Generalized Myasthenia Gravis MedDRA version: 20.0 Level: HLT Classification code 10071942 Term: Myasthenia gravis and related conditions System Organ Class: 100000004859
Conditions
Interventions
Trade Name: Ultomiris
Product Name: Ravulizumab
Product Code: ALXN1210
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibod
Sponsors
Alexion Pharmaceuticals Incorporated
Eligibility
Sex/Gender
All
Inclusion 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 16
Exclusion criteria
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)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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.;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
| Measure | Time frame |
|---|---|
| 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;Timepoint(s) of evaluation of this end point: At Week 26 | — |
Countries
Austria, Canada, Czechia, Czech Republic, Denmark, France, Germany, Israel, Italy, Japan, Korea, Republic of, Netherlands, Portugal, Spain, Switzerland, United Kingdom, United States
Contacts
Public ContactEuropean Clinical Trial Information
Alexion Europe SAS
Outcome results
None listed