Phase I Trial of LDE225 for Steroid-refractory Chronic GVHD After Allogeneic HSCT

Graft Versus Host Disease Clinical Trial

Official title: Phase I Trial of LDE225 for Steroid-refractory Chronic GVHD After Allogeneic HSCT

Status Terminated

Clinical Trial Summary

This is a phase I trial of LDE225 for the treatment of steroid-refractory chronic Graft Versus Host Disease (GVHD).

Clinical Trial Description

This phase I clinical trial will enroll participants with steroid-refractory chronic GVHD, and likely take approximately 24 months to complete accrual. Treatment will consist of LDE225 given daily for continuous dosing in 28-day cycles. Participants will have undergone allogeneic SCT and have developed chronic GVHD which has not responded sufficiently to systemic corticosteroids (dose of at least 0.25 mg/kg/day of ideal body weight), have relapsed disease while tapering steroids, or not having an adequate response to steroids plus other therapies. Participants who are responding will then be eligible to receive additional courses of therapy. Participants will be followed on trial for 12 months after starting therapy and the trial will be completed when all participants have reached 12 months of follow-up or have withdrawn from the trial.

The initial dose escalation phase of 4 cohorts of participants (each cohort 3-6 participants) will have a primary endpoint of safety and toxicity of administering LDE225 in this setting. ;

Related Conditions & MeSH terms

• Graft Versus Host Disease
• Graft vs Host Disease

• NCT number: NCT02086513
• Study type: Interventional
• Source: Massachusetts General Hospital
• Status: Terminated
• Phase: Phase 1
• Start date: April 2014
• Completion date: December 2016