Amifampridine for Botulism
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores whether amifampridine, a medication, can improve nerve and muscle communication in individuals who have received Botox injections (specifically OnabotulinumtoxinA) in their facial muscles. The researchers aim to determine if this medication can enhance muscle function following Botox treatment. Participants should have received a Botox injection between 80 and 150 days prior and should not have a history of heart issues, seizures, or severe asthma. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this potentially beneficial treatment.
Will I have to stop taking my current medications?
The trial requires that you stop taking pyridostigmine, as it can affect neuromuscular transmission. If you are using any investigational drug or device, you must stop at least 30 days before joining the trial.
Is there any evidence suggesting that amifampridine is likely to be safe for humans?
Research has shown that amifampridine, also known as 3,4-DAP, is generally safe and well-tolerated. Studies have found that it can be used over time without causing harm, as it doesn't accumulate to dangerous levels in the body. This suggests the treatment is relatively gentle on the body, which is reassuring for those considering joining a trial.
Although safety concerns exist if amifampridine is taken incorrectly, such as being harmful if swallowed in large amounts or absorbed through the skin, clinical settings manage these risks. Proper handling and dosing make it a suitable option for treating conditions like botulism, as it has been shown to quickly improve symptoms and muscle function.
Overall, these findings provide strong evidence that amifampridine is a safe option for trial participants, as long as it is used correctly.12345Why do researchers think this study treatment might be promising?
Amifampridine is unique because it offers a new approach to treating botulism by enhancing nerve-muscle communication. Most treatments for botulism focus on neutralizing the toxins or providing supportive care, but Amifampridine targets the symptoms directly by improving muscle strength. This is especially exciting for researchers because it could lead to faster recovery times and improved outcomes for patients suffering from this rare but serious condition.
What evidence suggests that amifampridine might be an effective treatment for botulism?
Research suggests that amifampridine, which participants in this trial will receive, might improve muscle function in people affected by botulinum toxin. This drug increases the release of acetylcholine, a chemical crucial for muscle movement. Some studies have shown that by boosting acetylcholine, amifampridine could help with breathing problems and support muscle recovery. Although 3,4-diaminopyridine, a similar drug, did not improve muscle strength in one case of botulism, researchers believe amifampridine might still be helpful because it can counteract the effects of botulinum toxin. These findings suggest amifampridine could be a promising option for those with muscle issues related to botulism.16789
Who Is on the Research Team?
James B Caress, MD
Principal Investigator
Wake Forest University Health Sciences
Are You a Good Fit for This Trial?
This trial is for adults aged 18-80 who have had Botox injections in facial muscles and are now looking to improve muscle function. They must be able to consent, not have a history of heart rhythm problems, seizures, severe asthma, liver or kidney disease, facial nerve issues, or use of investigational drugs recently.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Baseline Assessment
Baseline single fiber electromyography (SFEMG) is conducted before administration of amifampridine
Treatment
Amifampridine is orally administered, followed by post-dose SFEMG and observation
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Amifampridine
Find a Clinic Near You
Who Is Running the Clinical Trial?
Wake Forest University Health Sciences
Lead Sponsor