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DaxibotulinumtoxinA Injection for Treatment of Adductor Spasmodic Dysphonia

Primary Purpose

Adductor Spasmodic Dysphonia, Voice Disorders, Spasmodic Dysphonia

Status
Withdrawn
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
DaxibotulinumtoxinA
Sponsored by
University of California, San Francisco
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Adductor Spasmodic Dysphonia focused on measuring adductor spasmodic dysphonia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • greater than 18
  • diagnosis of adductor spasmodic dysphonia
  • previous successful treatment with BotoxA
  • stabilized dose for last 3 treatments

Exclusion Criteria:

  • age less than 18
  • exclusively having other neurologic conditions such as:

    • abductor spasmodic dysphonia
    • ALS
    • Multiple sclerosis
    • Parkinson's disease
    • Essential tremor

Sites / Locations

  • UCSF

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Administration of Daxi

Arm Description

Injectible daxibotulinumtoxinA (DAXI, Revance Therapeutics Inc., Newark, CA) is an investigational botulinum toxin type A which has been shown in large clinical trials to provide safe, effective treatment for glabellar lines and cervical dystonia and may offer longer-lasting results when compared with onabotulinumtoxinA. Dosage will be same number of units as prior Botox A dose.

Outcomes

Primary Outcome Measures

Our primary outcome is the difference in VHI-10 score at 6 weeks after injection with DAXI.
This will be determined using a paired t-test with subjects pre-treatment VHI-10 scores as a control.

Secondary Outcome Measures

A secondary outcome will be comparing the duration of effect of DAXI with patients' prior Botox treatment.
Specifically, we will determine the median time to baseline VHI-10 after treatment with DAXI and produce a Kaplan-Meier curve to illustrate the duration of clinical benefit.

Full Information

First Posted
November 19, 2021
Last Updated
March 2, 2022
Sponsor
University of California, San Francisco
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1. Study Identification

Unique Protocol Identification Number
NCT05158166
Brief Title
DaxibotulinumtoxinA Injection for Treatment of Adductor Spasmodic Dysphonia
Official Title
DaxibotulinumtoxinA Injection for Treatment of Adductor Spasmodic Dysphonia
Study Type
Interventional

2. Study Status

Record Verification Date
March 2022
Overall Recruitment Status
Withdrawn
Why Stopped
FDA Approval of DAXI
Study Start Date
October 2022 (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
University of California, San Francisco

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
Spasmodic Dysphonia (SD) is a neurologic condition causing inappropriate contraction of the laryngeal musculature, leading to abnormal voicing. The three types (adductor, abductor, and mixed) affect varying muscle groups which produce characteristic voice patterns. The vast majority of patients with SD have adductor type, which impacts the lateral cricoarytenoid and thyroarytenoid muscle complex. While many treatment modalities have been investigated, the most effective treatment is botulinum toxin injection to these muscle groups, performed transcervically with or without electromyography (EMG) guidance. Patients undergoing this treatment typically require re-injection every 3 months. Due to its specialized nature, the laryngeal injections are not performed routinely outside of academic medical centers; thus, patients may come from a distance to receive this treatment. Both due to the significant impact on voice quality when the injections wear off and the sometimes challenging access to treatment, a longer-acting agent is desired. Injectible daxibotuliumtoxinA (DAXI, Revance Therapeutics Inc., Newark, CA) has been shown in large clinical trials to provide safe, effective treatment for glabellar lines and cervical dystonia and may offer a longer-lasting result when compared with onabotulinumtoxinA. Thus, a study examining the effect of DAXI for patients with adductor spasmodic dysphonia is proposed. This study aims to assess the efficacy of DAXI for transcervical laryngeal injection in patients with adductor spasmodic dysphonia.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Adductor Spasmodic Dysphonia, Voice Disorders, Spasmodic Dysphonia
Keywords
adductor spasmodic dysphonia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Administration of Daxi
Arm Type
Experimental
Arm Description
Injectible daxibotulinumtoxinA (DAXI, Revance Therapeutics Inc., Newark, CA) is an investigational botulinum toxin type A which has been shown in large clinical trials to provide safe, effective treatment for glabellar lines and cervical dystonia and may offer longer-lasting results when compared with onabotulinumtoxinA. Dosage will be same number of units as prior Botox A dose.
Intervention Type
Drug
Intervention Name(s)
DaxibotulinumtoxinA
Other Intervention Name(s)
DAXI
Intervention Description
This is a long-acting alternative to traditional Botox A treatment.
Primary Outcome Measure Information:
Title
Our primary outcome is the difference in VHI-10 score at 6 weeks after injection with DAXI.
Description
This will be determined using a paired t-test with subjects pre-treatment VHI-10 scores as a control.
Time Frame
6 weeks
Secondary Outcome Measure Information:
Title
A secondary outcome will be comparing the duration of effect of DAXI with patients' prior Botox treatment.
Description
Specifically, we will determine the median time to baseline VHI-10 after treatment with DAXI and produce a Kaplan-Meier curve to illustrate the duration of clinical benefit.
Time Frame
Up to 1 year

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: greater than 18 diagnosis of adductor spasmodic dysphonia previous successful treatment with BotoxA stabilized dose for last 3 treatments Exclusion Criteria: age less than 18 exclusively having other neurologic conditions such as: abductor spasmodic dysphonia ALS Multiple sclerosis Parkinson's disease Essential tremor
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clark Rosen, MD
Organizational Affiliation
University of California, San Francisco
Official's Role
Principal Investigator
Facility Information:
Facility Name
UCSF
City
San Francisco
State/Province
California
ZIP/Postal Code
94118
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
28614091
Citation
Carruthers J, Solish N, Humphrey S, Rosen N, Muhn C, Bertucci V, Swift A, Metelitsa A, Rubio RG, Waugh J, Quiring J, Shears G, Carruthers A. Injectable DaxibotulinumtoxinA for the Treatment of Glabellar Lines: A Phase 2, Randomized, Dose-Ranging, Double-Blind, Multicenter Comparison With OnabotulinumtoxinA and Placebo. Dermatol Surg. 2017 Nov;43(11):1321-1331. doi: 10.1097/DSS.0000000000001206.
Results Reference
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PubMed Identifier
30009213
Citation
Jankovic J, Truong D, Patel AT, Brashear A, Evatt M, Rubio RG, Oh CK, Snyder D, Shears G, Comella C. Injectable DaxibotulinumtoxinA in Cervical Dystonia: A Phase 2 Dose-Escalation Multicenter Study. Mov Disord Clin Pract. 2018 Apr 26;5(3):273-282. doi: 10.1002/mdc3.12613. eCollection 2018 May-Jun.
Results Reference
background
PubMed Identifier
31609882
Citation
Carruthers JD, Fagien S, Joseph JH, Humphrey SD, Biesman BS, Gallagher CJ, Liu Y, Rubio RG; SAKURA 1 and SAKURA 2 Investigator Group; SAKURA 1 and SAKURA 2 Investigator Group includes the following. DaxibotulinumtoxinA for Injection for the Treatment of Glabellar Lines: Results from Each of Two Multicenter, Randomized, Double-Blind, Placebo-Controlled, Phase 3 Studies (SAKURA 1 and SAKURA 2). Plast Reconstr Surg. 2020 Jan;145(1):45-58. doi: 10.1097/PRS.0000000000006327.
Results Reference
background
PubMed Identifier
21658400
Citation
Stone HF, Zhu Z, Thach TQ, Ruegg CL. Characterization of diffusion and duration of action of a new botulinum toxin type A formulation. Toxicon. 2011 Aug;58(2):159-67. doi: 10.1016/j.toxicon.2011.05.012. Epub 2011 May 31.
Results Reference
background
PubMed Identifier
29655932
Citation
Rumbach A, Aiken P, Novakovic D. RETRACTED: Outcome Measurement in the Treatment of Spasmodic Dysphonia: A Systematic Review of the Literature. J Voice. 2019 Sep;33(5):810.e13-810.e39. doi: 10.1016/j.jvoice.2018.03.011. Epub 2018 Apr 11.
Results Reference
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DaxibotulinumtoxinA Injection for Treatment of Adductor Spasmodic Dysphonia

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