Nimble Study - Nimble Study

What is the NIMBLE Study?

Why are we doing this study?

If you are diagnosed with generalized myasthenia gravis (gMG), please know that you are not alone in your medical journey. gMG is a rare autoimmune disorder caused by autoantibodies produced by the immune system attacking the body’s own cells. These autoantibodies activate a pathway in the immune system called the complement pathway that leads to disruption of the communication between nerves and muscles, so that the muscle cannot function normally.
gMG affects between 50 and 200 people per million worldwide.

Currently, there is no cure for gMG but there is a growing need for more effective medications. NIMBLE is a clinical study that aims to determine whether a combination of two investigational study drugs given as a once monthly subcutaneous injection for adults with gMG may be effective and well-tolerated.

What study drugs will I receive?

If you meet all the requirements to participate in this study, there is about a 70% chance of receiving active study drugs and about a 30% chance of receiving a placebo in the first treatment period. After 24 weeks, all participants will receive active study drugs. Which study drug you receive depends on which group you are randomly assigned to and which part of the treatment period you are in. You may receive the following study drugs:

Investigational drug 1 (pozelimab):
A monoclonal antibody that blocks your complement component 5 (C5). In people with gMG, the action of C5 damages the communication between the nerves and muscles, which disturbs normal muscle contraction.

Combination therapy: Combines both investigational study drugs for complete suppression of the complement C5 system. This is being studied to see if this may reduce the damage caused by C5 at the neuromuscular junction (the site where the nerves and muscles connect), as well as reduce gMG symptoms and lead to lower and less frequent dosing.

Investigational drug 2 (cemdisiran): An RNAi therapeutic that suppresses the production of complement proteins (specifically C5) in the liver.

Placebo: Placebos allow researches to determine whether an investigational study drug is effective by understanding if the study drugs work better than a placebo. All participants receiving the placebo will receive active study drugs after 24 weeks.

What can I expect if I participate?

Taking part in a clinical study is a commitment. The study team will share information on what to expect, as well as your roles and responsibilities during the study.

The total duration of the NIMBLE study is approximately 3.5 years and will involve about 25 study visits ranging from weekly to quarterly.

Part of the treatment period will be “double-blind” – meaning neither you nor your study doctor or study team will know which study drug you are receiving. However, if necessary for a significant medical event or medical emergency, the study team can find out which group you are in.

Participants may be compensated for travel and accommodation costs.

During the NIMBLE study, in addition to receiving the study drugs, your health will be regularly checked during visits. Which assessments and procedures occur will vary between visits but will include for example, blood tests, urine collection, physical examinations, ECG scans, and health questionnaires. If you take part in this study, you will be given a detailed Visit Guide that explains exactly what will happen at each visit.

What if I no longer want to participate in this clinical trial?

Study participation is completely voluntary (your choice). You can change your mind and leave the study at any time. You do not need to give a reason. If you leave the study, your medical care outside of the study will not be affected by your decision.

How can I take part?

If you wish to take part in NIMBLE, you can fill in the pre-screening questionnaire to see if you may be eligible to participate:

What else should I know?

There is no guarantee that you will receive a medical benefit from taking part in this clinical study. The study drug may cause side effects or affect your health in an unknown way. However, your safety is our top priority and your health will be closely monitored throughout this study.

Participants in clinical studies are essential to developing treatments and increasing our medical understanding of myasthenia gravis. The information from this clinical study may benefit others in the future.

If you think you may be interested in taking part, or would like more information, please contact your local NIMBLE team – they will be more than happy to help. Find a clinical study site near you.

Find a clinical site near you

To find out more about NIMBLE, and whether it may be right for you, please contact the NIMBLE team at a clinical site near you.