NCT02123966

An Open Label Phase II Trial of Topical Sirolimus for the Treatment of Refractory Oral Chronic Graft-versus-Host-Disease

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Dana-Farber Cancer Institute · Academic / Other
• Sex: All
• Age: 4 Years
• Healthy volunteers: Not accepted

Summary

This research study is evaluating the effectiveness of topical sirolimus combined with topical steroid therapy, as a possible treatment for oral cGVHD.

Detailed description

Topical steroid therapy can be effective in managing oral cGVHD symptoms. However, a certain proportion of participants will not experience an adequate response to topical steroids and continue to have some degree of discomfort. Sirolimus is a non-steroidal immunosuppressive medication that has demonstrated efficacy in the management of cGVHD. Based on this, it is believed that a topical formulation applied inside the mouth may also demonstrate efficacy on a localized basis. The purpose of this study is to assess the safety and efficacy of topical sirolimus as a swish and spit solution for the treatment of oral cGVHD in participants that have not had an adequate clinical response to topical steroid therapy alone.

Conditions

• Chronic Graft-versus-Host-Disease
• Oral Mucosal Disease Due to Graft-versus-host Disease

Interventions

Type	Name	Description
DRUG	Sirolimus

Timeline

• Start date: 2014-07-01
• Primary completion: 2016-05-01
• Completion: 2016-05-01
• First posted: 2014-04-28
• Last updated: 2018-07-24
• Results posted: 2018-07-24

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study