NCT00144430

Pentostatin for High Risk and Refractory Chronic Graft Versus Host Disease in Children

A Phase II Study of Pentostatin For the Treatment of High Risk or Refractory Chronic GVHD in Children

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago · Academic / Other
• Sex: All
• Age: 1 Month – 21 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Graft vs Host Disease

Interventions

Type	Name	Description
DRUG	Pentostatin	The drug, Pentostatin, is given every 2 weeks. This drug will be given by intravenous (IV) infusion.

Timeline

• Start date: 2004-01-01
• Primary completion: 2008-08-01
• Completion: 2008-08-01
• First posted: 2005-09-05
• Last updated: 2009-02-03

Locations

25 sites across 1 country: United States