The purpose of this study is to evaluate the safety and effectiveness of a study medication, called ALXN1720, in reducing symptoms in adults with gMG. This therapy differs from current treatment options, as it can be administered at home through injection with a prefilled syringe by you or your caregiver. Those who are eligible to participate and decide to enroll will be randomly (by chance) assigned to receive ALXN1720 or a placebo. The placebo used in this study looks the same as ALXN1720, but it does not contain active substance. Neither the participants nor the study doctor will know who has been assigned to receive ALXN1720 or placebo.
The total duration of the study can last up to approximately 32 months (a little over two and a half years), and it consists of the following periods:
- Screening: Lasts up to four weeks. The purpose of this period is to evaluate all interested individuals to see whether the study is a good match for them. There will typically be one visit to the study site.
- Treatment: Lasts up to 26 weeks. Participants will be randomly assigned to receive either ALXN1720 or a placebo. Both ALXN1720 and the placebo will be administered via injection under the skin on a weekly basis. There will be 15 total visits, nine of which are in person, and six that can be performed remotely.
- Open-Label Extension: Can last up to approximately 96 weeks (almost two years). All participants will receive the study medication during this period. There will be up to 23 total visits, 14 of which are in person, and nine that can be performed remotely. Final study assessments will be performed three weeks after the participant has received their final treatment dose.
About the Study Medication
The study medication is called ALXN1720 and has not been approved by country-specific regulatory health authorities, such as the Food and Drug Administration (FDA) in the US, to be used for gMG. Participants will be assigned at random to receive either ALXN1720 or the placebo. Neither the participant nor the study team will know which treatment option has been assigned, but in case of an emergency, they can quickly find out. Both ALXN1720 and the placebo are administered as injections.
You may be eligible for the study if you:
- Have a diagnosis of Myasthenia Gravis
- Experience generalized muscle weakness
- Have received a positive serological test for antibodies against AChR
- If you are unsure, testing can be done at screening
- Have not had a thymectomy nor have a currently untreated thymic malignancy, carcinoma, or thymoma
Individuals will be evaluated to determine their eligibility to participate. Those who are eligible and decide to take part will receive ALXN1720 (or placebo), study-specific medical exams, and study-specific laboratory tests at no cost.
View study on ClinicalTrials.gov