Patients affected with MG showing clinically meaningful improvement after therapeutic plasmapheresis. MedDRA version: 9.1 Level: LLT Classification code 10028417 Term: Myasthenia gravis
Conditions
Interventions
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:
Sponsors
ISTITUTO NEUROLOGICO "CARLO BESTA"
Eligibility
Sex/Gender
All
Inclusion criteria
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
Exclusion criteria
Exclusion criteria: ocular MG (MGFA class I) MG crisis thymectomy in the last 3 months immunosuppressive therapy in the last 3 months other than corticosteroids subjects with history of myocardial infarction subjects with clinically evident cardiac disease (ischemic heart disease or congestive heart failure) subjects with uncontrolled hypertension contraindication to plasmapheresis subjects with active malignancy female subjects who are pregnant or lactating subjects who have received an experimental drug or have participated in a clinical trial within 3 months prior to screening employees of the investigator or study centre with direct involvement in the proposed study or other studies under the direction of that investigator or study centre.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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.;Secondary Objective: Select appropriate dose schedules to be further investigated with long-term confirmative studies.;Primary end point(s): Primary evaluation parameter will be the QMGS (Quantitative Myasthenia Gravis Score) | — |
Countries
Italy
Outcome results
None listed