A Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Patients with Generalized Myasthenia Gravis

A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects with Generalized Myasthenia Gravis - RAISE

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2019-001564-30-DE

Enrollment

156

Registered

2019-10-01

Start date

2020-03-31

Completion date

Unknown

Last updated

2022-03-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Generalized Myasthenia Gravis MedDRA version: 20.0 Level: LLT Classification code 10028415 Term: Myasthenia System Organ Class: 100000004859

Interventions

Product Name: Zilucoplan Pharmaceutical Form: Solution for injection in pre-filled syringe INN or Proposed INN: Zilucoplan CAS Number: 1841136-73-9 Current Sponsor code: RA101495 Concentration unit: m

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: To be eligible for this study, subjects must meet ALL the following 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 110 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 46

Exclusion criteria

Exclusion criteria: Subjects who meet ANY of the following exclusion criteria must be excluded from the study: 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

Design outcomes

Primary

Measure	Time frame
Main Objective: •To confirm the efficacy of zilucoplan in subjects with gMG •To confirm the safety and tolerability of zilucoplan in subjects with gMG ;Secondary Objective: Not Applicable;Primary end point(s): •Change from Baseline (CFB) in Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score;Timepoint(s) of evaluation of this end point: week 12	—

Secondary

Measure	Time frame
Secondary end point(s): 1. CFB to Week 12 in the Quantitative Myasthenia Gravis (QMG) Score 2. CFB to Week 12 in the Myasthenia Gravis Composite (MGC) Score 3. CFB to Week 12 in the Myasthenia - -Gravis – Quality of Life revised (MG-QOL15r) Score 4. Time to first receipt of rescue therapy over the 12-week Treatment Period 5. Achieving Minimal Symptom Expression (MSE), defined as an MG-ADL of 0 or 1 at Week 12 without rescue therapy 6. Achieving a = 3-point reduction in MG-ADL Score at Week 12 without rescue therapy 7. Achieving a =5-point reduction in QMG Score without rescue therapy at Week 12 8. Incidence of treatment-emergent AEs ;Timepoint(s) of evaluation of this end point: Endpoints 1-7: Week 12 Endpoint 8: Throughout the study	—

Measure

Time frame

Secondary end point(s): 1. CFB to Week 12 in the Quantitative Myasthenia Gravis (QMG) Score 2. CFB to Week 12 in the Myasthenia Gravis Composite (MGC) Score 3. CFB to Week 12 in the Myasthenia - -Gravis – Quality of Life revised (MG-QOL15r) Score 4. Time to first receipt of rescue therapy over the 12-week Treatment Period 5. Achieving Minimal Symptom Expression (MSE), defined as an MG-ADL of 0 or 1 at Week 12 without rescue therapy 6. Achieving a = 3-point reduction in MG-ADL Score at Week 12 without rescue therapy 7. Achieving a =5-point reduction in QMG Score without rescue therapy at Week 12 8. Incidence of treatment-emergent AEs ;Timepoint(s) of evaluation of this end point: Endpoints 1-7: Week 12 Endpoint 8: Throughout the study

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Countries

Canada, France, Germany, Italy, Japan, Korea, Republic of, Norway, Poland, Spain, United Kingdom, United States

Contacts

Public ContactClin Trial Reg & Results Disclosure

UCB Biosciences GmbH

clinicaltrials@ucb.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026