E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
refractory bullous pemphigoid |
Pemphigoïde bulleuse réfractaire |
|
E.1.1.1 | Medical condition in easily understood language |
Bullous pemphigoid |
Pemphigoïde bulleuse |
|
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 | 10006567 |
E.1.2 | Term | Bullous pemphigoid |
E.1.2 | System Organ Class | 100000004858 |
|
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 |
To demonstrate the efficacy of QGE031 240mg q2w relative to placebo at 12 weeks in patients with BP by reducing disease activity as determined by Clinical Global Assessment of Change (CGA-C) responder rate. |
Démontrer l’efficacité du QGE031 comparé au placebo après 12 semaines chez les patients atteints de PB en réduisant l’activité de la maladie comme déterminé par le Clinical Global Assessment of Change (CGA-C) – taux de réponse |
|
E.2.2 | Secondary objectives of the trial |
1.To evaluate the CGA-C responder rate in QGE031 240mg q2w vs. Placebo treated patients at 6 weeks 2.To evaluate the effect of QGE031 240mg q2w in BP patients as assessed by Investigator Global Assessment (IGA) 3.To evaluate the safety of QGE031 in BP patients
|
1. Evaluer le taux de réponse CGA-C des patients traités au QGE031 comparé aux patients traités au placebo après 6 semaines.
2. Evaluer les effets du QGE031 chez les patients atteints de la PB, évalués selon l’IGA (Investigator Global Assessment)
3. Evaluer la sécurité du QGE031 chez les patients atteints de PB.
|
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Patients diagnosed with bullous phemphigoid - Stable dose of prednisone at or above 10mg per day but no greather than 1mg/kg/day - Weight between 40-120kg - Total IgE level up to 5000 IU/mL
listing incomplete, additional inclusion criteria applicable |
- patients atteints de PB - dose stable de prednisone de ou plus de 10 mg par jour mais n'excédant pas 1 mg/kg/jour - poids entre 40 - 120 kg - taux total IgE jusqu'à 5000 IU/ml
liste incomplète, critères d'inclusion supplémentaires valables |
|
E.4 | Principal exclusion criteria |
- Use of rifuximab within 1 year
listing incomplete, additional exclusion criteria applicable |
- Utilisation de rituximab jusqu’à un an précédent la visite initiale.
liste incomplète, critères d'exclusion supplémentaires valables |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Change in the Clinical Global Assessment of change from baseline to week 12. |
changement dans le Clinical Global Assessment of change de la base de référence/ visite de base à la 12ème semaine. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
- Change in the Clinical Global Assessment of change from baseline to week 6. - Change from baseline in the Investigator Global Assessment over 48 weeks. - Safety of QGE031 over 48 weeks. |
- changement dans le Clinical Global Assessment of change de la visite de base à la 6ème semaine. - changement de la base de référence dans l’évaluation globale de l’investigateur après 48 semaines. - sécurité du QGE031 après 48 semaines.
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
- 6 weeks - baseline, every 2 weeks up to 12 weeks, every 4 weeks up to 24 weeks, every 8 weeks up to 48 weeks - baseline, every 2 weeks up to 12 weeks, every 4 weeks up to 24 weeks, every 8 weeks up to 48 weeks
|
- 6 semaines - baseline, toutes les 2 semaines jusqu'à 12 semaines, toutes les 4 semaines jusqu'à 24 semaines, toutes les 8 semaines jusqu'à 48 semaines - baseline, toutes les 2 semaines jusqu'à 12 semaines, toutes les 4 semaines jusqu'à 24 semaines, toutes les 8 semaines jusqu'à 48 semaines
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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 | 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 |
PRO tool |
PRO (questionnaires VAS) |
|
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 | 4 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 10 |
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 |
|
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
|
LPLV |
dernier patient dernière visite |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |