Phase II Study on Gusperimus in Patients With Refractory Wegener's Granulomatosis
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|ClinicalTrials.gov Identifier: NCT00530075|
Recruitment Status : Completed
First Posted : September 17, 2007
Results First Posted : March 6, 2017
Last Update Posted : March 6, 2017
|Condition or disease||Intervention/treatment||Phase|
|Wegener's Granulomatosis||Drug: Gusperimus||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||45 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase II Study on Gusperimus in Patients With Refractory Wegener's Granulomatosis|
|Study Start Date :||December 2003|
|Actual Primary Completion Date :||February 2006|
|Actual Study Completion Date :||February 2006|
SC, 0.5mg/kg/day, consecutive 21 days administration, 1 to 2 weeks rest, 6 cycles
- Remission of Vasculitis [ Time Frame: At Entry (Day 1 of Cycle 1), Day 22 of cycles 1-6, up to 24 weeks ]
The primary efficacy outcome measure was remission of vasculitis. Complete remission was defined as a Birmingham vasculitis activity score (BVAS) of 0 sustained for at least 2 months. Partial remission was defined as a reduction in BVAS of 50% or more, sustained for at least 2 months, when compared with the BVAS at entry.
Entry required active Wegener's granulomatosis with a BVAS >= 4. Their disease had to be active, as measured with BVAS in which clinical manifestations caused by active vasculitis are scored on a list of predefined organ-specific items.
- Duration of Clinical Response [ Time Frame: At Entry (Day 1 of Cycle 1), Day 22 of cycles 1-6, up to 24 weeks, End of treatment period, and 3 and 6 months of follow-up period ]Time from Complete Remission or Partial Remission to Relapse.
- Haematuria [ Time Frame: At Entry (Day 1 of Cycle 1), End of treatment period, up to 24 weeks ]Assessment of anti-inflammatory activity of gusperimus using surrogate marker: number of hematuria-positive patients.
- Creatinine [ Time Frame: At Entry (Day 1 of Cycle 1), End of treatment period, up to 24 weeks ]Assessment of anti-inflammatory activity of gusperimus using surrogate marker: serum creatinine level
- ANCA [ Time Frame: At Entry (Day 1 of Cycle 1), End of treatment period, up to 24 weeks ]
Assessment of anti-neutrophil cytoplasmic antibody (ANCA): Number of ANCA-positive patients was counted.
ANCA are highly associatred with active WG, with c-ANCA titres observed in 90% of WG. In addition to their diagnostic value, it has been suggested that ANCA may have a predictive value for relapse in patients with systemic vasculitis.
- CRP [ Time Frame: At Entry (Day 1 of Cycle 1), End of treatment period, up to 24 weeks ]Assessment of anti-inflammatory activity of gusperimus using surrogate marker: serum C-reactive protein level.
- Vasculitis Damage Index (VDI) [ Time Frame: At Entry (Day 1 of Cycle 1), End of treatment period, up to 24 weeks, 6 months of follow-up period ]Assessment of the degree of irreversible damage due to the vasculitis using VDI scoring system. The VDI comprises 64 items of damage (grouped into 11 organ-based systems). Total VDI score is 0 - 64. The higher scores represent the more severe damage occurred in patients. The VDI score can either increase or remain the same over time.
- SF-36 [ Time Frame: At Entry (Day 1 of Cycle 1), End of treatment period, up to 24 weeks ]Assessment of the impact of gusperimus on general health using the Short form-36 (SF-36) questionaire. The SF-36 is a self-report, 36 item survey measuring health-related quality-of-life. Thirty-five items are used to construct 8 scales: (1) physical functioning, (2) role physical, (3) bodily pain, (4) general health, (5) vitality, (6) social function, (7) role emotional, and (8) mental health. Raw scores are calculated as the sum of re-coded scale items and transformed to a 0 to 100 scale. If scores for all 8 scales are available, two summary measures known as component scores are derived: the Physical Health Component Score (PCS) and the Mental Health Component Score (MCS). First each scale standardized to the relevant population. Then PCS and MCS are calculated as the weighted sum of standardized scores. All scales and the component scores are positively scored so that higher scores represent better health-related quality-of-life.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00530075
|General Faculty Hospital|
|Prague, Czech Republic, 12808|
|Copenhagen, Denmark, 2100|
|Luebeck, Germany, 23538|
|University Hospital Maastricht|
|Maastricht, Netherlands, 6202|
|Karolinska University Hospital|
|Stockholm, Sweden, 14186|
|Western General Hospital|
|Edinburgh, Scotland, United Kingdom, EH4 2XU|
|Cambridge, United Kingdom, CB2 2QQ|
|Principal Investigator:||David Jayne||Addenbrookes Hospital|