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

Nippon Kayaku

Status and phase

Completed

Phase 2

Conditions

Wegener's Granulomatosis

Treatments

Drug: Gusperimus

Study type

Interventional

Funder types

Industry

Identifiers

NCT00530075

102

Details and patient eligibility

About

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 Immunosuppressive drugs were withdrawn at entry and prednisolone doses adjusted according to clinical status.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.

Enrollment

45 patients

Sex

All

Ages

18 to 80 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Documented diagnosis of WG according to American College of Rheumatology (ACR) and Chapel Hill Consensus Conference (CHCC) definition
BVAS >= 4
Total disease duration >= 3 months treated with CYC or >= 6 months with MTX
Age 18 - 80
WBC >= 4,000/mm3, haemoglobin >= 8g/dl, neutrophils >= 2,500/mm3, platelets >= 100,000/mm3
ALT, bilirubin and alkaline phosphatase levels within 2x the upper limits of normal
Documented to be non-pregnant by serum/urine pregnancy test
Willing to participate in this study
Provide signed informed consent
Able and prepared to self-administer the study drug or have a close friend/relative able to do this

Exclusion criteria

Participation in another clinical research study
Pregnant or nursing mothers and women of childbearing age not using appropriate contraception
Clear evidence of active disease due to bacteria/viral infection
Patient has an unacceptable risk for participation in a study of immunosuppressive therapy
History of substance abuse or psychotic disorders
Previous treatment with Gusperimus