Phase II Study on Gusperimus in Patients With Refractory Wegener's Granulomatosis
Status:
Completed
Trial end date:
2006-02-01
Target enrollment:
Participant gender:
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Immunosuppressive drugs were withdrawn at entry and prednisolone doses adjusted according to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.