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.

What is a clinical trial? Is participating in a clinical trial right for you? Learn more

Myasthenia Gravis Clinical Trial in Los Angeles CA
NCT03304054 | Phase 3 | Interventional

What you may receive

Study related treatment at no cost

Study related procedures

Travel expenses covered

Medical related treatment

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.

Completed

Male & Female

18 Years +

This study has recruited 93 Participants

The MuSK Trial is a clinical trial for an oral, investigational medicine for the treatment of MuSK-Positive Myasthenia Gravis (MuSK-MG). The trial will evaluate the effectiveness of amifampridine phosphate in controlling, reducing, and/or eliminating the symptoms of MuSK-MG. If you or someone you know has been diagnosed with MuSK-MG or has received a positive anti-AChR antibody test, you may be eligible to participate in the MuSK trial.

Why should I participate?

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.

We believe that this intervention could improve activities of daily living for patients with MuSK-MG.

This study is needed for FDA approval. By participating in this study you will help:
• Increase our understanding of amifampridine phosphate in the treatment of MuSK-MG
• Provide important clinical data needed for FDA approval
• Develop a new treatment option for MuSK-MG

What's involved?

Prior to enrolling in the study, you will need to understand what the trial involves by talking to the study staff and reading the Informed Consent Form (ICF). The ICF outlines all the known safety information of this oral, investigational medicine and what is involved in the study. Once enrolled, you will visit your study doctor for certain tests to make sure you are eligible to be included. Your doctor and study team will be available to answer questions that you or your family might have as you go through the process.

After an initial screening period (up to 14 days), the study will take approximately
38 days. Women participating in the study cannot be pregnant and must use
contraceptives throughout the study and then for 30 days after completion.
The study will be examining the most effective and tolerable dose of amifampridine
phosphate so, at some point during the study, you may receive a higher dose of the
study medication. Close monitoring and 24-hour medical oversight will ensure safety.

Who can participate?

The MuSK Trial is for adult patients in the United States with:

• a positive anti-MuSK antibody test, or
• a positive anti-AChR antibody test

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.