NCT02086513

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

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: Massachusetts General Hospital · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed 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starting therapy and the trial will be completed when all participants have reached 12 months of follow-up or have withdrawn from the trial.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.

Conditions

• Graft Versus Host Disease

Interventions

Type	Name	Description
DRUG	LDE225	Treatment with LDE225

Timeline

• Start date: 2014-04-01
• Primary completion: 2016-12-01
• Completion: 2016-12-01
• First posted: 2014-03-13
• Last updated: 2018-03-19

Locations

3 sites across 1 country: United States