NCT00530075

Phase II Study on Gusperimus in Patients With Refractory Wegener's Granulomatosis

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 45 (actual)
• Sponsor: Nippon Kayaku Co., Ltd. · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

Wegener's granulomatosis is a primary systemic vasculitis characterized by granulomatous and necrotizing inflammation predominantly affecting the respiratory tract and the kidneys. Conventional therapy of Wegener's granulomatosis with cyclophosphamide and corticosteroids is limited by incomplete remissions and a high relapse rate. Patients accumulate irreversible damage due to the disease and the consequences of prolonged drug exposure. The efficacy and safety of an alternative immunosuppressive drug, gusperimus, was evaluated in patients with refractory disease. A prospective, international, nulti-centre, single limb, open label study. Entry required active Wegener's granulomatosis with a Birmingham Vasculitis Activity Score (BVAS) \>=4 and previous therapy with cyclophosphamide or methotrexate. Immunosuppressive drugs were withdrawn at entry and prednisolone doses adjusted according to clinical status. Gusperimus, 0.5mg/kg/day, was self-administered by subcutaneous injection in six treatment cycles of 21 days with a seven day washout between cycles. Cycles were stopped early for white blood count \< 4,000/mm3. The primary endpoint was complete remission (BVAS=0 for at least 2 months) or partial remission (BVAS\<50% of entry score). After the sixth cycle azathioprine was commenced and follow-up continued for a further six months.

Conditions

• Wegener's Granulomatosis

Interventions

Type	Name	Description
DRUG	Gusperimus	SC, 0.5mg/kg/day, consecutive 21 days administration, 1 to 2 weeks rest, 6 cycles

Timeline

• Start date: 2003-12-01
• Primary completion: 2006-02-01
• Completion: 2006-02-01
• First posted: 2007-09-17
• Last updated: 2017-03-06
• Results posted: 2017-03-06

Locations

7 sites across 6 countries: Czechia, Denmark, Germany, Netherlands, Sweden, United Kingdom