E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients with ANCA-associated Vasculitis |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10047888 |
E.1.2 | Term | Wegener's granulomatosis |
|
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Response to treatment will be measured by the relapse rates in patients who have achieved remission over 24 month study period. |
|
E.2.2 | Secondary objectives of the trial |
Response to treatment will also be measured by: The proportion of patients in sustained remission at 6, 12, 18 months and 24 months; The time to remission; The average steroid dosage at 6, 12,18 and 24 months; The time to ANCA negativity by immunofluorescence or negative anti-PR3 or anti-MPO Ab test by ELISA; Urinary MCP-1 measurement to assess disease activity. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Males and females (not nursing and not pregnant) at least 18 years of age. Women of child bearing potential are eligible if they are practicing effective contraceptive measures With Acute AAV, presenting at first diagnosis or relapse (not grumblers, to maximize effect seen), defined by clinical presentation with Wegeners Granulomatosis (WG), microscopic polyangiitis (MPA) or Churg-Strauss syndrome (CSS)* and ANCA positivity (anti-MPO or anti-PR3 positive) or historical ANCA positivity, and a BVAS score of > 8. Written informed consent given |
|
E.4 | Principal exclusion criteria |
Those with severe life-threatening disease, i.e. lung haemorrhage at the time of presentation, renal impairment with SCr>150 mcmol/l, or severe CNS dysfunction thought to be due to vasculitis. Subjects with current symptoms of severe, progressive, or uncontrolled renal, hepatic, hematological, gastrointestinal, pulmonary, cardiac, neurological or cerebral disease, or other medical conditions that, in the opinion of the investigator, might place the subject at unacceptable risk for participation in this study. With any other non-vasculitic multisystem autoimmune disease. With any serious acute bacterial infection unless treated and completely resolved with antibiotics prior to enrollment With any severe chronic or recurrent bacterial infection, e g. bronchiectasis, osteomyelitis, chronic pyelonephritis With Hepatitis B or C or HIV. With Herpes zoster infection that resolved less than 2 months prior to enrollment. Subjects who have received any live vaccines* within 3 months of the first dose of study medication or who will have need of a live vaccine at any time in the year following enrollment. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Response to treatment will be measured by the relapse rates in patients who have achieved remission over 24 month study period. |
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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 | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
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 | No |
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.8.3 |
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 18 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | No |
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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 0 |