Open-Label Extension Study of Zilucoplan in Subjects with Generalized Myasthenia Gravis

Phase 1

• Conditions: Generalized Myasthenia Gravis; MedDRA version: 20.0Level: LLTClassification code 10028415Term: MyastheniaSystem Organ Class: 100000004859; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2019-001565-33-IT
• Lead Sponsor: Ra Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-01-07
• Last Update Posted: 11 January 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 200

Inclusion Criteria

To be eligible for this study, subjects must meet ALL of the following inclusion criteria:
1.Completion of a qualifying Ra Pharmaceuticals 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. Postmenopausal women are, for the purposes of this protocol, defined as women who have gone 12 consecutive months without menstruation
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 150
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 50

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.Did not start 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) or any other reason that, in the opinion of the investigator or Sponsor, would disqualify the subject from participation in this study

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: •To provide access to zilucoplan for subjects with gMG who have completed a qualifying Ra Pharmaceuticals sponsored zilucoplan study and who wish to continue receiving zilucoplan<br>•To evaluate the long-term efficacy of zilucoplan in subjects with gMG who have completed a qualifying Ra Pharmaceuticals sponsored zilucoplan clinical study<br>•To evaluate the long-term safety and tolerability of zilucoplan in subjects with gMG who have completed a qualifying Ra Pharmaceuticals sponsored zilucoplan clinical study;Secondary Objective: na;Primary end point(s): Efficacy endpoints:<br>•MG-ADL Score<br>•QMG Score<br>•MG-QOL15r<br>•MGC Score<br>Safety endpoints;Timepoint(s) of evaluation of this end point: during each visit