A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 75 Years With gMG.
- Conditions
- Generalized Myasthenia Gravis
- Registration Number
- NCT06517758
- Lead Sponsor
- Novartis Pharmaceuticals
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- Not specified
Inclusion Criteria:<br><br> - Adult patients with generalized Myasthenia Gravis (age 18-75 years)<br><br> - Positive serology testing for AChR+ antibody at screening<br><br> - Myasthenia Gravis Foundation of America (MGFA) Class II-IV gMG and likely not in<br> need v of a respirator for the duration of the study, as judged by the Investigator.<br><br> - The confirmation of the diagnosis of gMG should be documented and supported by =1 of<br> the following 3 tests:<br><br> - History of abnormal neuromuscular transmission demonstrated by single-fiber<br> electromyography or repetitive nerve stimulation.<br><br> - History of positive edrophonium chloride test<br><br> - Patient has demonstrated improvement in MG signs on oral acetylcholinesterase<br> inhibitors as assessed by the treating physician.<br><br> - Baseline MG-ADL score =6, with =50% of the total score due to non-ocular symptoms<br><br> - Participants not optimally controlled for = 6 months on<br><br> - just one NSIST; or<br><br> - two or more NSISTs; or<br><br> - on frequent (at least quarterly) plasmapheresis, plasma exchange, or intravenous<br> immunoglobulin to control symptoms despite treatment with steroids and NSISTs; or<br><br> - one of the following gMG treatments:<br><br> - a FcRN antagonist approved for gMG<br><br> - rituximab<br><br> - other approved gMG therapies excluding complement inhibitors.<br><br> - Consistent with all other iptacopan trials, participants will have to be vaccinated<br> against Neisseria meningitidis and Streptococcus pneumoniae. In addition,<br> participants will be vaccinated against Haemophilus influenzae, depending on the<br> local regulations and on the availability of this vaccine in the countries of study<br> conduct. The vaccination will be performed at least 2 weeks prior to first dosing<br> with iptacopan, covering as many serotypes as possible. If iptacopan treatment will<br> start earlier than 2 weeks post vaccination, prophylactic antibiotic treatment must<br> be initiated and administered until 2 weeks post vaccination.<br><br>Exclusion Criteria:<br><br> - Have been treated with intravenous immunoglobulin (IVIG)/plasma exchange (PLEX) in<br> the past month, with rituximab in the past 6 months, eculizumab in the past 2<br> months, ravulizumab or other complement inhibitors in the past 3 months,<br> efgartigimod or other anti- FcRn therapies in the past 3 months, or had a thymectomy<br> in the past 6 months or a planned thymectomy during the trial period.<br><br> - Participants with clinically significant active or chronic uncontrolled bacterial,<br> viral, or fungal infection at screening, including patients who test positive for an<br> active viral infection at screening with: Active Hepatitis B Virus (HBV): serologic<br> panel test results indicative of an active (acute or chronic) infection; Active<br> Hepatitis C Virus (HCV): serology positive for HCV-Ab; Human Immunodeficiency Virus<br> (HIV) positive serology associated with an Acquired Immune Deficiency Syndrome<br> (AIDS)-defining condition or with a cluster of differentiation 4 (CD4) count<br><br> - 200 cells/mm3<br><br> - Female participants who are pregnant or lactating, or are intending to become<br> pregnant.<br><br> - Women of child-bearing potential, defined as all women physiologically capable of<br> becoming pregnant, unless they are using effective methods of contraception during<br> dosing of study treatment.<br><br> - Active systemic bacterial, viral (including COVID-19) or fungal infection or any<br> major episode of infection that required hospitalization or injectable antimicrobial<br> therapy within 14 days prior to study drug administration.<br><br> - History of recurrent invasive infections caused by encapsulated organisms, e.g., N.<br> meningitidis and S. pneumoniae.<br><br> - Presence of fever = 38 °C (100.4 °F) within 7 days prior to study drug<br> administration
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change from baseline to Month 6 in Myasthenia Gravis Activity of Daily Living (MG-ADL) total score
- Secondary Outcome Measures
Name Time Method