All studies
Active not recruitingPHASE3INTERVENTIONAL

Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis

This research study is investigating a new medication called ALXN1720 for adults living with generalised myasthenia gravis (gMG). We want to understand if this new treatment is both safe to use and effective at reducing the symptoms of gMG. Specifically, we're focusing on people whose gMG is caused by particular antibodies that attack nerve-muscle connections. Participants will either receive the new medication or a placebo (a dummy treatment that looks the same but has no active drug), so we can fairly compare their effects. This type of study, known as a Phase 3 trial, is a crucial step in determining if new medicines can become available to patients. Your involvement could help advance our understanding and treatment of gMG.

At a glance

Status
Active not recruiting
Phase
PHASE3
Sponsor
Alexion Pharmaceuticals, Inc.
Enrolment target
261
Start
21 Nov 2022
Estimated completion
31 Aug 2027

What is this study about?

This study is an important step in finding new ways to help people living with generalised myasthenia gravis, or gMG. GMB is a long-term condition that causes muscle weakness. This weakness can affect many parts of the body, making everyday tasks difficult. The specific type of gMG this study focuses on is caused by the body's immune system mistakenly attacking special connections between nerves and muscles (called acetylcholine receptors).

The main goal of this research is to test a new medicine called ALXN1720. We want to find out two key things: first, if ALXN1720 is safe for adults with gMG, meaning it doesn't cause too many serious side effects. Second, we want to know if it's effective at improving their symptoms and overall quality of life. Participants will be randomly assigned to receive either ALXN1720 or a placebo, which is an inactive substance that looks just like the real medicine. This allows us to accurately assess the true effects of ALXN1720.

Undertaking careful studies like this one is vital for medical progress. If ALXN1720 proves to be safe and effective, it could offer a new treatment option for many people living with generalised myasthenia gravis. Your participation could contribute significantly to helping others in the future.

Key takeaways

  • Investigating a new medicine (ALXN1720) for generalised myasthenia gravis (gMG).
  • Aims to check safety and if it improves gMG symptoms in specific antibody-positive adults.
  • Participants will receive either the new medicine or a dummy treatment (placebo).
  • Close medical monitoring throughout the study period.
  • Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

This study is looking for adults aged 18 or over who have been diagnosed with generalised myasthenia gravis (gMG). Your gMG should have been confirmed by a doctor, based on specific criteria that indicate muscle weakness in more than one area of your body.

Importantly, your gMG must be linked to certain antibodies in your blood called 'AChR antibodies'. A blood test will confirm if you have these antibodies, as this study specifically targets this type of gMG.

There are also some reasons why you might not be able to join. For example, if you've had surgery on your thymus gland in the last year, or if you have certain problems with your thymus gland. You also can't take part if you've had a type of infection called Neisseria meningitidis, or if you are pregnant, breastfeeding, or planning to become pregnant during the study.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

  1. Are you an adult (18 years or older)?
  2. Have you been diagnosed with generalised myasthenia gravis (gMG) by a doctor?
  3. Has a blood test shown you have 'AChR antibodies' for your gMG?
  4. Have you *not* had thymus surgery in the last 12 months?
  5. Are you *not* pregnant, breastfeeding, or planning to get pregnant during the study?
Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll be involved in the study for a certain period, which will be fully explained to you before you commit. You'll have regular visits to the clinic where the study team will monitor your health, perform examinations, and ask you about your symptoms. You'll receive either the study medication (ALXN1720) or a placebo, which you'll take as instructed. There will be blood tests and other assessments to track how you're responding to the treatment and to check for any side effects. After the main treatment period, there will be follow-up visits to ensure your well-being. All procedures, including the number of visits and their duration, will be thoroughly explained in the informed consent process.

Potential risks and benefits

Taking part in this study could potentially offer benefits, such as receiving a new investigational treatment that might improve your gMG symptoms, and you'll receive close monitoring from a medical team. However, as with any medication, there are potential risks, including side effects from the study drug, which will be fully detailed. There's also no guarantee that the treatment will help you, or that you'll receive the active drug. Your participation is completely voluntary, and you are free to withdraw from the study at any time, for any reason, without affecting your usual medical care.

Locations (132)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • Research Site
    Verified postcode
    Orange, United States
  • Research Site
    Verified postcode
    Fort Collins, United States
  • Research Site
    Verified postcode
    Washington D.C., United States
  • Research Site
    Verified postcode
    Bradenton, United States
  • Research Site
    Verified postcode
    Maitland, United States
  • Research Site
    Verified postcode
    Tampa, United States
  • Research Site
    Verified postcode
    Augusta, United States
  • Research Site
    Verified postcode
    Gainesville, United States
  • Research Site
    Verified postcode
    Savannah, United States
  • Research Site
    Verified postcode
    Springfield, United States
  • Research Site
    Verified postcode
    Kansas City, United States
  • Research Site
    Verified postcode
    Lexington, United States

Common questions

What is generalised myasthenia gravis (gMG)?

Generalised myasthenia gravis is a long-term condition that causes muscle weakness throughout the body because of problems with the signals between nerves and muscles.

What is a placebo?

A placebo is a 'dummy' treatment that looks identical to the actual study medication but contains no active drug. It helps us fairly compare the effects of the new medicine.

Why is this a 'Phase 3' study?

Phase 3 studies are a crucial stage where a new medicine is tested on a larger group of people to confirm its safety and effectiveness, before it can be approved for wider use.

Will I know if I'm getting the actual medicine or the placebo?

No, during the main part of the study, neither you nor your study doctor will know whether you are receiving the active medication or the placebo. This is to ensure objective results.

Can I leave the study once I've joined?

Yes, your participation is completely voluntary. You can choose to leave the study at any time, for any reason, and this will not affect your ongoing medical care.

How to find out more

Always speak to your GP or specialist before deciding to take part in a study.

Discussion

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