E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Wegener’s granulomatosis (WG)
Microscopic polyangiitis (MPA)
Vasculitis |
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E.1.1.1 | Medical condition in easily understood language |
Wegener’s granulomatosis (WG)
Microscopic polyangiitis (MPA)
Vasculitis |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10047888 |
E.1.2 | Term | Wegener's granulomatosis |
E.1.2 | System Organ Class | 10047065 - Vascular disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10063344 |
E.1.2 | Term | Microscopic polyangiitis |
E.1.2 | System Organ Class | 10047065 - Vascular disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
•To evaluate the efficacy of belimumab in the maintenance of remission following a standard induction regimen in subjects with Wegener’s granulomatosis (WG) or microscopic polyangiitis (MPA).
•To evaluate the safety of belimumab in subjects with WG or MPA.
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Title: Pharmacogenetic Research
Date: 22 June 2012
Objectives:
• Relationship between genetic variants and the pharmacokinetics of belimumab.
• Relationship between genetic variants and safety and/or tolerability of belimumab.
• Relationship between genetic variants and efficacy of belimumab. |
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E.3 | Principal inclusion criteria |
•Clinical diagnosis Wegener's granulomatosis or microscopic polyangiitis by Chapel Hill criteria.
•Disease flare in the past 26 weeks requiring treatment with high dose corticosteroids and 1 of the following medications: rituximab, oral cyclophosphamide OR IV cyclophosphamide.
•Tested positive for anti-proteinase 3 (anti-PR3) or anti-myeloperoxidase (anti-MPO) antibodies at any time prior to enrollment.
•Achieve remission no more than 26 weeks after first dose of induction treatment. Remission is defined as a Birmingham Vasculitis Activity (BVAS) score of 0 and receiving less than 10 mg/day of oral prednisone (or equivalent) on 2 consecutive visits at least 14 days apart, between 6 and 26 weeks after the first dose of induction therapy.
•Maintenance therapy on this study must start no more than 2 weeks after confirmation of remission.
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E.4 | Principal exclusion criteria |
•Pregnant or nursing.
•Receipt of a B cell targeted therapy (other than rituximab) at anytime
•Receipt of an investigational biological agent within the 60 days.
•Required management of acute or chronic infections within the past 60 days.
•Current drug or alcohol abuse or dependence.
•Current or past positive test for human immunodeficiency virus (HIV), hepatitis B, or hepatitis C or abnormal liver function tests.
•History of severe allergic reaction to contrast agents or biological medicines.
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary efficacy endpoint is time from Day 0 to the first relapse, defined as:
•At least 1 major BVAS item OR
•A minimum total BVAS score of 6 OR
•Receipt of protocol prohibited medications. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
12 months have elapsed after the last subject is randomised |
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E.5.2 | Secondary end point(s) |
Time from Day 0 to the first major relapse (defined as experiencing at least 1 major BVAS item) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
12 months have elapsed after the last subject is randomised |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 70 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Austria |
Belgium |
Canada |
Czech Republic |
Finland |
France |
Germany |
India |
Ireland |
Italy |
Mexico |
Peru |
Poland |
Romania |
Russian Federation |
Spain |
Sweden |
Switzerland |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 0 |