Trial Outcomes & Findings for The Use of Etanercept Enbrel as Sole Treatment for Grade I Acute Graft Versus Host Disease (NCT NCT00726375)

Last Updated: 2016-01-05

Results Overview

We hypothesized that treatment of grade 1 acute GVHD (Graft Versus Host Disease) with etanercept would reduce the proportion of patients who progressed to grade 2 to 4 acute GVHD within 4 weeks of diagnosis from 58%, historically observed at our institution, to 38%.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

34 participants

Primary outcome timeframe

28 days

Results posted on

2016-01-05

Participant Flow

Participant milestones

Participant milestones
Measure	Etanercept a maximum of 8 SQ doses of 'Etanercept (Enbrel) at 0.4mg/kg per dose up to a maximum of 25 mg per dose Etanercept (Enbrel): Etanercept will begin within 72 hours of the diagnosis of Grade I acute GVHD and after consent for this study. Subjects receive eight doses of etanercept over four weeks. All doses will be administered by SQ injection. All subsequent doses will be given as subcutaneous injections into the skin. Injections will be given twice weekly with at least one day in between injections. The injections can be given in clinic, in the hospital, or self administered injections.
Overall Study STARTED	34
Overall Study COMPLETED	34
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

The Use of Etanercept Enbrel as Sole Treatment for Grade I Acute Graft Versus Host Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Etanercept n=34 Participants a maximum of 8 SQ doses of 'Etanercept (Enbrel) at 0.4mg/kg per dose up to a maximum of 25 mg per dose Etanercept (Enbrel): Etanercept will begin within 72 hours of the diagnosis of Grade I acute GVHD and after consent for this study. Subjects receive eight doses of etanercept over four weeks. All doses will be administered by SQ injection. All subsequent doses will be given as subcutaneous injections into the skin. Injections will be given twice weekly with at least one day in between injections. The injections can be given in clinic, in the hospital, or self administered injections.
Age, Continuous	51 years n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	26 Participants n=5 Participants
Race/Ethnicity, Customized White	30 participants n=5 Participants
Race/Ethnicity, Customized Black or African American	0 participants n=5 Participants
Race/Ethnicity, Customized Other	4 participants n=5 Participants
Diagnosis Acute myelogenous leukemia	13 participants n=5 Participants
Diagnosis Acute lymphoblastic leukemia	6 participants n=5 Participants
Diagnosis Multiple myeloma	4 participants n=5 Participants
Diagnosis Myelodysplastic syndrome	4 participants n=5 Participants
Diagnosis Non-Hodgkin's lymphoma	2 participants n=5 Participants
Diagnosis Myelofibrosis	2 participants n=5 Participants
Diagnosis Chronic lymphocytic leukemia	1 participants n=5 Participants
Diagnosis Chronic myelogenous leukemia	0 participants n=5 Participants
Diagnosis Nonmalignant disease	2 participants n=5 Participants
Disease Risk Status Low	13 participants n=5 Participants
Disease Risk Status Intermediate	5 participants n=5 Participants
Disease Risk Status High	16 participants n=5 Participants
Donor Matched related	12 participants n=5 Participants
Donor Matched unrelated	16 participants n=5 Participants
Donor Mismatched related	0 participants n=5 Participants
Donor Mismatched unrelated	6 participants n=5 Participants
CMV Status R+, D+	9 participants n=5 Participants
CMV Status R+, D-	12 participants n=5 Participants
CMV Status R-, D+	3 participants n=5 Participants
CMV Status Recipient and donor negative	10 participants n=5 Participants

PRIMARY outcome

Timeframe: 28 days

Outcome measures

Outcome measures
Measure	Etanercept n=34 Participants a maximum of 8 SQ doses of 'Etanercept (Enbrel) at 0.4mg/kg per dose up to a maximum of 25 mg per dose Etanercept (Enbrel): Etanercept will begin within 72 hours of the diagnosis of Grade I acute GVHD and after consent for this study. Subjects receive eight doses of etanercept over four weeks. All doses will be administered by SQ injection. All subsequent doses will be given as subcutaneous injections into the skin. Injections will be given twice weekly with at least one day in between injections. The injections can be given in clinic, in the hospital, or self administered injections.
The Percentage of Patients Who Progress Within 28 Days of Initiation of Etanercept Treatment	29 percentage of patients

SECONDARY outcome

Timeframe: 28 days

Estimate the proportion of patients in complete remission (CR) at four weeks who remain alive and never require additional therapy four weeks after the last dose of etanercept. Complete remission is defined as the resolution of all manifestations of GVHD (Graft Versus Host Disease) within the first four weeks of treatment. All organs must have a Grade 0.

Outcome measures

Outcome measures
Measure	Etanercept n=34 Participants a maximum of 8 SQ doses of 'Etanercept (Enbrel) at 0.4mg/kg per dose up to a maximum of 25 mg per dose Etanercept (Enbrel): Etanercept will begin within 72 hours of the diagnosis of Grade I acute GVHD and after consent for this study. Subjects receive eight doses of etanercept over four weeks. All doses will be administered by SQ injection. All subsequent doses will be given as subcutaneous injections into the skin. Injections will be given twice weekly with at least one day in between injections. The injections can be given in clinic, in the hospital, or self administered injections.
The Number of Patients in Complete Remission (CR) at Four Weeks.	14 patients

Adverse Events

Etanercept

Serious events: 3 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Etanercept n=34 participants at risk a maximum of 8 SQ doses of 'Etanercept (Enbrel) at 0.4mg/kg per dose up to a maximum of 25 mg per dose Etanercept (Enbrel): Etanercept will begin within 72 hours of the diagnosis of Grade I acute GVHD and after consent for this study. Subjects receive eight doses of etanercept over four weeks. All doses will be administered by SQ injection. All subsequent doses will be given as subcutaneous injections into the skin. Injections will be given twice weekly with at least one day in between injections. The injections can be given in clinic, in the hospital, or self administered injections.
General disorders Fever Without Neutropenia	2.9% 1/34 • Number of events 1 • Patients were followed for adverse events from the first day treatment until 14 days after the treatment was stopped. Patients typically develop numerous complications such as infections, chemotherapy and medication side effects as part of a typical bone marrow transplant. Therefore AEs and SAES that coincide with a typical transplant course, unless fatal, or possibly related to the investigational therapy, were not reported.
Gastrointestinal disorders Diarrhea	2.9% 1/34 • Number of events 1 • Patients were followed for adverse events from the first day treatment until 14 days after the treatment was stopped. Patients typically develop numerous complications such as infections, chemotherapy and medication side effects as part of a typical bone marrow transplant. Therefore AEs and SAES that coincide with a typical transplant course, unless fatal, or possibly related to the investigational therapy, were not reported.
Investigations Creatinine Elevated	2.9% 1/34 • Number of events 1 • Patients were followed for adverse events from the first day treatment until 14 days after the treatment was stopped. Patients typically develop numerous complications such as infections, chemotherapy and medication side effects as part of a typical bone marrow transplant. Therefore AEs and SAES that coincide with a typical transplant course, unless fatal, or possibly related to the investigational therapy, were not reported.

Other adverse events

Other adverse events
Measure	Etanercept n=34 participants at risk a maximum of 8 SQ doses of 'Etanercept (Enbrel) at 0.4mg/kg per dose up to a maximum of 25 mg per dose Etanercept (Enbrel): Etanercept will begin within 72 hours of the diagnosis of Grade I acute GVHD and after consent for this study. Subjects receive eight doses of etanercept over four weeks. All doses will be administered by SQ injection. All subsequent doses will be given as subcutaneous injections into the skin. Injections will be given twice weekly with at least one day in between injections. The injections can be given in clinic, in the hospital, or self administered injections.
Cardiac disorders Hypertension	2.9% 1/34 • Number of events 1 • Patients were followed for adverse events from the first day treatment until 14 days after the treatment was stopped. Patients typically develop numerous complications such as infections, chemotherapy and medication side effects as part of a typical bone marrow transplant. Therefore AEs and SAES that coincide with a typical transplant course, unless fatal, or possibly related to the investigational therapy, were not reported.
General disorders Fever Without Neutropenia	2.9% 1/34 • Number of events 1 • Patients were followed for adverse events from the first day treatment until 14 days after the treatment was stopped. Patients typically develop numerous complications such as infections, chemotherapy and medication side effects as part of a typical bone marrow transplant. Therefore AEs and SAES that coincide with a typical transplant course, unless fatal, or possibly related to the investigational therapy, were not reported.
Metabolism and nutrition disorders Anorexia	2.9% 1/34 • Number of events 1 • Patients were followed for adverse events from the first day treatment until 14 days after the treatment was stopped. Patients typically develop numerous complications such as infections, chemotherapy and medication side effects as part of a typical bone marrow transplant. Therefore AEs and SAES that coincide with a typical transplant course, unless fatal, or possibly related to the investigational therapy, were not reported.
Gastrointestinal disorders Ascites	2.9% 1/34 • Number of events 1 • Patients were followed for adverse events from the first day treatment until 14 days after the treatment was stopped. Patients typically develop numerous complications such as infections, chemotherapy and medication side effects as part of a typical bone marrow transplant. Therefore AEs and SAES that coincide with a typical transplant course, unless fatal, or possibly related to the investigational therapy, were not reported.
Gastrointestinal disorders Diarrhea	2.9% 1/34 • Number of events 1 • Patients were followed for adverse events from the first day treatment until 14 days after the treatment was stopped. Patients typically develop numerous complications such as infections, chemotherapy and medication side effects as part of a typical bone marrow transplant. Therefore AEs and SAES that coincide with a typical transplant course, unless fatal, or possibly related to the investigational therapy, were not reported.
Infections and infestations Infection	2.9% 1/34 • Number of events 1 • Patients were followed for adverse events from the first day treatment until 14 days after the treatment was stopped. Patients typically develop numerous complications such as infections, chemotherapy and medication side effects as part of a typical bone marrow transplant. Therefore AEs and SAES that coincide with a typical transplant course, unless fatal, or possibly related to the investigational therapy, were not reported.
Renal and urinary disorders Bladder Infection	2.9% 1/34 • Number of events 1 • Patients were followed for adverse events from the first day treatment until 14 days after the treatment was stopped. Patients typically develop numerous complications such as infections, chemotherapy and medication side effects as part of a typical bone marrow transplant. Therefore AEs and SAES that coincide with a typical transplant course, unless fatal, or possibly related to the investigational therapy, were not reported.
Investigations Creatinine Elevated	2.9% 1/34 • Number of events 1 • Patients were followed for adverse events from the first day treatment until 14 days after the treatment was stopped. Patients typically develop numerous complications such as infections, chemotherapy and medication side effects as part of a typical bone marrow transplant. Therefore AEs and SAES that coincide with a typical transplant course, unless fatal, or possibly related to the investigational therapy, were not reported.

Additional Information

Dr. Sung Choi

University of Michigan Comprehensive Cancer Center

Phone: 734-615-5707

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place