Firdapse for Post-BOTOX Vocal Weakness
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|ClinicalTrials.gov Identifier: NCT05123053|
Recruitment Status : Recruiting
First Posted : November 17, 2021
Last Update Posted : November 17, 2021
|Condition or disease||Intervention/treatment||Phase|
|Post-BOTOX Vocal Weakness||Drug: Amifampridine||Phase 2|
Spasmodic dysphonia (SD) is a dystonia which results in vocal breaks. The mainstay of treatment involves injections using Botox (onabotulinumtoxinA), a neuromuscular blocker which inhibits pre-synaptic release of acetylcholine into the neuromuscular junction. Botox injections into the thyroarytenoid muscle are a predictable and effective treatment for SD, but typically result in transient symptoms of voice weakness and breathiness during the first 2-3 weeks after injection. These symptoms can be present for even longer if Botox is over- dosed.
We hypothesize that these initial, transient symptoms of voice weakness and breathiness after Botox treatment can be alleviated using amifampridine which acts at the neuromuscular junction to increase synaptic presence of the neurotransmitter acetylcholine. In these initial studies we will look at patients who have significant breathiness following an injection.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Amifampridine for the Treatment of Transient Vocal Weakness After OnabotulinumtoxinA Injection for Spasmodic Dysphonia|
|Actual Study Start Date :||October 28, 2021|
|Estimated Primary Completion Date :||June 30, 2023|
|Estimated Study Completion Date :||June 30, 2023|
Experimental: Treatment Arm
subjects will be given active drug and titrated up in 10mg increments (with max single dose being 30mg) until a change or drug side effect is noticed.
Other Name: Firdapse
- Voice Handicap Index-10 [ Time Frame: over 2 months ]scale to assess the severity of problematic symptoms associated with the voice
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05123053
|Contact: Brandy Quarles, MPHfirstname.lastname@example.org|
|Contact: Kristy Bouchard, BSemail@example.com|
|United States, Georgia|
|Augusta, Georgia, United States, 30912|
|Contact: Brandy M Quarles, MPH 706-721-0390 firstname.lastname@example.org|
|Contact: Kristy Bouchard, BS 706-721-0390 email@example.com|
|Principal Investigator: Michael H Rivner, MD|
|Principal Investigator:||Michael H Rivner, MD||Charbonnier Professor Emeritus of Neurology|