Study to evaluate the safety and efficacy of ravulizumab in adult patients with generalized Myasthenia Gravis who have never been treated with a complement inhibitor.

Phase 1

• Conditions: Generalized Myasthenia Gravis; MedDRA version: 20.0Level: HLTClassification code 10071942Term: Myasthenia gravis and related conditionsSystem Organ Class: 100000004859; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2018-003243-39-NL
• Lead Sponsor: Alexion Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-05-01
• Last Update Posted: 18 July 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 175

Inclusion Criteria

• Male and female patients = 18 years of age
• Diagnosed with MG at least 6 months (180 days) prior to the date of the Screening Visit as confirmed by protocol-specific criteria
• Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at screening
• MG-ADL profile must be = 6 at screening and randomization (Day 1)
• Vaccinated against meningococcal infections within 3 years prior to, or at the time of, initiating study drug to reduce the risk of meningococcal infection (N meningitidis).
• Body weight = 40 kg at the time of screening
• Patients of childbearing potential and patients with partners of childbearing potential must follow protocol-specified contraception guidance for avoiding pregnancy while on treatment and for 8 months after last dose of study drug

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 144
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 16

Exclusion Criteria

Medical Conditions:
• Any active or untreated thymoma. History of thymic carcinoma or thymic malignancy unless deemed cured by adequate treatment with no evidence of recurrence for = 5 years before Screening
• History of thymectomy, thymomectomy, or any thymic surgery within the 12 months prior to screening
• History of N meningitidis infection
• Human immunodeficiency virus (HIV) infection
• History of hospitalization for = 24 hours, for any reason, within the 4 weeks (28 days) prior to screening
• Females who plan to become pregnant during the study, or are currently pregnant or breastfeeding, or who have a positive pregnancy test result at screening or on Day 1
• History of unexplained infections
• Active systemic bacterial, viral, or fungal infection within 14 days prior to study drug administration on Day 1
• Presence of fever = 38°C (100.4°F) within 7 days prior to study drug administration on Day 1

Prior/Concomitant Therapy
•Use of the following within the time period specified below:
- IVIg within the 4 weeks (28 days) prior to randomization (Day 1)
- Use of PE within the 4 weeks (28 days) prior to randomization (Day 1)
- Use of rituximab within the 6 months (180 days) prior to screening
• Patients who have received previous treatment with complement-inhibitors (eg, eculizumab)

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) profile.;Secondary Objective: • To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in the Quantitative Myasthenia Gravis (QMG) total score.<br>• To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in quality of life measures.<br>• To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on other efficacy endpoints.;Primary end point(s): Change from Baseline in MG-ADL total score ;Timepoint(s) of evaluation of this end point: At Week 26 of the Randomized-Controlled Period.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): • Change from Baseline in QMG total score<br>• Change from Baseline in the Revised 15-Component Myasthenia Gravis Quality of Life (MG-QoL15r) score<br>• Change from Baseline in Neuro-QoL Fatigue score <br>• Improvement from Baseline of at least 3 points in the MG-ADL total score<br>• Improvement from Baseline of at least 5 points in the QMG total score;Timepoint(s) of evaluation of this end point: At Week 26