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A randomized, double-blind, placebo-controlled phase III study to evaluate the efficacy, safety, and tolerability of iptacopan in patients with generalized Myasthenia Gravis (gMG), followed by an open label extension phase (CLNP023Q12301)

A study to investigate efficacy, safety and tolerability with iptacopan capsules compared with placebo capsules in participants aged 18 to 75 years with gMG. (CLNP023Q12301)

Maruyama Hideki

Novartis Pharma. K.K.

Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan

rinshoshiken.toroku@novartis.com

Maruyama Hideki

Novartis Pharma. K.K.

Toranomon Hills Mori Tower 23-1, Toranomon 1-chome Minato-ku, Tokyo 105-6333, Japan

+81-120-003-293

rinshoshiken.toroku@novartis.com

Recruiting

Sept. 13, 2024

Interventional

randomized controlled trial

double blind

placebo control

parallel assignment

treatment purpose

- Adult patients with generalized Myasthenia Gravis (age 18-75 years)
- Positive serology testing for AChR+ antibody at screening
- Myasthenia Gravis Foundation of America (MGFA) Class II-IV gMG 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 edrophonium chloride test
- 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 not optimally controlled for =>6 months on
- just one NSIST; or
- two or more NSISTs; or
- on frequent (at least quarterly) plasmapheresis, plasma exchange, or intravenous immunoglobulin to control symptoms despite treatment with steroids and NSISTs; or
- one of the following gMG treatments:
- a FcRN antagonist approved for gMG
- rituximab
- other approved gMG therapies excluding complement inhibitors.
- Consistent with all other iptacopan trials, participants will have to be vaccinated against Neisseria meningitidis and Streptococcus pneumoniae. In addition, participants will be vaccinated against Haemophilus influenzae, depending on the local regulations and on the availability of this vaccine in the countries of study conduct. The vaccination will be performed at least 2 weeks prior to first dosing with iptacopan, covering as many serotypes as possible. If iptacopan treatment will start earlier than 2 weeks post vaccination, prophylactic antibiotic treatment must be initiated and administered until 2 weeks post vaccination.

- Have been treated with intravenous immunoglobulin (IVIG)/plasma exchange (PLEX) in the past month, with rituximab in the past 6 months, eculizumab in the past 2 months, ravulizumab or other complement inhibitors in the past 3 months, efgartigimod or other anti-FcRn therapies in the past 3 months, or had a thymectomy in the past 6 months or a planned thymectomy during the trial period.
- Participants with clinically significant active or chronic uncontrolled bacterial, viral, or fungal infection at screening, including patients who test positive for an active viral infection at screening with: Active Hepatitis B Virus (HBV): serologic panel test results indicative of an active (acute or chronic) infection; Active Hepatitis C Virus (HCV): serology positive for HCV-Ab; Human Immunodeficiency Virus (HIV) positive serology associated with an Acquired Immune Deficiency Syndrome (AIDS)-defining condition or with a cluster of differentiation 4 (CD4) count =<200 cells/mm3
- Female participants who are pregnant or lactating, or are intending to become pregnant.
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of study treatment.
- Active systemic bacterial, viral (including COVID-19) or fungal infection or any major episode of infection that required hospitalization or injectable antimicrobial therapy within 14 days prior to study drug administration.
- History of recurrent invasive infections caused by encapsulated organisms, e.g., N. meningitidis and S. pneumoniae.
- Presence of fever =>38 degree Celsius (100.4 degree Fahrenheit) within 7 days prior to study drug administration

Both

generalized Myasthenia Gravis

Participants will be assigned to one of the following 2 treatment arms/groups in a ratio of 1:1
Iptacopan arm: iptacopan orally at 200 mg b.i.d. for 6 months (double-blind) followed by open-label iptacopan at 200 mg b.i.d. for an additional 24 months
Placebo arm: placebo orally b.i.d. for 6 months (double-blind) followed by open-label iptacopan at 200 mg b.i.d. for 24 months

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

+81-3-5712-5050

Mar. 13, 2024

Yes

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

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