A randomized, double-blind, placebo-controlled phase III study to evaluate the efficacy, safety, and tolerability of iptacopan in patients with generalized Myasthenia Gravis, followed by an open label extension phase

Conditions

generalized Myasthenia Gravis

Brief summary

Change from baseline to Month 6 in Myasthenia Gravis Activity of Daily Living (MG-ADL) total score

Detailed description

Change from baseline to Month 6 in Quantitative MG (QMG) total score, Proportion of participants with ≥5 points reduction from baseline to Month 6 of QMG total score without rescue medication and/or strongly confounding prohibited medication, Proportion of participants with ≥3 points reduction from baseline to Month 6 of MG-ADL total score without rescue medication and/or strongly confounding prohibited medication, Change from baseline to Month 6 in Myasthenia Gravis Composite (MGC) total score, Change from baseline to Month 6 in revised MG Quality of Life Questionnaire (MG-QOL15r) survey score, Incidence of adverse events and changes in clinical laboratory values, vital signs, and electrocardiograms, Columbia Suicide Severity Rating, Proportion of time during which participants showed a reduction of ≥ 2 points in MG-ADL total score that was maintained up to Month 6, Proportion of early MG-ADL responders during treatment (early responders with first MG-ADL improvement from baseline of ≥2 points occurring by week 4）, Change from baseline to Month 6 in EuroQol-5 Dimensions-5 Level (EQ-5D-5L), Change from baseline in MG-ADL total score, Proportion of participants achieving a reduction in oral corticosteroids (OCS) dose compared to Core Part that was maintained up to end of Extension Part, Incidence of adverse events and changes in clinical laboratory values, vital signs, and electrocardiograms, Columbia Suicide Severity Rating, Proportion of participants achieving MSE at Month 6, defined as MG-ADL score of 0 or 1 at Month 6 without rescue therapy and/or strongly confounding prohibited medication, Proportion of participants with reduction of ≥ 3 points from baseline to Month 6 in MGC total score

Related papers

No related papers found yet.

Papers published before 2007-09-01 may not appear yet. Historical indexing is in progress.

Interventions

DRUGIPTACOPAN

DRUGPlacebo 0 mg hard gelatin capsule size 0

DRUG(placebo to LNP023)

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Change from baseline to Month 6 in Myasthenia Gravis Activity of Daily Living (MG-ADL) total score	—

Secondary

Measure	Time frame
Change from baseline to Month 6 in Quantitative MG (QMG) total score, Proportion of participants with ≥5 points reduction from baseline to Month 6 of QMG total score without rescue medication and/or strongly confounding prohibited medication, Proportion of participants with ≥3 points reduction from baseline to Month 6 of MG-ADL total score without rescue medication and/or strongly confounding prohibited medication, Change from baseline to Month 6 in Myasthenia Gravis Composite (MGC) total score, Change from baseline to Month 6 in revised MG Quality of Life Questionnaire (MG-QOL15r) survey score, Incidence of adverse events and changes in clinical laboratory values, vital signs, and electrocardiograms, Columbia Suicide Severity Rating, Proportion of time during which participants showed a reduction of ≥ 2 points in MG-ADL total score that was maintained up to Month 6, Proportion of early MG-ADL responders during treatment (early responders with first MG-ADL improvement from baseline of	—

Measure

Time frame

Change from baseline to Month 6 in Quantitative MG (QMG) total score, Proportion of participants with ≥5 points reduction from baseline to Month 6 of QMG total score without rescue medication and/or strongly confounding prohibited medication, Proportion of participants with ≥3 points reduction from baseline to Month 6 of MG-ADL total score without rescue medication and/or strongly confounding prohibited medication, Change from baseline to Month 6 in Myasthenia Gravis Composite (MGC) total score, Change from baseline to Month 6 in revised MG Quality of Life Questionnaire (MG-QOL15r) survey score, Incidence of adverse events and changes in clinical laboratory values, vital signs, and electrocardiograms, Columbia Suicide Severity Rating, Proportion of time during which participants showed a reduction of ≥ 2 points in MG-ADL total score that was maintained up to Month 6, Proportion of early MG-ADL responders during treatment (early responders with first MG-ADL improvement from baseline of

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Countries

Denmark, France, Germany, Greece, Italy, Netherlands, Poland, Portugal, Spain

Outcome results

None listed