Active not recruitingPHASE3INTERVENTIONAL

A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis

This research is investigating new treatments, pozelimab and cemdisiran, for adults living with generalised myasthenia gravis (gMG). gMG is a condition where the connection between nerves and muscles is affected, leading to muscle weakness and tiredness. The study aims to find out if these drugs, used alone or together, are safe and effective in improving gMG symptoms. Researchers will also look at any side effects, how the drugs work in the body, and if the body develops a reaction to them. This is a Phase 3 study, meaning it's a later stage of research that involves more people to confirm earlier findings and gather more information on safety and effectiveness.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Regeneron Pharmaceuticals

Enrolment target

288

Start

14 Dec 2021

Estimated completion

11 Nov 2028

Generalized Myasthenia Gravis

What is this study about?

This study is looking into new potential treatments for adults who have a condition called generalised myasthenia gravis, or gMG for short. gMG is a long-term illness where your body's immune system mistakenly attacks the connections between your nerves and muscles. This can lead to your muscles becoming weak and easily tired, affecting things like your eyesight, speech, swallowing, and movement.

The researchers want to see if two investigational drugs, called pozelimab and cemdisiran, can help people with gMG. They will be testing these drugs in a few ways: some participants will receive both drugs together, some will receive only cemdisiran, some only pozelimab, and others will receive a placebo (a dummy drug with no active ingredients). This allows the team to compare the effects and understand which treatments are most helpful.

Beyond simply seeing if the drugs work, the study also aims to understand their full impact. This includes carefully monitoring for any side effects, learning how the drugs behave inside the body, checking how much of the drug is present in the blood over time, and seeing if the body produces antibodies against the drugs. Understanding these details is crucial for developing safe and effective new medicines.

Key takeaways

This study is testing new drugs for generalised myasthenia gravis (gMG).
It's a Phase 3 trial, which means it's a later stage of research.
Researchers want to check drug safety, effectiveness, and how they work in the body.
Participation involves regular hospital visits over an extended period.
You might receive active drug or a placebo, but you can withdraw anytime.

Who may be eligible?

To be part of this study, you would generally need to be an adult aged 18 or over with a confirmed diagnosis of generalised myasthenia gravis (gMG). Importantly, your medical records must show that you have specific antibodies related to gMG, either anti-acetylcholine receptor (AChR) or anti-LRP4 antibodies. Also, your gMG symptoms should be at a certain level according to a standard medical scale, and you should be taking, or have a valid reason for not taking, a common gMG medication called an acetylcholinesterase inhibitor and/or other immune-suppressing treatments.

There are also some reasons why you might not be able to join. For example, if your gMG is solely due to another type of antibody called MuSK, or if you've had a specific type of surgery called a thymectomy recently (within the last year). People with a history of certain cancers in the last five years (excluding some less serious skin cancers) would also not be able to participate. These rules are in place to help make sure the study is safe and that the results are clear.

Quick self-check

Are you 18 years old or older?
Do you have a confirmed diagnosis of generalised myasthenia gravis (gMG) with specific antibodies (AChR or LRP4)?
Are your gMG symptoms at a certain level of severity?
Are you currently taking certain gMG medications, or have a reason not to?
Have you had a thymectomy (a type of surgery) more than 12 months ago, or not at all?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, your involvement would be split into different periods. There's an initial 'Double-Blind Treatment Period' lasting 24 weeks, followed by an 'Extension Treatment Period' of 28 weeks, and then an 'Open Label Treatment Period' which lasts about 68 weeks. After these treatment phases, there's a 'Follow-Up Period' of 52 weeks where researchers will check on your health even after you've stopped taking the study medication. During these periods, you'll have regular hospital visits for check-ups, assessments, and to receive your study medication. You'll need to be willing to follow all the study's instructions, including attending these appointments and having certain vaccinations.

Potential risks and benefits

Taking part in a clinical trial might offer benefits like access to new treatments before they are widely available, and you would receive close medical monitoring. However, there are potential risks, including side effects from the study drugs, which will be carefully watched for. You might also receive a placebo, meaning you wouldn’t get the active treatment. You would be fully informed about these before deciding to join, and importantly, you have the right to withdraw from the study at any time for any reason, without it affecting your usual medical care.

Locations (116)

HonorHealth Neurology 2018
Scottsdale, United States
University of California Irvine
Irvine, United States
University of Southern California
Los Angeles, United States
Colorado Springs Neurological Associates
Colorado Springs, United States
SFM Clinical Research, LLC
Boca Raton, United States
Diverse Clinical Research
Miami, United States
Aqualane Clinical Research
Naples, United States
Neurological Services of Orlando
Orlando, United States
Medsol Clinical Research Center Inc
Port Charlotte, United States
University of South Florida Morsani Center for Advanced Healthcare
Tampa, United States
NorthShore University Health System
Glenview, United States
St. Elizabeth's Hospital
O'Fallon, United States

+104 more sites — see the official record for the full list.

Common questions

What is generalised myasthenia gravis (gMG)?

It's a long-term condition causing muscle weakness and tiredness because of problems with how nerves and muscles communicate.

What are pozelimab and cemdisiran?

These are investigational drugs being tested to see if they can help improve symptoms of gMG.

Will I know if I'm getting the actual drug or a dummy pill (placebo)?

During the main part of the study, neither you nor your doctor will know if you're getting the active drug or a placebo. This is to ensure fair results.

How long will the study last?

The study involves different treatment periods lasting over a year, plus a follow-up period, so your total participation could be quite long.

Can I leave the study if I change my mind?

Yes, you are free to leave the study at any time, for any reason, and it will not affect your future medical care.