BotulismPhase 1NCT05769478
Effect of Amifampridine on Neuromuscular Transmission in Patients Treated With OnabotulinumtoxinA
A Phase 1 study testing Amifampridine in people with Botulism and Iatrogenic Botulism, run by Wake Forest University Health Sciences at 1 site in United States.
Trial summary
if amifampridine can improve neuromuscular transmission in muscles previously injected with OnabotulinumtoxinA (BTX-A) Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Botulism, Iatrogenic Botulism
- What is being tested?
- Amifampridine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Sep 2023to 29 Oct 2024
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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®)
Show all 9Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157
About this study
Official title: 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)
Read the 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
- Conditions
- Botulism, Iatrogenic Botulism
- Being tested
- Amifampridine
- Keywords
- botulinum toxin injections, Systemic botulism, neuromuscular transmission, single fiber electromyography, amifampridine
- Medical subject headings
- Botulism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AmifampridineDrug
Also called Firdapse
a single dose of amifampridine (20mg) will be orally administered
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Amifampridine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of abnormal pairs
Wilcoxon's test for paired data will be used to analyze baseline and post-treatment percent abnormal pairs
Measured: Hour 3
Percentage of Jitter
Wilcoxon's test for paired data will be used to analyze baseline and post-treatment mean jitter
Measured: 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
Measured: Hour 3
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Mar 2023
- Study start15 Sep 2023
- Primary completion29 Oct 2024
- Study completion29 Oct 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for BotulismStudies of the same condition, open and under way.
- More trials run by Wake Forest University Health SciencesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- before-after study that will measure neuromuscular jitter before and after administration of amifampridine
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Wake Forest University Health Sciences
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- James B Caress, MD, Principal investigator, Wake Forest University Health Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05769478
- Sponsor's protocol ID
- IRB00091315
- Status last verified
- Mar 2024
- First submitted
- 3 Mar 2023
- Last updated
- 3 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Proof of Concept Study of the Effect of Amifampridine (Firdapse®) on Neuromuscular Transmission in Patients Treated With OnabotulinumtoxinA (Botox®, BTX-A)
- ClinicalTrials.gov ID
- NCT05769478
- Acronym / sponsor ID
- IRB00091315
- Conditions
- Botulism, Iatrogenic Botulism
- Keywords
- botulinum toxin injections, Systemic botulism, neuromuscular transmission, single fiber electromyography, amifampridine
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Wake Forest University Health SciencesUniversity, hospital or other
- Study start
- 15 Sep 2023 (actual)
- Primary completion
- 29 Oct 2024 (actual)
- Study completion
- 29 Oct 2024 (actual)
- First posted
- 15 Mar 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- before-after study that will measure neuromuscular jitter before and after administration of amifampridine
| Arm | Type | Receives |
|---|---|---|
| Amifampridine will be orally administered to study participants | Experimental | Drug: AmifampridineAmifampridine 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. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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)
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AmifampridineAlso: Firdapsea single dose of amifampridine (20mg) will be orally administered | Drug | Amifampridine will be orally administered to study participants |
Primary outcome
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
Percentage of Jitter
Time frame: 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
Time frame: Hour 3
Wilcoxon's test for paired data will be used to analyze baseline and post-treatment percent blocking
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Feb 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
James B Caress, MD principal investigator
Wake Forest University Health Sciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Amifampridine in people with Botulism and Iatrogenic Botulism, run by Wake Forest University Health Sciences at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of abnormal pairs (time frame: Hour 3). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Feb 2025.