Myasthenia Gravis MedDRA version: 8.1 Level: LLT Classification code 10028417 Term: Myasthenia gravis
Conditions
Interventions
Product Name: MONARSEN
Product Code: EN101
Pharmaceutical Form: Oral solution
Current Sponsor code: EN101
Other descriptive name: MONARS
Sponsors
Ester Neuroscience Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: To be considered eligible to participate in this study, a patient must meet the inclusion criteria listed below: (1) Clinical diagnosis of Myasthenia Gravis according to the MGFA (MG Foundation of America) classification being defined as a minimum of class II in whom it is safe to interrupt Mestinon treatment. (2) Patients (male or female) aged 18 years or above. (3) Patients must be seropositive for AChR antibodies (greater than 0.5 nM) or seropositive for anti MUSK antibodies. (Levels of anti MUSK greater then 0.05 fMol/L) (4) Laboratory Test of TSH and T4, within the normal range. (5) Previously treated with Mestinon (at least 3 tab. a day of 60mg each) until 12-18 hours prior to study initiation, with or without concomitant prednisolone or immunosuppressive treatment, which has been stable for at least 2 months. (6) Have hepatic and renal function, as well as coagulation parameters as documented by the following laboratory parameters, within local laboratory normal limits: – AST – Bilirubin – Creatinine – Platelets – Prothrombin time – Activated partial thromboplastin time (aPTT) (7) Women of childbearing potential are excluded Post-menopausal women (ie women who have been post-menopausal for at least 1 year) and women who have had a hysterectomy can be included. Men in relationship with women of childbearing potential, must agree to use effective contraceptive methods during the course of the study and for a 3 month follow-up period. (8) Have ability to understand the requirements of the study, have provided written informed consent, and agree to abide by the study restrictions and to return for the required assessments. (9) QMG score (while on Mestinon) > 3 points and QMG score while on washout > 3 points greater than score while on Mestinon. Are the trial subjects under 18? no 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) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: To be eligible for entry into the study, the patient must not meet any of the exclusion criteria listed below: (1) Patients have one of the following symptoms: acute respiratory failure/insufficiency, major difficulty in swallowing, functional disability responsible for the discontinuation of physical activity, within 1 month prior to screening. (2) Patients have exclusive mild ocular MG. (3) Body weight >100 kg (4) Patients in whom disease is so severe that it would be unethical to delay conventional therapy. (5) Have participated in any experimental protocol within the preceding one month. (6) Have received an IVIG infusion or plasmapheresis treatment, within 8 weeks prior to study initiation. (7) Abnormal CBC count (Hgb less than 12 g/dl, WBC less than 4,000/ml, or platelet count less than 130,000/ml). (8) Have developed other autoimmune disease within one month prior to study initiation. (9) Have uncontrolled hypertension, severe hepatic or renal disease, or other severe general or psychiatric disease. (10) Be pregnant or lactating.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Secondary Objective: - To assess the quality of life using the ADL-MG ; Main Objective: - To assess the efficacy of three doses of Monarsen given once daily for one week. Efficacy will be assessed by evaluating changes in the QMG score. - Safety will be assessed by evaluating adverse events and laboratory tests during a 3 week treatment period (dosing on alternative weeks with Mestinon, over 5 weeks) and a 4 week follow up period. ; Primary end point(s): Efficacy The study is not designed to achieve a certain power to detect effectiveness of the study drug. The efficacy analysis will therefore involve evaluating the degree of success with which the study drug ameliorates symptoms that appear when Monarsen treatment is given. Safety Changes from the screening period (prior to removal from Mestinon) of vital signs, hematology, clinical chemistry, urinalysis, ECG parameters and physical exam to the end of the study will be examined. Treatment emergent and/or clinically significant changes from normal to abnormal values in key laboratory parameters will be identified. | — |
Countries
United Kingdom
Outcome results
None listed