Effect of Amifampridine on Neuromuscular Transmission in Patients Treated With OnabotulinumtoxinA

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Primary Purpose

Status

Not yet recruiting

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Amifampridine

About this trial

This is an interventional treatment trial for Botulism focused on measuring botulinum toxin injections, Systemic botulism, neuromuscular transmission, single fiber electromyography, amifampridine

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

Wake Forest University Health Sciences

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Amifampridine will be orally administered to study participants

Arm Description

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.

Outcomes

Primary Outcome Measures

Percentage of abnormal pairs

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

Percentage of Jitter

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

Percentage of pairs that show blocking

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

Secondary Outcome Measures

Full Information

NCT ID

NCT05769478

First Posted

March 3, 2023

Last Updated

August 29, 2023

Sponsor

Wake Forest University Health Sciences

1. Study Identification

Unique Protocol Identification Number

NCT05769478

Brief Title

Effect of Amifampridine on Neuromuscular Transmission in Patients Treated With OnabotulinumtoxinA

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

March 2023

Overall Recruitment Status

Not yet recruiting

Study Start Date

October 2023 (Anticipated)

Primary Completion Date

June 2024 (Anticipated)

Study Completion Date

June 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Wake Forest University Health Sciences

4. Oversight

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

Data Monitoring Committee

No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Botulism, Iatrogenic Botulism

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Model Description

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

Masking

None (Open Label)

Allocation

N/A

Enrollment

20 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Amifampridine will be orally administered to study participants

Arm Type

Experimental

Arm Description

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.

Intervention Type

Drug

Intervention Name(s)

Amifampridine

Other Intervention Name(s)

Firdapse

Intervention Description

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

Primary Outcome Measure Information:

Title

Percentage of abnormal pairs

Description

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

Time Frame

Hour 3

Title

Percentage of Jitter

Description

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

Time Frame

Hour 3

Title

Percentage of pairs that show blocking

Description

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

Time Frame

Hour 3

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

80 Years

Accepts Healthy Volunteers

No

Eligibility Criteria

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Katherinne S Velasquez

Phone

336.716.6031

Email

kserrano@wakehealth.edu

First Name & Middle Initial & Last Name or Official Title & Degree

Mozhdeh Marandi

Phone

336.713.8577

Email

mmarandi@wakehealth.edu

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

James B Caress, MD

Organizational Affiliation

Wake Forest University Health Sciences

Official's Role

Principal Investigator

Facility Information:

Facility Name

Wake Forest University Health Sciences

City

Winston-Salem

State/Province

North Carolina

ZIP/Postal Code

27157

Country

United States

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Katherinne S Velasquez

Phone

336-716-6031

Email

kserrano@wakehealth.edu

First Name & Middle Initial & Last Name & Degree

James B Caress, MD

12. IPD Sharing Statement

Plan to Share IPD

No

Botulism, Iatrogenic Botulism

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial