E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Myasthenia Gravis
Systemic Lupus Erythematosus
Rheumatoid Arthritis
|
Myasthenia Gravis
Systemischer Lupus Erythematosus
Rheumatoide Arthritis
|
|
E.1.1.1 | Medical condition in easily understood language |
Myasthenia Gravis
Systemic Lupus Erythematosus
Rheumatoid Arthritis
|
Myasthenia Gravis
Systemischer Lupus Erythematosus
Rheumatoide Arthritis
|
|
E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
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 investigate the change in respective disease-specific antibody-titers after application of Bortezomib. Proof-of-concept study. |
Untersuchung der Veränderung des jeweiligen krankheitsspezifischen Antikörper-Titers nach Applikation von Bortezomib |
|
E.2.2 | Secondary objectives of the trial |
To investigate the impact of Bortezomib on clinical scores, co-medicaton, need for hospitalisation, quality of life, immunological parameter in the course of disease and treatment |
klinische Scores, Co-Medikation, Notwendigkeit stationärer Aufnahmen, Lebensqualität, immunologische Parameter (Zahl von aktivierten Plasmazellen) im Verlauf der Erkrankung und Therapie |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- age 18 - 75 years at screening
- ability to give written consent, informed wirtten consent
- negative pregnancy test at screening
- therapy-refractory Myasthenia Gravis (generalized) or Systemic Lupus Erythematosus or Rheumatoid Arthritis
|
|
E.4 | Principal exclusion criteria |
- known intolerability to Bortezomib
- participation in another interventional trial within the last 3 months
- heart or kidney insufficiency
- liver cirrhosis |
|
E.5 End points |
E.5.1 | Primary end point(s) |
change in disease specific antibody titers after application of Bortezomib compared to baseline |
Veränderung des jeweiligen krankheitsspezifischen Auto-Antikörper-Titers nach Applikation von Bortezomib |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
After 6 months observations after 2 cycles |
|
E.5.2 | Secondary end point(s) |
- clinical scores (MG-Qol, MG-ADL, Lupus-Qol, RA-Qol, HAQ)
- co-medication
- need for hospitalisation
- quality of life (Activities of Daily Living)
- immunological parameter in the course of disease and treatment |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
at every visit - during 6 months - at the optional follow-up visits at month 12 and 18 |
|
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 | No |
E.6.5 | Efficacy | No |
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
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 | No |
E.8.2.3 | Other | No |
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 | No |
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 | No |
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
|
LVLS (plus 2 optional follow-up visits) |
|
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 | 0 |
E.8.9.1 | In the Member State concerned days | |