Effect of Amifampridine on Neuromuscular Transmission in Patients Treated With OnabotulinumtoxinA

NCT05769478 | Completed Phase 1 FDA Drug

• 1 other identifier: IRB00091315
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Botulism; Iatrogenic Botulism

Keywords

botulinum toxin injections; Systemic botulism; neuromuscular transmission; single fiber electromyography; amifampridine

Outcome Measures

Primary Outcomes (3)

• Percentage of abnormal pairs

  Wilcoxon's test for paired data will be used to analyze baseline and post-treatment percent abnormal pairs

  Hour 3

• Percentage of Jitter

  Wilcoxon's test for paired data will be used to analyze baseline and post-treatment mean jitter

  Hour 3

• Percentage of pairs that show blocking

  Wilcoxon's test for paired data will be used to analyze baseline and post-treatment percent blocking

  Hour 3

Study Arms (1)

Amifampridine will be orally administered to study participants

EXPERIMENTAL

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

Interventions

Amifampridine DRUG

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

Also known as: Firdapse

Amifampridine will be orally administered to study participants

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Wake Forest University Health Sciences: lead

Study Sites (1)

Wake Forest University Health Sciences

Winston-Salem, North Carolina, 27157, United States

Location

MeSH Terms

Conditions

Botulism

Interventions

Amifampridine

Condition Hierarchy (Ancestors)

Clostridium Infections; Gram-Positive Bacterial Infections; Bacterial Infections; Bacterial Infections and Mycoses; Infections; Neuromuscular Junction Diseases; Neuromuscular Diseases; Nervous System Diseases; Neurotoxicity Syndromes; Foodborne Diseases; Poisoning; Chemically-Induced Disorders

Intervention Hierarchy (Ancestors)

4-Aminopyridine; Aminopyridines; Amines; Organic Chemicals; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• James B Caress, MD

  Wake Forest University Health Sciences

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: before-after study that will measure neuromuscular jitter before and after administration of amifampridine

Study Record Dates

• First Submitted: March 3, 2023
• First Posted: March 15, 2023
• Study Start: September 15, 2023
• Primary Completion: October 29, 2024
• Study Completion: October 29, 2024
• Last Updated: February 3, 2025

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)