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

Graft vs Host Disease Clinical Trial

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

Status Completed

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Graft vs Host Disease

• NCT number: NCT00144430
• Study type: Interventional
• Source: Ann & Robert H Lurie Children's Hospital of Chicago
• Status: Completed
• Phase: Phase 2
• Start date: January 2004
• Completion date: August 2008