Generalized Myasthenia Gravis MedDRA version: 20.0 Level: LLT Classification code 10028415 Term: Myasthenia System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
Conditions
Sponsors
Ra Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: To be eligible for this study, subjects must meet ALL of the following inclusion criteria: 1.Completion of a qualifying sponsored zilucoplan study 2.Able to provide informed consent, including signing and dating the informed consent form (ICF) 3.Vaccination with a quadrivalent meningococcal vaccine and, where available, meningococcal serotype B vaccine. A booster vaccination should also be administered as clinically indicated, according to the local SOC, for subjects who have been previously vaccinated against Neisseria meningitidis 4.Female subjects of childbearing potential must have a negative urine pregnancy test within 24 hours prior to the first dose of study drug 5.Sexually active female subjects of childbearing potential (i.e., women who are not postmenopausal or who have not had a hysterectomy, bilateral oophorectomy, or bilateral tubal ligation) and all male subjects (who have not been surgically sterilized by vasectomy) must agree to use effective contraception during the study and during the safety follow-up period of 40 days after the last dose of study drug. Postmenopausal women are, for the purposes of this protocol, defined as women who have not had menses for 12 months without an alternative medical cause. A high follicle stimulating hormone (FSH) level in the postmenopausal range may be used to confirm a postmenopausal state in women not using hormonal contraception or hormonal replacement therapy. However, in the absence of 12 months of amenorrhea, a single FSH measurement is insufficient. 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 165 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 55
Exclusion criteria
Exclusion criteria: Subjects who meet ANY of the following exclusion criteria must be excluded from the study: 1.Pregnant, planning to become pregnant, or nursing female subjects 2.With the exception of a prior zilucoplan trial, participation in another concurrent clinical trial involving an experimental therapeutic intervention (participation in observational studies and/or registry studies is permitted) 3.Unable or unwilling to comply with the requirements of the study 4.Commenced any disallowed medication per the exclusion criteria from the qualifying zilucoplan study or alter the dose of any other concomitant medication, unless medically indicated 5.Any new or worsening medical condition (since entry into the qualifying zilucoplan study) including active malignancy (except curatively resected squamous or basal cell carcinoma of the skin); significant medical disorder; psychiatric disorder; laboratory abnormality or any other reason that, in the opinion of the investigator or Sponsor, would disqualify the subject from participation in this study 6. Hypersensitivity to zilucoplan, placebo or any of its excipients
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: •To provide access to zilucoplan for subjects with gMG who have completed a qualifying sponsored zilucoplan study and who wish to continue receiving zilucoplan (not applicable for Norway) •To evaluate the long-term efficacy of zilucoplan in subjects with gMG who have completed a qualifying sponsored zilucoplan clinical study •To evaluate the long-term safety and tolerability of zilucoplan in subjects with gMG who have completed a qualifying sponsored zilucoplan clinical study ;Secondary Objective: Not applicable;Primary end point(s): Incidence of TEAEs;Timepoint(s) of evaluation of this end point: throughout the study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • CFB to Week E12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score • CFB to Week E12 in the Quantitative Myasthenia Gravis (QMG) Score • CFB to Week E12 in the Myasthenia Gravis Composite (MGC) Score • CFB to Week E12 in the Myasthenia Gravis – Quality of Life revised (MG-QOL15r) Score;Timepoint(s) of evaluation of this end point: week E12 | — |
Countries
Canada, France, Germany, Italy, Japan, Norway, Poland, Spain, United Kingdom, United States
Contacts
Public ContactClin Trial Reg & Results Disclosure
UCB Biosciences GmbH
Outcome results
None listed