A Phase I Study of Abatacept in the Treatment of Patients With Steroid Refractory Chronic Graft Versus Host Disease (cGVHD)
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 will be treated at a higher dose. Three to four tablespoons of blood will be drawn at every 2 week visit in order to determine your blood counts, kidney and liver function. Some of the blood will be used in a research lab in order to study measures of your immune system and how they might be affected by the treatment.
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||A Phase I Study of Abatacept in the Treatment of Patients With Steroid Refractory Chronic Graft Versus Host Disease (cGVHD)|
- 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 ]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.
- 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.
|Study Start Date:||October 2013|
|Estimated Study Completion Date:||December 2024|
|Estimated Primary Completion Date:||December 2019 (Final data collection date for primary outcome measure)|
Abatacept will be administered for a total of 6 doses. Doses 1-3 will be administered at two week intervals (+/-2 days). One month following Dose 3, abatacept will be administered, and given at four-week intervals (+/-2 days) for three doses (Doses 4-6.) Patients will be followed for toxicity for 28 days following last dose of Abatacept. Patients will then be seen monthly for six months.
The study will follow a standard 3+3 design with two escalating doses of abatacept to determine the maximum tolerated dose (MTD): 3 mg/kg (dose level 1) and 10 mg/kg (dose level 2). Dose-limiting toxicities (DLTs) are defined as any Grade 3 or 4 toxicities judged to be probably or definitely related to abatacept.
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01954979
|Contact: Jacalyn Rosenblatt, MD||(617) 667-9920 ext email@example.com|
|Contact: Emma Logan||(617) firstname.lastname@example.org|
|United States, Massachusetts|
|Dana-Farber Cancer Institute||Recruiting|
|Boston, Massachusetts, United States, 02115|
|Contact: Robert Soiffer 617-632-4711|
|Principal Investigator: Robert Soiffer, MD|
|Beth Israel Deaconess Medical Center||Recruiting|
|Boston, Massachusetts, United States, 02215|
|Contact: Jacalyn Rosenblatt, MD 617-632-4218 email@example.com|
|Principal Investigator: Jacalyn Rosenblatt, MD|
|Principal Investigator:||Jacalyn Rosenblatt, MD||Beth Israel Deaconess Medical Center|