NCT02337517

Vismodegib in Treating Patients With Steroid-Refractory Chronic Graft-Versus-Host Disease

Pilot Study for the Treatment of Steroid-Refractory Sclerodermatous Chronic Graft-Versus-Host Disease (GVHD) With GDC-0449 (GDC-0449)

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This pilot clinical trial studies how well vismodegib works in treating patients with chronic graft-versus-host disease that did not respond to previous steroid treatment. Chronic graft-versus-host disease can cause a build-up of scar tissue under the skin and lead to symptoms such as sclerodermatous skin changes, dry mouth, dry eye, narrowing of the esophagus, or vaginal graft-versus-host disease. Vismodegib may work against the build-up of scar tissue and be a better treatment for chronic graft-versus-host disease caused by a hematopoietic stem cell transplant.

Detailed description

PRIMARY OBJECTIVES: I. To determine the clinical effects of GDC-0449 (vismodegib), in steroid-refractory chronic graft-versus-host disease (GVHD). SECONDARY OBJECTIVES: I. To determine the safety of GDC-0449 in patients with steroid-refractory GVHD. II. To determine the change in National Institutes of Health (NIH) Consensus Criteria (CC) global score of chronic GVHD at 6 and 12 months from baseline. III. To determine one-year non relapse mortality (NRM) and one-year relapse rate. IV. To determine one-year failure free survival (FFS) and one-year overall survival (OS). V. To determine baseline clinical characteristics that may be associated with decreased FFS. OUTLINE: Patients receive vismodegib orally (PO) daily, every other day, every three days, or twice weekly for 6-12 months in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up every 3 months for 6 months.

Conditions

• Chronic Graft Versus Host Disease

Interventions

Type	Name	Description
OTHER	Laboratory Biomarker Analysis	Correlative studies
DRUG	Vismodegib	Given PO

Timeline

• Start date: 2015-09-08
• Primary completion: 2018-06-28
• Completion: 2018-06-28
• First posted: 2015-01-13
• Last updated: 2021-07-19
• Results posted: 2021-06-16

Locations

2 sites across 1 country: United States