Advancing 
Care Through
Clinical Trials

What Is a Clinical Trial?

A clinical trial is a research study to determine whether an investigational new medicine is deemed safe and effective before being approved and made available to the general public. Researchers study how well a treatment works in the people it’s meant to help, including the benefit(s) of the investigational treatment, its side effects, and if it can help improve daily life. Clinical trials follow strict rules to protect participants, and taking part in a study is always voluntary.

NMD Pharma is conducting several clinical trials to study an investigational treatment across several neuromuscular diseases including generalized myasthenia gravis (gMG), spinal muscular atrophy (SMA), and Charcot-Marie-Tooth disease (CMT). These studies are being conducted to assess the potential of our investigational treatment to provide improvements in muscle strength and function, allowing for improvements in quality of life and greater independence for people living with these neuromuscular conditions.

3 Steps to Joining an

NMD Pharma Clinical Trial

  1. Find a convenient location

    Look for a study site that works best for you.

  2. Do you qualify for the study?

    Answer a few simple questions to find out if you're eligible.

  3. We'll be in touch

    If you're eligible, someone from our team will reach out to answer your questions and help you schedule an appointment.

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Ready to get started?

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What to Expect as a Participant

Participants will meet with the study doctor for an initial assessment and if accepted attend multiple scheduled visits and may be prescribed the study medication or placebo (a sugar pill that looks and tastes like the real drug being tested but does not have any investigational medicine in it). Your health is of utmost importance and closely monitored throughout the study. Study-related care is provided at no cost, with travel reimbursements and possible compensation for your time.

The Different Phases of a Clinical Trial

Each phase of a clinical trial is designed to give researchers the information they need to assess safety, effectiveness, and possible side effects of the investigational treatment. This step-by-step process helps make sure an investigational treatment is fully understood and the proper information is available for the FDA to review during the approval process.

Phase 1

This first phase focuses on safety and exploring different dose levels for the treatment to be used in patients. Researchers work with a small group of people to learn how the treatment works in the body and watch carefully for any side effects.

Phase 2

Next, researchers see how well the treatment works in a well-defined group of patients with the disease and continue to watch carefully for any side effects. These studies usually include having a group of patients taking a placebo pill, to determine whether the investigational treatment is providing benefit for those people with the disease.

Phase 3

Next, the treatment is tested in more people with the disease. Some may get the new treatment, some may get current treatments, and some may get a placebo. These studies often take place for a longer period of time.

This phase is designed to gather strong evidence about effectiveness, safety, and any side effects before the FDA reviews all results and considers approval for prescribing and patient use.

Often, there will be an opportunity for all patients in the Phase 3 clinical trial to remain on the drug in an “open-label extension” (often called an “OLE”) after the formal end of the placebo-controlled portion of the phase 3 study. This allows all patients in the study to continue to receive the study drug if they believe it would provide them benefit. Patients can choose to enter into the OLE, often until the FDA makes a determination on the approval of the drug (or the company decides not to develop it further).

Phase 4 (ongoing)

Finally, after FDA-approval of the treatment, researchers may continue to monitor safety and effectiveness as more people use the treatment over time.

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