Trial Title:
A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 75 Years With gMG.
NCT ID:
NCT06517758
Condition:
Generalized Myasthenia Gravis
Conditions: Official terms:
Myasthenia Gravis
Conditions: Keywords:
Myasthenia Gravis
gMG
randomized
placebo-controlled
double-blind
phase III
AChR+
SOC treatment
iptacopan
LNP023
Study type:
Interventional
Study phase:
Phase 3
Overall status:
Recruiting
Study design:
Allocation:
Randomized
Intervention model:
Parallel Assignment
Intervention model description:
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.
Primary purpose:
Treatment
Masking:
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking description:
This is a participant, investigator, and sponsor-blinded study. Participants,
investigator staff, persons performing the assessments and the Clinical Trial Team will
remain blinded to the identity of treatment from the time of randomization until database
lock after all participants have completed the double-blind treatment period. The
following methods will be used to ensure that blinding is properly maintained:
1. Randomization data are kept strictly confidential until the time of unblinding and
will not be accessible by anyone involved in the study
2. The identity of the treatment will be concealed by the use of study treatments that
are all identical in packaging, labeling, schedule of administration, appearance,
taste, and odor
Intervention:
Intervention type:
Drug
Intervention name:
Iptacopan
Description:
Hard gelatin capsule
Arm group label:
Iptacopan
Intervention type:
Other
Intervention name:
Matching Placebo
Description:
Hard gelatin capsule
Arm group label:
Matching Placebo
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.
Detailed description:
The study consists of a 6-month double-blind treatment period for the primary efficacy
and safety analysis followed by a 24 month open label extension period. A safety follow
up assessment will be performed, one 7 days after the last administration of study
treatment and one 30 days after the last administration of study treatment for all
participants.
Criteria for eligibility:
Criteria:
Inclusion Criteria:
- 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 v 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.
Exclusion Criteria:
- 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 °C (100.4 °F) within 7 days prior to study drug
administration
Gender:
All
Minimum age:
18 Years
Maximum age:
75 Years
Healthy volunteers:
No
Locations:
Facility:
Name:
Fullerton Neuro and Headache Ctr .
Address:
City:
Fullerton
Zip:
92835
Country:
United States
Status:
Recruiting
Investigator:
Last name:
Jack H Florin
Email:
Principal Investigator
Facility:
Name:
Neurology Offices Of South Florida
Address:
City:
Boca Raton
Zip:
33482
Country:
United States
Status:
Recruiting
Contact:
Phone:
561-482-1027
Investigator:
Last name:
Brian Costell
Email:
Principal Investigator
Facility:
Name:
Central TX Neuro Consultants P A
Address:
City:
Round Rock
Zip:
78681
Country:
United States
Status:
Recruiting
Contact:
Phone:
512-218-1222
Investigator:
Last name:
Adam Horvit
Email:
Principal Investigator
Facility:
Name:
Novartis Investigative Site
Address:
City:
Larissa
Zip:
411 10
Country:
Greece
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Patras
Zip:
265 00
Country:
Greece
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Lisboa
Zip:
1349-019
Country:
Portugal
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Lisboa
Zip:
1649 035
Country:
Portugal
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Barcelona
Zip:
08036
Country:
Spain
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Alicante
Zip:
03010
Country:
Spain
Status:
Recruiting
Facility:
Name:
Novartis Investigative Site
Address:
City:
Pozuelo de Alarcon
Zip:
28223
Country:
Spain
Status:
Recruiting
Start date:
July 31, 2024
Completion date:
January 12, 2029
Lead sponsor:
Agency:
Novartis Pharmaceuticals
Agency class:
Industry
Source:
Novartis
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT06517758
