Effect of Amifampridine on Neuromuscular Transmission in Patients Treated With OnabotulinumtoxinA

Sponsor

Wake Forest University Health Sciences (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT05769478

Collaborator

(none)

Enrollment

Location

Arm

Anticipated Duration (Months)

2.5

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Botulism Iatrogenic Botulism	Drug: Amifampridine	Phase 1

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 Design

Study Type:

Interventional

Anticipated Enrollment :

20 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Intervention Model Description:

before-after study that will measure neuromuscular jitter before and after administration of amifampridinebefore-after study that will measure neuromuscular jitter before and after administration of amifampridine

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Proof of Concept Study of the Effect of Amifampridine (Firdapse®) on Neuromuscular Transmission in Patients Treated With OnabotulinumtoxinA (Botox®, BTX-A)

Anticipated Study Start Date :

Apr 1, 2023

Anticipated Primary Completion Date :

Dec 1, 2023

Anticipated Study Completion Date :

Dec 1, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Amifampridine will be orally administered to study participants 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.	Drug: Amifampridine a single dose of amifampridine (20mg) will be orally administered Other Names: Firdapse

Arm

Intervention/Treatment

Experimental: Amifampridine will be orally administered to study participants

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.

Drug: Amifampridine

a single dose of amifampridine (20mg) will be orally administered

Other Names:

Firdapse

Outcome Measures

Primary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 80 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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