Pentostatin for High Risk and Refractory Chronic Graft Versus Host Disease in Children
This is a multicenter trial through the Pediatric Blood and Marrow Transplant Consortium.
The Primary hypothesis of this study is that because of its effect as a potent immunosuppressive agent targeting lymphocytes, pentostatin will show a sustained response in pediatric subjects with severe chronic GVHD. Secondary hypotheses include that the infection and toxicity rate of pentostatin in this setting will be acceptable given its lack of severe myelosuppression, and subjects with refractory chronic GVHD will have significant QOL impairment and symptomatology. These may change as subjects are being treated for their chronic GVHD with pentostatin.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase II Study of Pentostatin For the Treatment of High Risk or Refractory Chronic GVHD in Children|
- To estimate the response rate (CR and PR) of pentostatin when used to treat refractory-chronic GVHD in children. [ Time Frame: To end of study ] [ Designated as safety issue: No ]
- To gather initial efficacy and safety data in high-risk untreated chronic GVHD in children. [ Time Frame: To end of study ] [ Designated as safety issue: Yes ]
- To evaluate toxicities of pentostatin when used to treat chronic GVHD in children. [ Time Frame: To end of study ] [ Designated as safety issue: Yes ]
- To evaluate quality of life (QOL) and symptoms at diagnosis and after therapy with pentostatin in pediatric patients with refractory chronic GVHD. [ Time Frame: To end of study ] [ Designated as safety issue: No ]
|Study Start Date:||January 2004|
|Study Completion Date:||August 2008|
|Primary Completion Date:||August 2008 (Final data collection date for primary outcome measure)|
To participate in this study, subjects must have diagnosed chronic Graft versus Host Disease that is refractory to therapy or that is considered high risk (i.e. low platelet count, progressive onset and greater than 50% of body surface area affected). Subjects must have not failed more than 2 immunosuppressive regimens in order to be considered for this trial. Eligible subjects will receive intravenous pentostatin every 2 weeks for 24 weeks. If the subject has had a complete response, the therapy will end at 24 weeks. If the subject has had a partial or mixed response or stable disease, they will continue on study receiving pentostatin for 52 weeks.
Show 25 Study Locations
|Principal Investigator:||David Jacobsohn, MD, MSc||Children's Memorial Hospital, Chicago, IL|