BridgeMD
Compares treatment options for Generalized Myasthenia Gravis
Official title A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG.
ClinicalTrials.gov ID: NCT06517758
What this study is testing
What is Iptacopan?
Iptacopan is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for generalized myasthenia gravis.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 85
You may be able to join if
- 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...
- 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.
You likely can't join if
- Have been treated with intravenous immunoglobulin (IVIG)/plasma exchange (PLEX) in the past month, with rituximab in the past 6 months, eculizumab in...
- Participants with clinically significant active or chronic uncontrolled bacterial, viral, or fungal infection at screening, including patients who...
- Human Immunodeficiency Virus (HIV) positive serology associated with an Acquired Immune Deficiency Syndrome (AIDS)-defining condition or with a...
- 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 from menarche until becoming post-menopausal...
See the full eligibility 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 participant has not been previously vaccinated, or if a booster was required...
- 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...
- 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...
- 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 from menarche until becoming post-menopausal, unless they are using effective methods of contraception during dosing of...
- 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...
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Scottsdale, Arizona, United States
- Fullerton, California, United States
- Pasadena, California, United States
- Sacramento, California, United States
- Boca Raton, Florida, United States
- Hialeah, Florida, United States
- Augusta, Georgia, United States
- Honolulu, Hawaii, United States
- Chicago, Illinois, United States
- Springfield, Illinois, United States
- Bethesda, Maryland, United States
- Boston, Massachusetts, United States
- Detroit, Michigan, United States
- Durham, North Carolina, United States
- Canton, Ohio, United States
- Columbus, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Nashville, Tennessee, United States
- Houston, Texas, United States
- Round Rock, Texas, United States
+ 94 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 85 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Scottsdale, Arizona, United States; Fullerton, California, United States; Pasadena, California, United States; Sacramento, California, United States; Boca Raton, Florida, United States; Hialeah, Florida, United States and 108 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.