Effect of Amifampridine on Neuromuscular Transmission in Patients Treated With OnabotulinumtoxinA

Phase 1

Completed

• Conditions: Botulism; Iatrogenic Botulism
• Interventions: Drug: Amifampridine

• Registration Number: NCT05769478
• Lead Sponsor: Wake Forest University Health Sciences

Brief Summary

if amifampridine can improve neuromuscular transmission in muscles previously injected with OnabotulinumtoxinA (BTX-A)

Detailed Description

Amifampridine may also be effective in treating the sequelae of botulinum toxin injections and this study will determine if neuromuscular transmission as measured by single fiber electromyography is improved in patients treated with BTX-A following administration of amifampridine

Study Timeline

• Study First Submitted: 2023-03-03
• Study First Submitted QC: 2023-03-03
• Study First Posted: 2023-03-15
• Study Start: 2023-09-15
• Status Verified: 2024-03
• Primary Completion: 2024-10-29
• Study Completion: 2024-10-29
• Last Update Submitted: 2025-01-30
• Last Update Posted: 2025-02-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 21

Inclusion Criteria

• Age: 18-80 years of either gender and of any racial background
• Underwent BTX-A injection of facial muscles including frontalis with a total dose between 100-200 units between 80 and 150 days prior to study
• Have decision-making capacity to provide informed consent to study drug dosing and Single Fiber Electromyography (SFEMG)

Exclusion Criteria

• History of cardiac arrhythmia
• History of seizures or uncontrolled asthma
• History of renal or hepatic disease
• History of any generalized neuromuscular disease
• History of Bell's Palsy or facial nerve trauma
• History of treatment with or sensitivity to amifampridine, 3,4 diaminopyridine (DAP) or 4-aminopyridine (Ampyra®)
• Currently experiencing sequelae of previous BTX-A treatment
• Current use of pyridostigmine (known to alter neuromuscular transmission)
• Use of any investigational drug or device within 30 days of enrollment

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Amifampridine will be orally administered to study participants	Amifampridine	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.

Primary Outcome Measures

Name	Time	Method
Percentage of abnormal pairs	Hour 3	Wilcoxon's test for paired data will be used to analyze baseline and post-treatment percent abnormal pairs
Percentage of Jitter	Hour 3	Wilcoxon's test for paired data will be used to analyze baseline and post-treatment mean jitter
Percentage of pairs that show blocking	Hour 3	Wilcoxon's test for paired data will be used to analyze baseline and post-treatment percent blocking

Trial Locations

Locations (1)

Wake Forest University Health Sciences; 🇺🇸 Winston-Salem, North Carolina, United States