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.

• Register: EUCTR
• Last refreshed on: 21 October 2024
• Main ID: EUCTR2018-003243-39-IT
• Date of registration: 22/01/2021
• Prospective Registration: No
• Primary sponsor: ALEXION PHARMACEUTICALS INCORPORATED
• Public title: 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.
• Scientific title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis - NA
• Date of first enrolment: 17/06/2019
• Target sample size: 158
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2018-003243-39
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Austria, Belgium, Canada, Czech Republic, Czechia, Denmark, France, Germany, Israel, Italy, Japan, Korea, Republic of, Netherlands, Portugal, Russian Federation, Spain, Switzerland, United Kingdom, United States

Contacts

• Name: European Clinical Trial Information
• Affiliation: Alexion Europe SAS

• Name: European Clinical Trial Information
• Affiliation: Alexion Europe SAS

Key inclusion & exclusion criteria

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 14

Exclusion criteria:
Medical Conditions:
• Any active or untreated thymoma. History of thymic carcinoma or thymic malignancy
• 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 complementinhibitors
(eg, eculizumab


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Generalized Myasthenia Gravis
MedDRA version: 20.0 Level: HLT Classification code 10071942 Term: Myasthenia gravis and related conditions System Organ Class: 100000004859

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Trade Name: Ultomiris
Product Name: Ravulizumab
Product Code: [ALXN1210]
Pharmaceutical Form: Concentrate for solution for infusion
Current Sponsor code: ALXN1210
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Concentrate for solution for infusion
Route of administration of the placebo: Intravenous use

Primary Outcome(s)

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.
- To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on the improvement in quality of life measures.
- To assess the efficacy of ravulizumab compared with placebo in the treatment of gMG based on other efficacy endpoints.

Timepoint(s) of evaluation of this end point: At Week 26 of the Randomized-Controlled Period.

Primary end point(s): Change from Baseline in MG-ADL total score

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: At Week 26

Secondary end point(s): • Change from Baseline in QMG total score.
• Change from Baseline in the Revised 15-Component Myasthenia Gravis Quality of Life (MG-QOL15r) score
• Change from Baseline in Neuro-QOL Fatigue score
• Improvement from Baseline of at least 3 points in the MG-ADL total score
• Improvement from Baseline of at least 5 points in the QMG total score

Secondary ID(s)

ALXN1210-MG-306

2018-003243-39-DE

Source(s) of Monetary Support

Alexion Pharmaceuticals Incorporated

Ethics review

Status: Approved
Approval date: 03/04/2019
Contact:

Results

• Results available: Yes
• Date Posted: 03/06/2022
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2018-003243-39/results