Biomarkers in Acute Graft-Versus-Host Disease and Extracorporeal Photopheresis Added to Investigator Chosen Therapies of Steroid Refractory Acute GVHD
| Status: | Suspended |
|---|---|
| Conditions: | Orthopedic, Orthopedic, Hematology |
| Therapuetic Areas: | Hematology, Orthopedics / Podiatry |
| Healthy: | No |
| Age Range: | 18 - 99 |
| Updated: | 2/17/2019 |
| Start Date: | December 20, 2014 |
| End Date: | October 1, 2027 |
Biomarkers in Acute Graft-Versus-Host Disease (GVHD) and Extracorporeal Photopheresis Added to Investigator Chosen Therapies of Steroid Refractory Acute GVHD
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 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.
- 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 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.
Background:
- Acute graft versus host disease (GVHD) remains a difficult to manage complication of
allogeneic hematopoietic stem cell transplantation causing significant morbidity and
mortality.
- Biomarkers have recently been described in acute GVHD that have the potential to better
predict onset, severity, steroid failure, and non-relapse mortality.
- First line treatment of acute GVHD with high dose corticosteroids will fail in
approximately 30% of patients and is associated with significant steroid related
complications.
- No second line treatment of acute GVHD after a failure of steroids has been established
as a standard approach.
- Choice of second line therapy for acute GVHD is currently based primarily on physician
familiarity, existing toxicities, and patient's ability to tolerate new potential
toxicities.
- Extracorporeal photopheresis (ECP) is an attractive therapy to combine with other
therapies for steroid refractory disease due to a unique mechanism of action involving
immunomodulation as well as an extremely low rate of reported side effects and
complications.
- Biomarkers may also prove useful in predicting the success or failure of specific
treatments for steroid refractory disease, including those combined with ECP.
- This study will allow for collection of biomarker data in patients undergoing allogeneic
transplantation on NCI protocols, including those who develop acute GVHD and investigate
their role in predicting outcomes in initial corticosteroid therapy as well as in
currently used treatments in the management of patients with steroid refractory acute
GVHD with or without the addition of ECP.
Objective:
-To study biomarkers in patients undergoing allogeneic transplantation, with acute GVHD
including their ability to predict steroid refractoriness and predict outcome of investigator
chosen second line therapies with and without Extracorporeal Photopheresis (ECP).
Eligibility:
- Adult patients on an NCI allogeneic transplantation protocol.
Design:
- Non-randomized, single institution study.
- Research blood for biomarkers will be collected on all patients enrolled.
- ECP will be offered as an addition to investigator chosen treatments in patients who
develop steroid refractory acute GVHD.
- The study will enroll a total of up to 450 patients.
- Acute graft versus host disease (GVHD) remains a difficult to manage complication of
allogeneic hematopoietic stem cell transplantation causing significant morbidity and
mortality.
- Biomarkers have recently been described in acute GVHD that have the potential to better
predict onset, severity, steroid failure, and non-relapse mortality.
- First line treatment of acute GVHD with high dose corticosteroids will fail in
approximately 30% of patients and is associated with significant steroid related
complications.
- No second line treatment of acute GVHD after a failure of steroids has been established
as a standard approach.
- Choice of second line therapy for acute GVHD is currently based primarily on physician
familiarity, existing toxicities, and patient's ability to tolerate new potential
toxicities.
- Extracorporeal photopheresis (ECP) is an attractive therapy to combine with other
therapies for steroid refractory disease due to a unique mechanism of action involving
immunomodulation as well as an extremely low rate of reported side effects and
complications.
- Biomarkers may also prove useful in predicting the success or failure of specific
treatments for steroid refractory disease, including those combined with ECP.
- This study will allow for collection of biomarker data in patients undergoing allogeneic
transplantation on NCI protocols, including those who develop acute GVHD and investigate
their role in predicting outcomes in initial corticosteroid therapy as well as in
currently used treatments in the management of patients with steroid refractory acute
GVHD with or without the addition of ECP.
Objective:
-To study biomarkers in patients undergoing allogeneic transplantation, with acute GVHD
including their ability to predict steroid refractoriness and predict outcome of investigator
chosen second line therapies with and without Extracorporeal Photopheresis (ECP).
Eligibility:
- Adult patients on an NCI allogeneic transplantation protocol.
Design:
- Non-randomized, single institution study.
- Research blood for biomarkers will be collected on all patients enrolled.
- ECP will be offered as an addition to investigator chosen treatments in patients who
develop steroid refractory acute GVHD.
- The study will enroll a total of up to 450 patients.
- INCLUSION CRITERIA:
- Age greater than or equal to18 years.
- Ability of subject to understand and the willingness to sign a written informed
consent document.
- Subject must be also enrolled on an NCI allogeneic transplant protocol.
- Patients must agree to practice effective contraception (both male and female
subjects, if the risk of conception exists)The effects of ECP on the developing human
fetus are unknown. For this reason and as well as other Methoxsalen used in this trial
is in a class of agents that is known to be teratogenic, men must agree to use
adequate contraception (hormonal or barrier method of birth control; abstinence) prior
to study entry and for the duration of study participation, and for 4 months after the
completion of study treatment. Should a woman become pregnant or suspect she is
pregnant while her partner is participating in this study, she should inform her
treating physician immediately.
EXCLUSION CRITERIA:
- Any physical or mental condition that, in the opinion of the PI, would cause the
risk/benefit ratio of participation to be unacceptable.
- Inclusion of ECP in the treatment of any patient is contraindicated by any of the
following:
- Unstable hemodynamics requiring vasopressors or other support measures not amenable to
or medically appropriate for continuation during the procedure.
- Uncontrolled infection.
- Inability to maintain acceptable venous access.
- Uncontrolled or uncorrectable coagulopathy.
- Pregnant women are excluded from ECP because methoxsalen, an agent utilized for the
study procedure, may cause fetal harm. Because there is an unknown but potential risk
for adverse events in nursing infants secondary to treatment of the mother with
methoxsalen, breastfeeding should be discontinued if the mother is treated with
methoxsalen. Pregnancy will be evaluated prior to initiation of ECP.
- History of allergic or idiosyncratic/hypersensitivity reactions to 8-
methoxypsoralen/psoralen compounds.
- History of a light-sensitive cutaneous disease
- Subjects with aphakia
We found this trial at
1
site
9000 Rockville Pike
Bethesda, Maryland 20892
Bethesda, Maryland 20892
Phone: 888-624-1937
Click here to add this to my saved trials
