Purpose of this Study
We are doing this study to find out if an investigational drug called iptacopan (the study drug) is safe and has beneficial effects for people who have generalized Myasthenia Gravis (gMG).
Who Can Participate?
Eligibility
Adults ages 18-75 who:
- Are diagnosed with gMG
- Have a positive lab test for AChR+ antibody
- Have been receiving a stable regimen of standard treatment for gMG for at least 6 months before joining the study
Age Range
18-75
Sex/Genders
Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No
What is Involved?
Description
If you choose to join this study, you will need to come to our clinic for screening to confirm your eligibility. Screening can last up to 3-to-4 hours (your study doctor may conduct the screening procedures over more than one visit) and any subsequent visits should take approximately 1-to-3 hours. If you are found to be eligible during screening, you will proceed to the "core" portion of the study.
During the study's core portion, you will:
- Get a random assignment (like a coin flip) to either take the study drug or a placebo (inactive substance with no drug in it)
- Visit our clinic up to 11 times over the course of about 9 months
Locations
Duke University Hospital
Visit Timing
Weekdays
Compensation
Yes
Spanish Materials Available
No
Study Details
Full Title
DNA07: 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
Principal Investigator
Shruti
Raja
Protocol Number
PRO00115548
NCT ID
NCT06517758
Phase
III
Enrollment Status
Open to Enrollment