Myasthenia Gravis Clinical Trial in United States
Have you or a loved one been diagnosed with Myasthenia Gravis?
You may be eligible to participate in a Myasthenia Gravis clinical trial.
Have you or a loved one been diagnosed with Myasthenia Gravis? You may be eligible to participate in a Myasthenia Gravis clinical trial.
Frequently Asked Questions
What is Myasthenia Gravis (MG) and what are the symptoms?
Myasthenia gravis (MG) is a chronic condition that causes extreme muscle weakness and can impact a person’s ability to see, smile, walk, talk, chew, swallow and breathe.
Symptoms may include:
• difficulties with chewing and swallowing
• difficulties with breathing
• weakness in neck, face, arms and legs
• slurred speech
• drooping eyelids
• blurred/double vision
• difficulties with personal grooming and hygiene tasks.
What is known about the study drug?
The study drug is experimental for Myasthenia Gravis, but it is FDA approved for a different autoimmune, neuromuscular disease [called Lambert-Eaton Myasthenic Syndrome (LEMS)].
What happens in the clinical evaluation?
The evaluation includes:
• Strength tests
• Questions about how you are feeling
• Urine samples
• MuSK and/or AChR antibody testing(if not previously done and/or the report is not available)
• Vital signs and an electrocardiogram (ECG or EKG)
• Medical history
• An electromyography (EMG) if not previously done and/or the report is not available.
How do I get started?
If you are on your phone, click "apply to participate" at the bottom to provide your contact information and answer a few questions. If you are on your computer or tablet, fill out the "interested in participating?" section and click "apply to participate."
What is the time commitment?
The MuSK Trial will be for at least 38 days with 5-6 clinic visits including screening; the initial screening may take up to an additional 14 days.
Will I receive the active medicine or placebo on the MuSK trial?
All eligible patients enrolled in the study will receive the study drug during the first phase of the study. Only patients that improve in their activities of daily living after taking the study drug will be considered for the second phase. In the second phase, half of the patients will receive the placebo and half will remain on the study drug. At the conclusion of that phase, all completing patients will be eligible to participate in the extension if they wish to do so.
Will I have access to the medication after the study?
After the study, eligible participants can choose to enter a long-term extension study and will continue to receive the study medicine until the medicine is approved and available commercially.
If I receive the active medicine, what side effects should I expect?
The Informed Consent Form will review in detail all of the safety information known about the oral, investigational medicine. After you read it, please direct any questions to your doctor.
Do I need to sign an Informed Consent Document in order to participate?
Yes. This document is necessary for participation in all clinical trials. You’ll receive
it from the study physician and have time to review it and ask questions.
Your physician will also help answer any questions you may have.
Do I have to pay for anything? .
No. All treatment and study-related costs will be covered (treatment, study-related procedures, meals, travel and lodging accommodations). The study will include access to expert medical care for the duration of the study and access to continued treatment for eligible patients at the conclusion of the trial through the open-label extension study.
Can I bring my spouse, a friend, or a support person?
Yes. You may bring a support person to help assist you during the study. Their travel,
accommodations, and associated expenses may be covered by the sponsor.
Details for the study
Treatments and/or Procedures
Inclusion Criteria: 1. Willing and able to provide written informed consent after the nature of the study has been explained and before the start of any research-related procedures. 2. Male or female ≥18 years of age. 3. Positive serologic test for anti-MuSK antibodies or anti-AChR antibodies as confirmed at Screening or by previous antibody test, with report available. 4. Confirmatory EMG or EMG report. 5. Myasthenia Gravis Foundation of America (MGFA) Class II to IV at Screening. 6. MG-ADL score of ≥6 at Screening, with more than 50% of this score attributed to non-ocular items. 7. Patients receiving steroids or pyridostigmine should not have any modification of drug regimen during the month before Screening. 8. Female patients of childbearing potential must have a negative pregnancy test (serum human chorionic gonadotropin [HCG] at screening); and must practice an effective, reliable contraceptive regimen during the study and for up to 30 days following discontinuation of treatment. 9. Ability to participate in the study based on overall health of the patient and disease prognosis, as applicable, in the opinion of the Investigator; and able to comply with all requirements of the protocol, including completion of study questionnaires. Exclusion Criteria: 1. Epilepsy and currently on medication. 2. Concomitant use of medicinal products with a known potential to cause QTc prolongation. 3. Patients with long QT syndromes. 4. History of thymectomy within 12 months before Screening. 5. An electrocardiogram (ECG) within 6 months before starting treatment that shows clinically significant abnormalities, in the opinion of the Investigator. 6. Breastfeeding or pregnant at Screening or planning to become pregnant at any time during the study. 7. Patients receiving immunomodulatory treatment (e.g. plasma exchange [PE], therapeutic plasma exchange [TPE], intravenous immunoglobulin G [IVIG]) should not have any treatment in the previous 4 weeks prior to Randomization or at any time during the study. 8. Use of rituximab or other similar biologic medications for immunomodulation within 6 months prior to Screening. 9. Treatment with an investigational drug (other than amifampridine) or device within 30 days before Screening or while participating in this study. 10. Any medical condition that, in the opinion of the Investigator, might interfere with the patient's participation in the study, poses an added risk for the patient, or confound the assessment of the patient. 11. History of drug allergy to any pyridine-containing substances or any amifampridine excipient(s).