What side effects are associated with this type of intervention?

Common treatments for botulism include antitoxins and supportive care. Antitoxins can cause allergic reactions, including anaphylaxis, serum sickness, and rash. Supportive care, such as mechanical ventilation, may lead to complications like pneumonia or lung injury. Amifampridine, a potassium channel blocker that enhances acetylcholine release, is being studied for its potential benefits. Known side effects of amifampridine include seizures, dizziness, and gastrointestinal disturbances.

What prior FDA approvals exist?

The primary FDA-approved treatment for botulism is the administration of botulinum antitoxin, which neutralizes the toxin's effects. Supportive care, including mechanical ventilation, is often necessary due to the paralysis caused by the toxin. While amifampridine, a potassium channel blocker that enhances acetylcholine release, is being studied for its effects on neuromuscular transmission, it is not currently an FDA-approved treatment for botulism. The focus remains on antitoxins and supportive measures.

What other types of research exist?

One promising alternative to Amifampridine for Botulism patients is 4-aminopyridine, which has been shown to effectively reverse neuromuscular blockade induced by polymyxin B in animal studies. Another potential option is the 16-N-homopiperidinium analogue of vecuronium (Org 9991), which exhibits non-depolarizing neuromuscular blocking activity and is reversible by neostigmine. These agents could be considered for further investigation and potential use in treating Botulism.

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Potassium Channel Blocker

Amifampridine for Botulism

Phase 1

Recruiting

Led By James B Caress, MD

Research Sponsored by Wake Forest University Health Sciences

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up hour 3

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing if amifampridine can help improve muscle function in patients who have received BTX-A injections. Amifampridine works by boosting the signals between nerves and muscles. The goal is to see if it can counteract the muscle weakening effects of BTX-A.

Who is the study for?

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.

What is being tested?

The study tests if amifampridine can enhance muscle response in patients previously treated with Botox. Participants will receive the drug and undergo specialized muscle testing to measure any improvements in neuromuscular transmission.

What are the potential side effects?

Amifampridine may cause side effects such as tingling sensations, sleep disturbances, digestive discomforts like diarrhea or stomach pain. It's also known for potentially causing seizures in some individuals.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ hour 3

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~hour 3

This trial's timeline: 3 weeks for screening, Varies for treatment, and hour 3 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Percentage of Jitter

Percentage of abnormal pairs

Percentage of pairs that show blocking

Side effects data

From 2020 Phase 3 trial • 93 Patients • NCT03304054

43%

Paraesthesia oral

40%

Paraesthesia

16%

Nausea

14%

Diarrhoea

13%

Headache

12%

Fatigue

10%

Hypoaesthesia oral

Dizziness

Abdominal pain upper

Dyspepsia

Muscle spasms

Peripheral coldness

Abdominal discomfort

Pain in extremity

Hypoaesthesia

Feeling cold

Urinary tract infection

Sensory disturbance

Vomiting

Asthenia

Nasopharyngitis

Abdominal pain

Back pain

Tinnitus

Dyspnoea

Oropharyngeal pain

Rash maculo-papular

Palpitations

Anxiety

Feeling hot

Ear infection

Sinusitis

Diplopia

Fall

Muscular weakness

Speech disorder

Insomnia

Cough

Myasthenia gravis

Myasthenia gravis crisis

100%

80%

60%

40%

20%

Study treatment Arm

Amifampridine Phosphate

Placebo

Overall

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Amifampridine will be orally administered to study participantsExperimental Treatment1 Intervention

Amifampridine will be orally administered to study participants following completion of the baseline SFEMG. Post-dose SFEMG will commence at 30 minutes following dosing and will be completed within 30 minutes. The participant will remain under observation in the Diagnostic Neurology suite for 2 hours after dosing so it is estimated that the entire protocol including monitoring will be completed within 2-3 hours.

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Botulism focus on counteracting the neuromuscular blockade caused by botulinum toxin. Amifampridine, a potassium channel blocker, enhances acetylcholine release at the neuromuscular junction, improving muscle function. This is particularly important for Botulism patients as the toxin inhibits acetylcholine release, leading to muscle paralysis. Other treatments, such as 3,4-diaminopyridine, also work by increasing acetylcholine release, thereby antagonizing the effects of the toxin. Avoiding aminoglycoside antibiotics is crucial, as they can exacerbate the neuromuscular blockade. These treatments are vital for restoring muscle function and preventing severe complications in Botulism patients.

Aminoglycosides and 3,4-diaminopyridine on neuromuscular block caused by botulinum type A toxin.Interactions of anticholinesterases with Achatina fulica acetylcholine responses and electrogenic sodium pump.Pilocarpine for the treatment of refractory dry mouth (xerostomia) associated with botulinum toxin type B.