Brief Title
Phase II Study on Gusperimus in Patients With Refractory Wegener's Granulomatosis
Official Title
Phase II Study on Gusperimus in Patients With Refractory Wegener's Granulomatosis
Brief 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Immunosuppressive drugs were withdrawn at entry and prednisolone doses adjusted according to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.
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
Remission of Vasculitis
Secondary Outcome
Duration of Clinical Response
Condition
Wegener's Granulomatosis
Intervention
Gusperimus
Study Arms / Comparison Groups
1
Description: Gusperimus
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
45
Start Date
December 2003
Completion Date
February 2006
Primary Completion Date
February 2006
Eligibility Criteria
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
Gender
All
Ages
18 Years - 80 Years
Accepts Healthy Volunteers
No
Contacts
David Jayne, ,
Location Countries
Czech Republic
Location Countries
Czech Republic
Administrative Informations
NCT ID
NCT00530075
Organization ID
102
Study Sponsor
Nippon Kayaku Co., Ltd.
Study Sponsor
David Jayne, Principal Investigator, Addenbrookes Hospital
Verification Date
September 2007