Effect of Amifampridine on Neuromuscular Transmission in Patients Treated With OnabotulinumtoxinA

March 1, 2024 updated by: Wake Forest University Health Sciences

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

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Estimated)

20

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • North Carolina
      • Winston-Salem, North Carolina, United States, 27157
        • Recruiting
        • Wake Forest University Health Sciences
        • Contact:
        • Principal Investigator:
          • James B Caress, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of abnormal pairs
Time Frame: Hour 3
Wilcoxon's test for paired data will be used to analyze baseline and post-treatment percent abnormal pairs
Hour 3
Percentage of Jitter
Time Frame: Hour 3
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
Time Frame: Hour 3
Wilcoxon's test for paired data will be used to analyze baseline and post-treatment percent blocking
Hour 3

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Wake Forest University Health Sciences

Investigators

  • Principal Investigator: James B Caress, MD, Wake Forest University Health Sciences

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 15, 2023

Primary Completion (Estimated)

June 1, 2024

Study Completion (Estimated)

June 1, 2024

Study Registration Dates

First Submitted

March 3, 2023

First Submitted That Met QC Criteria

March 3, 2023

First Posted (Actual)

March 15, 2023

Study Record Updates

Last Update Posted (Estimated)

March 4, 2024

Last Update Submitted That Met QC Criteria

March 1, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IRB00091315

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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