A PHASE II, PLACEBO CONTROLLED, DOSE FINDING PILOT STUDY OF PIXANTRONE EFFICACY ADMINISTERED INTRAVENOUSLY IN PATIENTS AFFECTED WITH MYASTHENIA GRAVIS

INTERVENTION: Product Name: PIXANTRONE Product Code: BBR 2778 Pharmaceutical Form: Powder and solvent for suspension for injection INN or Proposed INN: pixantrone Current Sponsor code: BBR 2778 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 50‐ Pharmaceutical form of the placebo: Solvent for parenteral use Route of administration of the placebo: Intravenous use CONDITION: Patients affected with MG showing clinically meaningful improvement after therapeutic plasmapheresis. ; MedDRA version: 9.1 Level: LLT Classification code 10028417 Term: Myasthenia gravis PRIMARY OUTCOME: Main Objective: The primary objective of this pilot study is to obtain preliminary data on the potential therapeutic activity of PIX in patient affected with MG in a short‐term. Tolerability and safety will be assessed for all subjects for the entire duration of the study. Primary end point(s): Primary evaluation parameter will be the QMGS (Quantitative Myasthenia Gravis Score) Secondary Objective: Select appropriate dose schedules to be further investigated with long‐term confirmative studies. INCLUSION CRITERIA: caucasian male or female aged 18 to 55 years inclusive diagnosis of MG classes II, III or IV according to MGFA classification (MG Foundation of America) positive acetylcholine receptor antibodies (AChR Ab) LVEF ≥ 55% no conditions known to be contraindications to the use of PIX written informed consent. Positive response to plasmapheresis, i.e reduction of QMGS of at least 3,5 points (at randomization visit) Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18‐64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range