Investigator Chosen Second Line Therapy n=5 Participants Investigator chosen second line therapy is determined on an individual basis based on standard care and actual site of Graft -versus-Host Disease (GVHD). The frequency or duration of therapy is based on Principal Investigator (PI) discretion.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	44.62 years STANDARD_DEVIATION 11.65 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	5 Participants n=5 Participants

PRIMARY outcome

Timeframe: 14 Days

Population: No data was collected for this outcome measure and it not done because biomarker studies require large patient numbers to analyze data. Only five participants were enrolled in this study. With early termination, there are no biomarker data to report.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 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 change of therapy.

Outcome measures

Outcome measures
Measure	Investigator Chosen Second Line Therapy n=5 Participants Investigator chosen second line therapy is determined on an individual basis based on standard care and actual site of Graft -versus-Host Disease (GVHD). The frequency or duration of therapy is based on Principal Investigator (PI) discretion.
Number of Participants With Steroid Refractory Disease Who Had Improvement and/or Resolution of Graft-versus-host Disease (GVHD) Associated Symptoms	2 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Investigator Chosen Second Line Therapy n=5 Participants Investigator chosen second line therapy is determined on an individual basis based on standard care and actual site of Graft -versus-Host Disease (GVHD). The frequency or duration of therapy is based on Principal Investigator (PI) discretion.
Days to Overall Survival	180 Days Interval 117.0 to 318.0

SECONDARY outcome

Timeframe: 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 of the previous outcomes mentioned.

Outcome measures

Outcome measures
Measure	Investigator Chosen Second Line Therapy n=5 Participants Investigator chosen second line therapy is determined on an individual basis based on standard care and actual site of Graft -versus-Host Disease (GVHD). The frequency or duration of therapy is based on Principal Investigator (PI) discretion.
Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v4.0).	1 Participants

Adverse Events

Investigator Chosen Second Line Therapy

Serious events: 1 serious events

Other events: 0 other events

Deaths: 1 deaths

Second Line Therapy + Extracorporeal Photopheresis

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Investigator Chosen Second Line Therapy n=5 participants at risk Investigator chosen second line therapy is determined on an individual basis based on standard care and actual site of Graft -versus-Host Disease (GVHD). The frequency or duration of therapy is based on Principal Investigator (PI) discretion.	Second Line Therapy + Extracorporeal Photopheresis Second line therapy in addition to Extracorporeal Photopheresis (ECP) Extracorporeal Photopheresis (ECP): Twice weekly for 1 month or Twice every other week for 2 months or Twice during one week per month for 4 months for a total of up to 7 months of treatment Methoxsalen: Used in conjunction with photopheresis procedure
General disorders Death	20.0% 1/5 • Number of events 1 • Date treatment consent signed to date off study, approximately 8 months and 6 days. No participants were entered in to the Second Line Therapy + Extracorporeal Photopheresis group.	— 0/0 • Date treatment consent signed to date off study, approximately 8 months and 6 days. No participants were entered in to the Second Line Therapy + Extracorporeal Photopheresis group.

Other adverse events

Adverse event data not reported

Additional Information

Dr. Ronald Gress

National Cancer Institute

Phone: 240-760-6167

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place