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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. Investigators hypothesize that voice weakness and breathiness after Botox treatment can be alleviated using amifampridine.
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.
Investigators 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, investigators will look at patients who have significant breathiness following an injection.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Arm | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Amifampridine | Drug | 10mg tablet |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Voice Handicap Index-10 | VHI-10 is a scale used to assess the severity of problematic symptoms associated with the voice. It is a series of 10 questions. Each questions is answered using numbers 0-4, with 0 meaning the patient never has a problem with that symptom; and, 4 meaning the patient always has a problem with that symptom. | over 2 months |
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Inclusion Criteria:
A.NOTE: No adequate clinical data on exposed pregnancies are available for amifampridine. No nonclinical safety data are available regarding the effects of amifampridine on reproductive function. Amifampridine phosphate should not be used during pregnancy. It is unknown whether amifampridine is excreted in human breast milk. The excretion of amifampridine in milk has not been studied in animals. Amifampridine phosphate should not be used during breastfeeding. Acceptable methods of birth control include:
B. NOTE: Periodic abstinence (calendar, symptothermal, postovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhea method are not acceptable methods of contraception.
Exclusion Criteria:
History of epilepsy and/or on medication/treatment for seizures (such as but not limited to valproic acid, lamotrigine, topiramate, carbamazepine, and phenytoin); or, on certain medications that may lower seizure threshold, such as:
Any SGOT, SGPT that are more than 3x upper limit of normal; and, creatinine that is greater than 2.1
Women who are pregnant, expecting to get pregnant, or breastfeeding.
Any condition that, in the view of the Principal Investigator, places the subject at risk, or subjects with poor treatment compliance.
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| Name | Affiliation | Role |
|---|---|---|
| Michael H Rivner, MD | Charbonnier Professor Emeritus of Neurology | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Augusta University | Augusta | Georgia | 30912 | United States |
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| ID | Term |
|---|---|
| D055154 | Dysphonia |
| ID | Term |
|---|---|
| D014832 | Voice Disorders |
| D007818 | Laryngeal Diseases |
| D012140 | Respiratory Tract Diseases |
| D010038 | Otorhinolaryngologic Diseases |
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| ID | Term |
|---|---|
| D000077770 | Amifampridine |
| ID | Term |
|---|---|
| D015761 | 4-Aminopyridine |
| D000631 | Aminopyridines |
| D000588 | Amines |
| D009930 | Organic Chemicals |
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| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D011725 |
| Pyridines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |