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Ravulizumab

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Immunologic Factors

All participants will receive a weight-based loading dose of ravulizumab IV on Day 1, followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for participants weighing ≥ 20 kg, or once every 4 weeks (q4w) for participants weighing \< 20 kg, for a total of 122 weeks of treatment.

Ravulizumab will be administered by intravenous (IV) infusion.

Alexion Pharmaceuticals, Inc. (Sponsor)

The primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.

Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)

A Phase 3, Open-label, Single-arm, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants (6 to < 18 Years of Age) With Generalized Myasthenia Gravis (gMG)

Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

Participants ≥8 years of age will be evaluated.

Participants \<8 years of age will be evaluated.

Stable is defined as a ±5-point change from Baseline.

Stable is defined as a ±3-point change from baseline.

Alexion Pharmaceuticals, Inc.

Pharmaceutical Solutions

Participants will receive ravulizumab for the duration of the study.

Participants will receive placebo during the 26-week randomized-controlled period of the study, after which they will enter the open-label extension period of the study and receive ravulizumab.

Concentrated sterile, preservative-free aqueous solution (10 milligrams \[mg\]/milliliter \[mL\]) in single-use, 30-mL vial for intravenous (IV) infusion. Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight.

Matching, sterile, preservative-free aqueous solution in single-use, 30-mL vial for IV infusion. Single loading dose on Day 1, followed by regular maintenance dosing beginning on Day 15, based on weight.

Placebo

The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab for the treatment of participants with generalized myasthenia gravis (gMG).

All investigative site personnel, sponsor staff, sponsor designees, staff directly associated with the conduct of the study, and all participants will be blinded to treatment assignments.

Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis

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

The MG-ADL is an 8-point questionnaire that focused on relevant symptoms and functional performance of activities of daily living in participants with MG. The 8 items of the MGADL questionnaire were derived from symptom-based components of the original 13-item QMG scale to assess disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb (2 items) impairment related to effects from MG. In this functional status instrument, each response was graded 0 (normal) to 3 (most severe). The range of total MG-ADL score was 0 to 24. A decrease in score indicated improvement. Estimates were based on Mixed Effect Repeated Measures (MMRM) that included treatment group, stratification factor region, and MG-ADL total score at baseline, study visit, and study visit by treatment group interaction.

The QMG scoring system consisted of 13 items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item), and respiratory (1 item); each graded 0 to 3, with 3 being the most severe. The range of total QMG score is 0 to 39. The QMG scoring system was considered to be an objective evaluation of therapy for MG and was based on quantitative testing of sentinel muscle groups. A decrease in score indicated improvement. Estimates were based on MMRM that included treatment group, stratification factor region, and QMG total score at baseline, study visit, and study visit by treatment group interaction.

The QMG scoring system consisted of 13 items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item), and respiratory (1 item); each graded 0 to 3, with 3 being the most severe. The range of total QMG score is 0 to 39. A decrease in score indicated improvement. Percentage of participants with a ≥5-point reduction in the QMG total score are reported. Estimates were based on a generalized linear mixed model (GLMM) that included treatment group, stratification factor region and QMG total score at baseline, study visit and study visit by treatment group interaction.

The revised Myasthenia Gravis Qualify of Life 15-item scale (MG-QOL15r) is a health-related QoL evaluative instrument specific to participants with MG. The MG-QOL15r was designed to provide information about participants' perception of impairment and disability, determine the degree to which disease manifestations are tolerated, and to be administered and interpreted easily. Each item was graded on a scale of 0 to 2, with 2 being the most severe. The range of MG-QOL15r score is 0 to 30. Higher scores indicated greater extent of and dissatisfaction with MG-related dysfunction. Estimates are based on MMRM that included treatment group, stratification factor region and MG-QOL15r score at baseline, study visit and study visit by treatment group interaction.

The Neuro-QOL Fatigue is a reliable and validated brief 19-item survey of fatigue, completed by the participant. Each items was rated on a scale of 1 to 5, with 5 being the most severe. The range of total score is 19 to 95. Higher scores indicated greater fatigue and greater impact of MG on activities. Estimates were based on MMRM that included treatment group, stratification factor region and Neuro-QoL Fatigue score at baseline, study visit, and study visit by treatment group interaction.

The MG-ADL is an 8-point questionnaire that focused on relevant symptoms and functional performance of activities of daily living in participants with MG. The 8 items of the MGADL questionnaire were derived from symptom-based components of the original 13-item QMG scale to assess disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb (2 items) impairment related to effects from MG. In this functional status instrument, each response was graded 0 (normal) to 3 (most severe). The range of total MG-ADL score was 0 to 24. A decrease in score indicated improvement. Percentage of participants with a ≥3-point reduction in the MG-ADL total score are reported. Estimates were based on a GLMM that included treatment group, stratification factor region and MG-ADL total score at baseline, study visit and study visit by treatment group interaction.

Treatment-emergent adverse events were reported for 26-week randomized-controlled period and open-label extension period separately.

Participants received a weight-based single loading dose (2400 to 3000 mg) of ravulizumab IV on Day 1, followed by regular maintenance IV weight-based doses (3000 to 3600 mg) of ravulizumab beginning on Day 15 q8w during the 26-week Randomized-Controlled Period of the study.

Randomized-Controlled Period: Ravulizumab

Participants received a weight-based single loading dose of placebo IV on Day 1, followed by regular maintenance IV weight-based doses of placebo beginning on Day 15 q8w, during the 26-week Randomized-Controlled Period of the study.

Randomized-Controlled Period: Placebo

After the randomized-controlled part of the study, participants in the placebo group were transitioned to the ongoing open-label extension period to receive treatment with ravulizumab IV for up to 2 years.

Open-Label Extension Period: Placebo to Ravulizumab

After the randomized-controlled part of the study, participants in the ravulizumab group were transitioned to the ongoing open-label extension period to receive treatment with ravulizumab IV for up to 2 years.

Open-Label Extension Period: Ravulizumab to Ravulizumab

Participants received a weight-based single loading dose (2400 to 3000 mg) of ravulizumab IV on Day 1, followed by regular maintenance IV weight-based doses (3000 to 3600 mg) of ravulizumab beginning on Day 15 q8w during the 26-week Randomized-Controlled Period of the study. After the randomized-controlled part of the study, participants were transitioned to the ongoing open-label extension period to receive treatment with ravulizumab IV for up to 2 years.

Participants received a weight-based single loading dose of placebo IV on Day 1, followed by regular maintenance IV weight-based doses of placebo beginning on Day 15 q8w, during the 26-week Randomized-Controlled Period of the study. After the randomized-controlled part of the study, participants were transitioned to the ongoing open-label extension period to receive treatment with ravulizumab IV for up to 2 years.

Total

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

American Indian or Alaska Native

Asian

Native Hawaiian or Other Pacific Islander

Black or African American

White

Other

Unknown or Not Reported

Race/Ethnicity, Customized

Full Analysis Set: All randomized participants who received at least 1 dose of study drug.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)	NCT05644561	Generalized Mya... gMG	Ravulizumab	-	Alexion Pharmaceuticals, Inc.
Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab Administered Intravenously in Pediatric Participants With Generalized Myasthenia Gravis (gMG)	NCT05644561	Generalized Mya... gMG	Ravulizumab	-	Alexion Pharmaceuticals, Inc.
Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis	NCT03920293	Generalized Mya...	Ravulizumab Placebo	-	Alexion Pharmaceuticals, Inc.
Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis	NCT03920293	Generalized Mya...	Ravulizumab Placebo	-	Alexion Pharmaceuticals, Inc.

Spots Global Cancer Trial Database for ravulizumab

Every month we try and update this database with for ravulizumab cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for ravulizumab

Every month we try and update this database with for ravulizumab cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for ravulizumab cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.