Myasthenia Gravis MedDRA version: 20.0 Level: PT Classification code 10028417 Term: Myasthenia gravis System Organ Class: 10029205 - Nervous system disorders
Conditions
Sponsors
Grifols Therapeutics Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Male or female, ages 18 to 85 years 2. Anti-AChR antibody positive 3. Confirmed diagnosis of generalized MG. Historically, subjects may have previously had the Myasthenia Gravis Foundation of America (MGFA) Class II, III, IV, or V. 4. MGFA classification of Class II, III, or IVa inclusive at Screening. 5. QMG score =10 at Screening. Note: Subjects who only have a history of ocular MG may not enroll. 6. Receiving standard of care MG treatment at a stable dose consisting of any one of the following for the time intervals delineated below (time intervals apply to medications and maintenance of stable dose level): -Cholinesterase inhibitor (pyridostigmine or equivalent) for at least 2 weeks prior to Screening and no immunosuppressants - Cholinesterase inhibitor (pyridostigmine or equivalent) for at least 2 weeks priorto Screening and/or only one of the following: * Prednisone (up to 60 mg/day or equivalent) for at least two months prior to Screening, or * Azathioprine for at least 6 months prior to Screening, or * Mycophenolate mofetil for at least 6 months prior to Screening, or *Methotrexate for at least 6 months prior to Screening, or * Cyclosporine or tacrolimus for at least 3 months prior to Screening - Cholinesterase inhibitor (pyridostigmine or equivalent) for at least 2 weeks prior to Screening and/or prednisone (up to 60 mg/day or equivalent) for at least one month prior to Screening and only one of the following: * Azathioprine for at least 6 months prior to Screening, or * Mycophenolate mofetil for at least 6 months prior to Screening, or *Methotrexate for at least 6 months prior to Screening, or * Cyclosporine or tacrolimus for at least 3 months prior to Screening 7. Subjects must be willing and able to provide written informed consent 8. Subjects must be willing to comply with all aspects of the clinical trial protocol. 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 56 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 6
Exclusion criteria
Exclusion criteria: 1. Have received cyclophosphamide or any other immunosuppressive agent apart from the ones allowed per inclusion criteria within the past 6 months 2. Any change in MG treatment regimen between Screening (Week -3, Visit 0) and Baseline (Week 0, Visit 1) 3. Greater than two (>2) point change in QMG score, increased or decreased, between Screening (Week -3, Visit 0) and Baseline (Week 0, Visit 1) 4. Any episode of myasthenic crisis in the one month prior to Screening 5. Evidence of malignancy within the past 5 years (nonmelanoma skin cancer, carcinoma in situ of cervix is allowed) or thymoma potentially requiring surgical intervention during the course of the trial (intent to perform thymectomy) 6. Thymectomy within the preceding six months 7. Rituximab, belimumab, eculizumab or any monoclonal antibody used for immunomodulation within the past 12 months 8. Have received immune globulin (Ig) treatment given by IV, subcutaneous, or intramuscular route within the last 3 months 9. Current known hyperviscosity or hypercoagulable state 10. Currently receiving anti-coagulation therapy (vitamin K antagonists, nonvitamin K antagonist oral anticoagulants [e.g., dabigatran etexilate, rivaroxaban, edoxaban, and apixaban], parenteral anticoagulants [e.g., fondaparinux]). Note that oral anti-platelet agents are allowed (e.g., aspirin, clopidogrel, ticlodipine) 11. Plasma exchange (PLEX) performed within the last 3 months 12. History of non-response to IVIg when used in maintenance therapy of the subject’s MG, as judged by the Investigator 13. Any comorbid condition that in the opinion of the Investigator would put the subject at undue safety risk or compromise the ability of the subject to participate in the trial or the scientific integrity of the study 14. Inadequate venous access to support repeated intravenous infusions 15. History of anaphylactic reactions or severe reactions to any blood-derived product 16. History of intolerance to any component of the IP 17. Documented diagnosis of thrombotic complications to polyclonal IVIg therapy in the past 18. History of recent (within the last year) myocardial infarction or stroke 19. Uncontrolled congestive heart failure; embolism; or historically documented (within the last year) electrocardiogram (ECG) changes indicative of myocardial ischemia or atrial fibrillation 20. History of chronic alcoholism or illicit drug abuse (addiction) in the 12 months preceding the Screening/Week -3 (Visit 0) 21. Active psychiatric illness that interferes with compliance or communication with health care personnel 22. Females of child-bearing potential who are pregnant or have a positive serum pregnancy test (beta-human chorionic gonadotropin [ß-HCG]-based assay) 23. Females who are breastfeeding 24. Females of child-bearing potential who are unwilling to practice a highly effective method of contraception (oral, injectable or implanted hormonal methods of contraception, placement of an intrauterine device or intrauterine system, condom or occlusive cap with spermicidal foam/gel/film/cream/suppository, male sterilization, or true abstinence*) throughout the study. * True abstinence: When this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence [e.g., calendar, ovulation, symptothermal, post-ovulation methods], declaration of abstinence for the duration of a trial, and withdrawal are not acceptable methods of contraception.) 25. Currently receiving, or having re
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to evaluate the efficacy of IGIV-C in subjects with generalized myasthenia gravis (MG) on standard of care treatment at study entry in terms of improvement in MG symptoms as measured by the mean change in Quantitative Myasthenia Gravis (QMG) score from Baseline (Week 0) to Week 24 as compared to placebo.;Secondary Objective: 1) Percentage of subjects who experience a clinical improvement assessed by QMG score from Baseline (Week 0) to Week 24 where clinical improvement is defined as at least a 3-point decrease in QMG score 2) Percentage of subjects who experience a clinical improvement assessed by the MG Composite from Baseline (Week 0) to Week 24 where clinical improvement is defined as at least a 3-point decrease in the MG Composite 3) Percentage of subjects who experience a clinical improvement assessed by MG –Activities of Daily Living (MG-ADL) from Baseline (Week 0) to Week 24 where clinical improvement is defined as at least a 2-point decrease in MG-ADL;Primary end point(s): The primary endpoint is improvement in MG symptoms as measured by the mean change in QMG score from Baseline (Week 0) to Week 24 as compared to placebo.;Timepoint(s) of evaluation of this end point: Throuhout the study from Baseline (Week 0) to Week 24. | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: 1) Throughout the study, from Baseline (Week 0) to Week 24 2) Throughout the study, from Baseline (Week 0) to Week 24 3) Throughout the study, from Baseline (Week 0) to Week 24 ;Secondary end point(s): 1) Percentage of subjects who experience a clinical improvement assessed by QMG score from Baseline (Week 0) to Week 24 where clinical improvement is defined as at least a 3-point decrease in QMG score 2) Percentage of subjects who experience a clinical improvement assessed by the MG Composite from Baseline (Week 0) to Week 24 where clinical improvement is defined as at least a 3-point decrease in the MG Composite 3) Percentage of subjects who experience a clinical improvement assessed by MG-ADL from Baseline (Week 0) to Week 24 where clinical improvement is defined as at least a 2-point decrease in MG-ADL | — |
Countries
Belgium, Canada, Czech Republic, Estonia, France, Germany, Hungary, Lithuania, Poland, United States
Contacts
Public ContactRhonda Griffin-Director,ClinDev
Grifols Therapeutics Inc.
Outcome results
None listed