RECRUITING

A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 75 Years With gMG.

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Conditions

Generalized Myasthenia GravisMyasthenia GravisgMGrandomizedplacebo-controlleddouble-blindphase IIIAChR+SOC treatmentiptacopanLNP023

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2024-07-31
Study Completion:2029-01-12
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06517758

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 75 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Adult patients with generalized Myasthenia Gravis (age 18-75 years)
  2. * Positive serology testing for AChR+ antibody at screening
  3. * 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.
  4. * The confirmation of the diagnosis of gMG should be documented and supported by ≥1 of the following 3 tests:
  5. * History of abnormal neuromuscular transmission demonstrated by single-fiber electromyography or repetitive nerve stimulation.
  6. * History of positive edrophonium chloride test
  7. * Patient has demonstrated improvement in MG signs on oral acetylcholinesterase inhibitors as assessed by the treating physician.
  8. * Baseline MG-ADL score ≥6, with ≥50% of the total score due to non-ocular symptoms
  9. * Participants not optimally controlled for ≥ 6 months on
  10. * just one NSIST; or
  11. * two or more NSISTs; or
  12. * on frequent (at least quarterly) plasmapheresis, plasma exchange, or intravenous immunoglobulin to control symptoms despite treatment with steroids and NSISTs; or
  13. * one of the following gMG treatments:
  14. * a FcRN antagonist approved for gMG
  15. * rituximab
  16. * other approved gMG therapies excluding complement inhibitors.
  17. * 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.
  1. * 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.
  2. * 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
  3. * 200 cells/mm3
  4. * Female participants who are pregnant or lactating, or are intending to become pregnant.
  5. * 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.
  6. * 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.
  7. * History of recurrent invasive infections caused by encapsulated organisms, e.g., N. meningitidis and S. pneumoniae.
  8. * Presence of fever ≥ 38 °C (100.4 °F) within 7 days prior to study drug administration

Contacts and Locations

Study Contact

Novartis Pharmaceuticals
CONTACT
1-888-669-6682
novartis.email@novartis.com
Novartis Pharmaceuticals
CONTACT
+41613241111

Study Locations (Sites)

Fullerton Neuro and Headache Ctr
Fullerton, California, 92835
United States
SC3 Research Pasadena
Pasadena, California, 91105
United States
Neurology Offices Of South Florida
Boca Raton, Florida, 33482
United States
Superior Associates in Research LLC
Hialeah, Florida, 33012
United States
Hawaii Pacific Neuroscience LLC
Honolulu, Hawaii, 96817
United States
Prairie Heart Institute
Springfield, Illinois, 62769
United States
Neuroscience Research Ctr
Canton, Ohio, 44718
United States
Ohio State University Medical Center
Columbus, Ohio, 43210
United States
Central TX Neuro Consultants P A
Round Rock, Texas, 78681
United States

Collaborators and Investigators

Sponsor: Novartis Pharmaceuticals

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-07-31
Study Completion Date2029-01-12

Study Record Updates

Study Start Date2024-07-31
Study Completion Date2029-01-12

Terms related to this study

Keywords Provided by Researchers

  • Myasthenia Gravis
  • gMG
  • randomized
  • placebo-controlled
  • double-blind
  • phase III
  • AChR+
  • SOC treatment
  • iptacopan
  • LNP023

Additional Relevant MeSH Terms

  • Generalized Myasthenia Gravis