A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Pla… (NCT06517758) | Clinical Trial Compass
RecruitingPhase 3
A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG.
United States146 participantsStarted 2024-07-31
Plain-language summary
The study is a randomized, double-blind, placebo-controlled, multicenter, Phase III study, to evaluate efficacy, safety and tolerability of iptacopan in patients with AChR+ gMG who are on stable SOC treatment. Participants who meet the eligibility criteria will be randomized in a ratio of 1:1, to receive either iptacopan or matching placebo, for 6 months (180 days) while continuing on a stable SOC treatment. The randomization will be stratified based on region.
Who can participate
Age range18 Years – 85 Years
SexALL
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Inclusion Criteria:
* Adult patients with generalized Myasthenia Gravis (age 18-85 years) at screening
* Positive serology testing for AChR+ antibody at screening
* Myasthenia Gravis Foundation of America (MGFA) Class II-IV gMG at screening and likely not in need of a respirator for the duration of the study, as judged by the Investigator.
* The confirmation of the diagnosis of gMG should be documented and supported by ≥1 of the following 3 tests:
* History of abnormal neuromuscular transmission demonstrated by single-fiber electromyography or repetitive nerve stimulation.
* History of positive test with short-acting acetylcholinesterase inhibitors (e.g. neostigmine or edrophonium chloride)
* Patient has demonstrated improvement in MG signs on oral acetylcholinesterase inhibitors as assessed by the treating physician.
* Baseline MG-ADL score ≥6, with ≥50% of the total score due to non-ocular symptoms
* Participants receiving at least one of the following treatments for gMG for ≥ 6 months prior to baseline;
* One or more NSISTs or
* plasmapheresis, plasma exchange, or intravenous immunoglobulin (at least quarterly) to control symptoms despite treatment with steroids and NSISTs; or
* an approved FcRN antagonist approved for gMG; or
* rituximab or
* other approved gMG disease modifying therapies excluding complement inhibitors.
* Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the start of study treatment. If the participan…
What they're measuring
1
Change from baseline to Month 6 in Myasthenia Gravis Activity of Daily Living (MG-ADL) total score