A New Clinical Trial for
Myasthenia
Gravis Patients
Contact us to learn more about the study:
- Free efgartigimod for the entire 26-week study.
- Neurologist & nurse care at no cost.
- Easy home questionnaires during pauses.
- Guaranteed access to efgartigimod after completing the study through a compassionate use program.
- Participants do not incur any costs.
- Meticulous nursing and medical care by the study team.
If you or someone you know has MG, please contact Victorine.
Victorine Sikati Foko
Summary
This page invites individuals diagnosed with myasthenia gravis (MG) to participate in a study at Clinique Neuro-Outaouais. The study will investigate the drug efgartigimod as a replacement for IVIG treatment in patients with generalized MG who are anti-acetylcholine receptor (AChR) antibody positive and stable on IVIG for at least 6 months.
Efgartigimod works by blocking the neonatal Fc receptor (FcRn), which reduces the levels of IgG autoantibodies that mistakenly attack the body’s own acetylcholine receptors, leading to muscle weakness in MG. Unlike IVIG, efgartigimod is not a blood product and is administered via a one-hour IV infusion weekly for four weeks, followed by a four-week pause.
The purpose of this study is to determine if efgartigimod is as effective or better than IVIG in treating MG patients. Approximately thirty patients will participate in this 26-week study conducted at Clinique Neuro-Outaouais in Gatineau, Québec.
Dr. François Jacques, the lead investigator, will address any questions or concerns regarding participation.
FAQ
Click on the green sections below to learn more about the study.
Click on the blue sections to read questions and answers
What is this study about?
This study is investigating a drug called efgartigimod to see if it works as well as your current IVIG treatment for generalized myasthenia gravis (gMG). We also want to know if patients prefer efgartigimod or IVIG. We are also looking at the safety and how well you tolerate efgartigimod over 6 months.
Why have I been invited to participate?
You have been invited because you have been diagnosed with myasthenia gravis (MG).
What is efgartigimod?
Efgartigimod is a medication that blocks a protein in your body called the neonatal Fc receptor (FcRn). By doing this, it lowers the levels of IgG autoantibodies, which are immune system proteins that mistakenly attack your own body in MG. Unlike IVIG, efgartigimod is not a blood product.
How does efgartigimod work for myasthenia gravis?
In myasthenia gravis, IgG autoantibodies attack and damage acetylcholine receptors on your nerves, which prevents your muscles from contracting properly. Efgartigimod helps improve muscle contraction and reduce MG symptoms by lowering the amount of these harmful autoantibodies.
What is IVIG?
IVIG stands for intravenous immunoglobulin, which is a common treatment for myasthenia gravis. This study will compare efgartigimod to your current IVIG treatment.
How is efgartigimod different from IVIG?
Efgartigimod works by blocking a specific protein to lower autoantibody levels, while IVIG is a blood product that contains antibodies from many donors. Efgartigimod is given as a 1-hour intravenous infusion once a week for 4 weeks, followed by a 4-week break, whereas IVIG treatment schedules can vary.
Has efgartigimod been used before?
Yes, efgartigimod is already approved for treating generalized myasthenia gravis in adults who are anti-acetylcholine receptor (AChR) antibody positive. Previous studies have shown it can reduce IgG and anti-AChR autoantibody levels and has an established safety profile.
What is the purpose of this study?
The main purposes are to see if efgartigimod is as effective as IVIG for your gMG, if patients prefer one treatment over the other, and to further understand the safety and tolerability of efgartigimod when given in a regular dosing schedule over 6 months.
Where will the study take place?
The study will be conducted at the Clinique Neuro-Outaouais. 209 Rue Gamelin #200, Gatineau, Quebec J8Y 1W2.
How long will I be in the study?
Your participation in the study will last approximately 26 weeks (about 6 months).
What will I need to do if I participate?
If you decide to participate, you will have two screening visits, 16 treatment visits for the efgartigimod infusions, and one end-of-study visit. You will also need to complete questionnaires at the clinic and some from home during the treatment pauses. Your IVIG treatment will be stopped one week before the study drug starts.
What happens during the screening visits?
During the first screening visit, the study doctor will explain the study in detail, answer your questions, and you will sign the consent form if you wish to participate. You will also have a medical examination and your medical history and current medications will be reviewed. You will return about two weeks later to complete questionnaires, and then the doctor will determine if you are eligible for the study.
What happens during the treatment visits?
At each treatment visit, your vital signs will be checked, you will have a urine pregnancy test if applicable, and you will receive the efgartigimod infusion over one hour, followed by an hour of observation. The study coordinator will also ask about your medications and any new symptoms. A doctor will be present for each infusion.
What happens during the treatment pauses?
During the 4-week breaks between the efgartigimod treatment cycles, you will complete clinical assessment questionnaires from home and inform the study team of any changes in your medications or condition.
What happens at the end-of-study visit?
At the final visit, the study doctor will perform a physical and neurological examination, and you will complete clinical assessment questionnaires.
Can I withdraw from the study?
Yes, your participation is voluntary, and you can withdraw from the study at any time for any reason without penalty. If you withdraw, the study coordinator may contact you a couple of weeks later to check on how you are doing. You will also need to return for a physical and neurological examination.
What if the study doctor asks me to leave the study?
The study doctor may withdraw you if they believe it is in your best interest, if you have a medical emergency, or if you do not follow the study visit schedule.
What treatment will I receive?
If you are eligible for the study, you will receive efgartigimod as an intravenous (IV) infusion.
How often will I receive the treatment?
You will receive 4 treatments over a 3 week period followed by a 4 week pause. This cycle will be repeated four times over the 26-week study period.
How long does each infusion take?
Each efgartigimod infusion will take about one hour. You will also be observed for an additional hour after the infusion.
Will I have to pay for the study drug?
No, the efgartigimod will be provided to you free of charge for the duration of the study.
What happens once I have completed the study?
Click on the blue sections to read questions and answers
What are the possible side effects of efgartigimod?
In previous studies, common side effects included inflammation of airway passages, upper respiratory tract infection, urinary tract infection, headache, and muscle pain. Most side effects were mild or moderate and did not last long. There is a potential for immune reactions (fever, itching, rash) or allergic reactions (including severe symptoms like shortness of breath), although these have not been common in studies so far. Efgartigimod could also increase your risk of infections.
What are serious side effects?
A serious side effect is one that is life-threatening, requires hospitalization, results in significant disability, or has other serious medical implications. Serious side effects occurred in a similar percentage of patients who received efgartigimod and those who received a placebo in previous studies.
What are the risks of having blood drawn?
Drawing blood may cause pain, bruising, or infection at the injection site. Some people may feel dizzy, faint, or have an upset stomach.
What are infusion-related reactions?
Efgartigimod may cause infusion-related reactions, which are usually mild or moderate and can include fever, itching, or rash. These often happen during or shortly after the infusion.
What are the risks for women who can become pregnant?
You cannot become pregnant or breastfeed during the study. You will need to have a negative pregnancy test before starting treatment and use reliable contraception throughout the study and until your final dose. You will have regular urine pregnancy tests during the study.
What about contraception for men?
There are no specific contraceptive requirements for male participants.
What about breastfeeding?
You should not breastfeed while receiving efgartigimod.
What about vaccinations during the study?
Your vaccination status will be reviewed. You may be advised to get some vaccines (except live vaccines) during the pauses between treatment cycles, making sure there is at least a 48-hour window before or after an infusion. Live or live-attenuated vaccines are generally not recommended during efgartigimod treatment.
Are there any unknown risks?
As with any new drug, there may be rare or currently unknown side effects, and efgartigimod could potentially interact with other medications. You should always check with the study doctor before starting any new medications.
Click on the blue sections to read questions and answers
What are the possible benefits of participating in this study?
By participating, you will help gather important information about efgartigimod for treating myasthenia gravis, which could benefit other patients in the future. The study team hopes that the treatment will also benefit you personally, but there is no guarantee that your condition will improve.
What other treatment options are available?
There are other treatment options for myasthenia gravis. You should discuss these with your regular doctor or Dr. François Jacques. If efgartigimod is found to be less effective than IVIG for you, you will be advised to restart IVIG therapy.
Will I be told the results of the study?
A report of the study results will be available upon request, and your identity will be protected in any published information. A summary of the results will also be posted on the ClinicalTrials.gov website.
Click on the blue sections to read questions and answers
Will my information be kept confidential?
Yes, all information collected about you will be kept strictly confidential and stored securely at the Clinique Neuro-Outaouais. Your name will not be used in any study documents or publications.
Who will have access to my study information?
Authorized representatives from Health Canada, the Ethics Committee (Canadian Shield ERB), and other regulatory bodies may review your medical file for study monitoring purposes, but your identity will remain confidential.
Will I have to pay for participating in the study?
No, you will not have any expenses for participating in this study. All study-related costs, including the study drug, will be covered by the Clinique Neuro-Outaouais.
Will I be compensated for my time and travel?
You will not receive direct financial compensation for participating. However, you will be reimbursed for reasonable travel and parking expenses at a planned rate of $80 per visit.
Click on the blue sections to read questions and answers
Is my participation voluntary?
Yes, your participation is entirely voluntary. You can choose not to participate or withdraw at any time without any negative consequences to your future medical care.
Who can I contact if I have questions?
If you have any questions about the study, please contact Dr. François Jacques or the Study Coordinator at (819) 777-2500.
Who can I contact if I have concerns about my rights as a study participant?
If you have any questions about your rights as a research participant or concerns about the study, you can contact the Canadian Shield ERB at 905 681-8661 or by email at admin@cserb.com.