NCT00031824

Hydroxychloroquine in Treating Patients With Newly Diagnosed Chronic Graft-Versus-Host Disease

Phase III Trial of Hydroxychloroquine + Standard Therapy for Chronic Graft-Versus-Host Disease

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 82 (actual)
• Sponsor: Children's Oncology Group · Network
• Sex: All
• Age: 1 Year – 29 Years
• Healthy volunteers: Not accepted

Summary

RATIONALE: Hydroxychloroquine may decrease the immune response and be effective in treating chronic graft-versus-host disease. It is not yet known if standard therapy for graft-versus-host disease is more effective with or without hydroxychloroquine. PURPOSE: Randomized phase III trial to compare the effectiveness of standard therapy alone with that of standard therapy plus hydroxychloroquine in treating patients who have newly diagnosed chronic graft-versus-host disease.

Detailed description

OBJECTIVES: Primary * Compare the efficacy of prednisone and cyclosporine with vs without hydroxychloroquine in patients with newly diagnosed extensive chronic graft-versus-host disease (GVHD). Secondary * Compare the event-free and overall survival in patients treated with these regimens. * Compare the health-related quality of life, including longitudinal change in and magnitude of persistent disability, in patients treated with these regimens. * Correlate cytokine levels and T-helper cell subtypes with chronic GVHD activity and response in patients treated with these regimens. * Correlate whole blood hydroxychloroquine levels with response and toxicity in patients treated with these regimens. OUTLINE: This is a randomized, placebo-controlled, double-blind, multicenter study. Patients are randomized to one of two treatment arms. Patients may receive standard therapy comprising prednisone orally or IV 2-3 times daily or every other day and cyclosporine orally or IV twice daily or tacrolimus orally twice daily or IV by continuous infusion before randomization. Patients not receiving cyclosporine or tacrolimus prior to randomization may receive cyclosporine or tacrolimus after randomization according to institutional preference. * Arm I: Within 10-14 days of beginning therapy with prednisone and cyclosporine or tacrolimus, patients receive oral hydroxychloroquine twice daily. * Arm II: Patients receive standard therapy with prednisone and cyclosporine or tacrolimus as in arm I and oral placebo twice daily. In both arms, treatment continues for 9 months in the absence of disease progression or unacceptable toxicity. Patients with no response after 2 months of therapy are taken off study. Quality of life is assessed at baseline, 1 month, 9 months, and 1 year. Patients are followed every month for 3 months and at 9 months. PROJECTED ACCRUAL: A total of 232 patients (116 per treatment arm) will be accrued for this study within 3.6 years.

Conditions

• Graft Versus Host Disease

Interventions

Type	Name	Description
DRUG	cyclosporine
DRUG	hydroxychloroquine
DRUG	prednisone
DRUG	tacrolimus

Timeline

• Start date: 2002-04-01
• Primary completion: 2005-05-01
• Completion: 2011-01-01
• First posted: 2003-01-27
• Last updated: 2014-02-14

Locations

104 sites across 6 countries: United States, Australia, Canada, New Zealand, Puerto Rico, Switzerland