Clinical Trial: A Phase I Study of Abatacept in the Treatment of Patients With Steroid Refractory Chronic Graft Versus Host Disease (cGVHD)
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Phase I Study of Abatacept in the Treatment of Patients With Steroid Refractory Chronic Graft Versus Host Disease (cGVHD)
Brief Summary:
The participant is invited to take part in this study because they have chronic Graft versus Host Disease (cGVHD) that is not responding to standard treatment with steroids. This research study is a way of gaining new knowledge about the treatment of patients with cGVHD. This research study is evaluating a drug called abatacept.
Abatacept is a drug that alters and suppresses the immune system. Abatacept is approved by the Food and Drug Administration (FDA) for the treatment of moderate to severe active rheumatoid arthritis in adults and of severe juvenile idiopathic arthritis (JIA) in patients who have failed prior therapy with disease-modifying anti-rheumatic drugs (DMARDs). These are autoimmune conditions, ie caused by an overactive immune system that attacks normal tissues and organs. It is currently being tested in a variety of other autoimmune conditions. In this case it is considered experimental.
cGVHD is caused by the donor cells attacking various organs of the recipient. The investigators try to minimize this immune attack by using corticosteroids such as prednisone. In severe cases prednisone is not sufficient and other immunosuppressive medications are used in addition in order to more efficiently control cGVHD and to limit the dose and consequently the multiple side-effects of corticosteroids. This study is being done to determine if the use of abatacept is safe in patients with cGVHD and if it can facilitate a better control of cGVHD.
During this study the participants will be evaluated for side effects from the treatment with abatacept, and for response of the cGVHD to the treatment. There will be two groups of participants in the study. The first group will be treated at a relatively low dose of abatacept. If this is found to be safe then the second group wil
Detailed Summary:
Before the research starts (screening): After signing the consent form, the participant will be asked to undergo some screening tests or procedures to find out if they can be in the research study. Many of these tests and procedures are likely to be part of regular cGVHD care and may be done even if it turns out that the participant does not take part in the research study. If the participant has had some of these tests or procedures recently, they may or may not have to be repeated.
- Complete Medical History and Physical Examination
- Blood Collection: 3-4 tablespoons of blood will be drawn to measure the participants complete blood counts, kidney, liver and thyroid function
- Disease Assessment: Depending on what organs are affected by their cGVHD the participant may undergo blood tests or a skin exam or an eye exam or a dental exam.
- Electrocardiogram (EKG): a noninvasive test that measures the electrical activity of the participant's heart
- A tuberculin skin test and a quantiferon blood test to rule out tuberculosis
- Pregnancy testing if applicable
- HIV and Hepatitis blood test: 2-3 teaspoons of blood will be collected to perform an HIV test. The participant will be asked to sign a separate consent form for this test. The participant's doctor will discuss the results of this test with the participant and the results will become a part of their permanent medical record. The participant may seek private HIV testing prior to consenting, and based on the results may choose whether or not to participate in this study or have the HIV test become part of their medical records.
If these test
Sponsor: Beth Israel Deaconess Medical Center
Current Primary Outcome: Determination of the Maximum Tolerated Dose (among two dose levels) and toxicity profile of a 141 day/6 dose course of abatacept in patients with steroid refractory cGVHD. [ Time Frame: 2 Years ]
Original Primary Outcome: To determine the Maximum Tolerated Dose (among two dose levels) and toxicity profile of a 141 day/6 dose course of abatacept in patients with steroid refractory cGVHD. [ Time Frame: 2 Years ]
Current Secondary Outcome:
- Determination of the efficacy (in terms of cGVHD symptoms, score and steroid dose) of a 141 day/6 dose course of abatacept in patients with steroid refractory cGVHD [ Time Frame: 2 Years ]Determination of the efficacy (in terms of cGVHD symptoms, score and steroid dose) of a 141 day/6 dose course of abatacept in patients with steroid refractory cGVHD
- Examination of the immunologic effects associated with the administration of abatacept in patients with steroid refractory cGVHD. [ Time Frame: 2 Years ]Examination of the immunologic effects associated with the administration of abatacept in patients with steroid refractory cGVHD.
Original Secondary Outcome:
- To determine the efficacy (in terms of cGVHD symptoms, score and steroid dose) of a 141 day/6 dose course of abatacept in patients with steroid refractory cGVHD [ Time Frame: 2 Years ]To determine the efficacy (in terms of cGVHD symptoms, score and steroid dose) of a 141 day/6 dose course of abatacept in patients with steroid refractory cGVHD
- To examine the immunologic effects associated with the administration of abatacept in patients with steroid refractory cGVHD. [ Time Frame: 2 Years ]To examine the immunologic effects associated with the administration of abatacept in patients with steroid refractory cGVHD.
Information By: Dana-Farber Cancer Institute
Dates:
Date Received: September 27, 2013
Date Started: October 2013
Date Completion:
Last Updated: February 15, 2017
Last Verified: February 2017
