A Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Patients 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: EUCTR2019-001564-30-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: 130

Inclusion Criteria

1.Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IV] at Screening
2.Positive serology for acetylcholine receptor (AChR) binding autoantibodies
3.MG-ADL Score of =6 at Screening and Baseline
4.QMG Score of =12 at Screening and Baseline (off acetylcholinesterase inhibitor therapy for at least 10 hours)
5.No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the 12-week treatment period
6.No change in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the 12-week treatment period
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 100
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 30

Exclusion Criteria

1.Thymectomy within 12 months prior to baseline or scheduled to occur during the 12-week study
2.History of meningococcal disease
3.Current or recent systemic infection within 2 weeks prior to Baseline or infection requiring intravenous (IV) antibiotics within 4 weeks prior to Baseline

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: •To confirm the efficacy of zilucoplan in subjects with gMG<br>•To confirm the safety and tolerability of zilucoplan in subjects with gMG;Secondary Objective: na;Primary end point(s): •Change from Baseline (CFB) to Week 12 in the MG-ADL Score;Timepoint(s) of evaluation of this end point: week 12

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): •CFB to Week 12 in the QMG Score<br>•CFB to Week 12 in the MG Composite (MGC)<br>•CFB to Week 12 in the MG-QOL15r Survey;Timepoint(s) of evaluation of this end point: week 12