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Firdapse for Post-BOTOX Vocal Weakness

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05123053
Recruitment Status : Recruiting
First Posted : November 17, 2021
Last Update Posted : November 17, 2021
Sponsor:
Information provided by (Responsible Party):
Augusta University

Brief Summary:
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. We hypothesize that voice weakness and breathiness after Botox treatment can be alleviated using amifampridine.

Condition or disease Intervention/treatment Phase
Post-BOTOX Vocal Weakness Drug: Amifampridine Phase 2

Detailed Description:

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Botox

Arm Intervention/treatment
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.
Drug: Amifampridine
10mg tablet
Other Name: Firdapse




Primary Outcome Measures :
  1. Voice Handicap Index-10 [ Time Frame: over 2 months ]
    scale to assess the severity of problematic symptoms associated with the voice



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Male or female ≥18 years of age.
  2. Capable of providing informed consent.
  3. Confirmed physician diagnosis of spasmodic dysphonia.
  4. Receives onabotulinumtoxinA for treatment of their spasmodic dysphonia.
  5. Experiences significant breathiness ± 5 days following their injection.

Exclusion Criteria:

  1. History of epilepsy and on medication/treatment for the same.
  2. Women who are pregnant, expecting to get pregnant, or breastfeeding.
  3. Any condition that, in the view of the Principal Investigator, places the subject at risk, or subjects with poor treatment compliance.

Information from the National Library of Medicine

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


Contacts
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Contact: Brandy Quarles, MPH 706-721-0390 bquarles@augusta.edu
Contact: Kristy Bouchard, BS 706-721-0390 kbouchard@augusta.edu

Locations
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United States, Georgia
Augusta University Recruiting
Augusta, Georgia, United States, 30912
Contact: Brandy M Quarles, MPH    706-721-0390    bquarles@augusta.edu   
Contact: Kristy Bouchard, BS    706-721-0390    kbouchard@augusta.edu   
Principal Investigator: Michael H Rivner, MD         
Sponsors and Collaborators
Augusta University
Investigators
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Principal Investigator: Michael H Rivner, MD Charbonnier Professor Emeritus of Neurology
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Responsible Party: Augusta University
ClinicalTrials.gov Identifier: NCT05123053    
Other Study ID Numbers: SD-DAP-01
First Posted: November 17, 2021    Key Record Dates
Last Update Posted: November 17, 2021
Last Verified: November 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Augusta University:
BOTOX
Onabotulinumtoxin
Spasmodic Dysphonia
Additional relevant MeSH terms:
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Amifampridine
Neuromuscular Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action