Investigating the effectiveness of prednisone in the treatment of myasthenia gravis affecting only the eyes

Start Date
01/11/2010
Expected end Date
01/08/2013
Location
USA
Status
Not Yet Recruiting

What is the aim of the trial?

Myasthenia gravis is a neuromuscular disorder that presents in two main forms: ocular myasthenia gravis affects the eye muscles only and generalized myasthenia gravis which affects other muscle groups as well as the eye. This trial is testing a treatment for ocular myasthenia gravis which causes weakness and tiredness in the eye muscles controlling the eyes and eyelids. This can cause drooping of the eyelids (ptosis) which can affect one or both eyes and double vision (diplopia) which sometimes occurs only when looking in a particular direction.

Myasthenia gravis develops as the result of a defect in the immune system. The immune system’s job is to produce antibodies against bacteria and viruses. Unfortunately, it sometimes produces antibodies against “self” proteins causing “auto”immune disease.

The majority of individuals with myasthenia gravis produce antibodies against a self-protein called the acetylcholine receptor (AChR). This is found at the junction between the nerve and the muscle (the neuromuscular junction). It acts as a “receiver” for the chemical signal, acetylcholine that is released from the nerve when we want to use a muscle. The antibodies bind to the acetylcholine receptors on the muscle membrane and greatly reduce their ability to receive the chemical signal. As a result individuals experience muscle weakness which becomes worse as they repeatedly try to use the same muscle.

This phase 3 trial will test the effectiveness of the drug prednisone in the treatment of ocular myasthenia gravis. Prednisone is a corticosteroid, which suppresses the immune system, thereby reducing the severity of the attacks by the body’s own immune system. The study aims to investigate whether prednisone is safe, well tolerated as well as effective in improving the symptoms of double vision and drooping eyes that are experienced with ocular myasthenia gravis.

Who can be involved in the trial?

This trial aims to recruit 88 people with ocular myasthenia gravis whose weakness is confined to the muscles around the eyes and those that open and close the eyes. Diagnosis is based on a series of measurements including the detection of acetylcholine receptor antibodies in the blood and tests that measure the electrical activity of the muscle (electrophysiology). Individuals must be 18 years old or older and capable of providing informed consent. Individuals will not be recruited if they are pregnant or breastfeeding, have had symptoms of ocular myasthenia gravis for over two years, or have an illness that would, in the opinion of the neurologist, make it unsafe for the individual to participate in the trial. Recruited individuals will have had no prior treatment (or inadequate treatment) of the drug pyridostigmine (anti-cholinesterase drug).

What happens during the trial?

Participants will be treated for one month with pyridostigmine. If symptoms are not resolved within this first month of treatment, then the participants will be randomly assigned to receive capsules containing 10mg of the steroid prednisone or a matching placebo capsule. A placebo is an inactive substance designed to resemble the drug being tested. It is to rule out any benefits a drug might exhibit because the recipients believe they are taking it. Neither the patients nor the clinicians will know who is receiving the placebo.

Any changes in the individuals’ severity of symptoms and quality of life will be recorded. . A detailed record of anything which could be a treatment side effect will also be kept. The amount of treatment a participant receives will depend on how their symptoms respond to the medication and if they experience any side effects. Participants will be followed-up for 4 months.

Where is the study taking place?

The trial is taking place at Atlanta University, Georgia, United States.

In most circumstances, for somebody to participate in a clinical trial, they need to live near the team of people who are conducting the research, because they need to be closely monitored.

How could the results of the trial benefit patients?

This Phase 3 trial is testing the safety and efficacy of treating ocular myasthenia patients with prednisone. Although prednisone is already used in the clinic, this trial will formally assess the benefits of prednisone in myasthenia gravis affecting only the eyes. The results of the trial will inform clinicians of the risks and benefits of this treatment and help them decide on the best course of treatment for these patients.

Contact Details:

Michael Benatar

Tel: +1 404 727 3507

Email: Michael.benatar@emory.edu

Official name of the Trial:

Efficacy of Predisone in the treatment of ocular myasthenia: The EPITOME’ study.

Trial study number:

NCT00995722

Further trial details:

For further information on the trial and detailed inclusion and exclusion please click on the link below. Sometimes these details can be quite technical. If you have any questions please discuss this with your clinician or contact the clinical trial organisers.

www.clinicaltrials.gov/show/NCT00995722

Background information and related links:

Myasthenia gravis is a neuromuscular disorder that presents in two main forms: ocular myasthenia gravis affects the eye muscles only and generalized myasthenia gravis which affects other muscle groups as well as the eye. This trial is testing a treatment for ocular myasthenia gravis which causes weakness and tiredness in the eye muscles controlling the eyes and eyelids. This can cause drooping of the eyelids (ptosis) which can affect one or both eyes and double vision (diplopia) which sometimes occurs only when looking in a particular direction.

Myasthenia gravis develops as the result of a defect in the immune system. The immune system’s job is to produce antibodies against bacteria and viruses. Unfortunately, it sometimes produces antibodies against “self” proteins causing “auto”immune disease.

The majority of individuals with myasthenia gravis produce antibodies against a self-protein called the acetylcholine receptor (AChR). This is found at the junction between the nerve and the muscle (the neuromuscular junction). It acts as a “receiver” for the chemical signal, acetylcholine that is released from the nerve when we want to use a muscle. The antibodies bind to the acetylcholine receptors on the muscle membrane and greatly reduce their ability to receive the chemical signal. As a result individuals experience muscle weakness which becomes worse as they repeatedly try to use the same muscle.

This phase 3 trial will test the effectiveness of the drug prednisone in the treatment of ocular myasthenia gravis. Prednisone is a corticosteroid, which suppresses the immune system, thereby reducing the severity of the attacks by the body’s own immune system. The study aims to investigate whether prednisone is safe, well tolerated as well as effective in improving the symptoms of double vision and drooping eyes that are experienced with ocular myasthenia gravis.

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