DaxibotulinumtoxinA Injection for Spasmodic Dysphonia

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: University of California, San Francisco

Must be taking: BotoxA

Disqualifiers: Abductor spasmodic dysphonia, ALS, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 1

Duration: 6 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness of a new treatment called daxibotulinumtoxinA (DAXI) injection for individuals with adductor spasmodic dysphonia, a condition causing voice problems due to throat muscle spasms. The researchers aim to determine if DAXI can provide longer-lasting relief compared to current treatments, which require frequent doctor visits. This trial may suit those who have been successfully treated with BotoxA for adductor spasmodic dysphonia and have maintained a stable dose in recent treatments. As a Phase 1, Phase 2 trial, this research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It might be best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that DaxibotulinumtoxinA Injection is likely to be safe for humans?

Research has shown that DaxibotulinumtoxinA (DAXI) has been safely used for conditions like neck muscle spasms and frown lines. In these studies, participants received DAXI multiple times, and it generally went well. The most common side effects were mild, including muscle weakness (about 5%), pain at the injection site (about 4%), and difficulty swallowing (about 4%).

Overall, past research indicates that DAXI appears to be a safe treatment. This is encouraging for those considering it for voice box spasms, as it suggests the treatment might be well-tolerated. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for spasmodic dysphonia?

Researchers are excited about the DaxibotulinumtoxinA injection for spasmodic dysphonia because it offers a potentially longer-lasting effect compared to current botulinum toxin treatments like onabotulinumtoxinA, commonly known as Botox. This investigational treatment uses daxibotulinumtoxinA, a type of botulinum toxin A, which has already shown effectiveness in other conditions like glabellar lines and cervical dystonia. The promise of extended duration means patients might not need injections as frequently, which could enhance convenience and improve overall patient experience.

What evidence suggests that DaxibotulinumtoxinA Injection might be an effective treatment for spasmodic dysphonia?

Research shows that daxibotulinumtoxinA (DAXI) is safe and effective for treating conditions like cervical dystonia, a muscle disorder in the neck. This treatment may last longer than others, requiring fewer injections over time. In past studies, patients experienced benefits for about 16 weeks, longer than the usual 12 weeks for similar treatments. Although researchers have not specifically tested DAXI for spasmodic dysphonia yet, this trial will investigate its potential effectiveness for this condition. The longer-lasting effects observed in other conditions could be especially beneficial for those needing regular injections. ^{1 2 3 6 7}

Who Is on the Research Team?

Clark Rosen, MD

Principal Investigator

University of California, San Francisco

Are You a Good Fit for This Trial?

This trial is for adults with adductor spasmodic dysphonia, a voice disorder causing muscle contractions that affect speech. Participants must have had successful previous treatments with Botox and maintained a stable dose in their last three treatments. It's not open to individuals under 18 or those with certain neurological conditions like ALS, multiple sclerosis, Parkinson's disease, or essential tremor.

Inclusion Criteria

I have been diagnosed with adductor spasmodic dysphonia.

I have had successful treatment with Botox before.

My medication dose has been the same for my last 3 treatments.

Exclusion Criteria

I am under 18 years old.

You have a different neurological condition like ALS, multiple sclerosis, or Parkinson's disease.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive DAXI injections for treatment of adductor spasmodic dysphonia

6 weeks

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 1 year

What Are the Treatments Tested in This Trial?

Interventions

• DaxibotulinumtoxinA Injection

Trial Overview The study tests DaxibotulinumtoxinA injections as a treatment for adductor spasmodic dysphonia. This condition affects the muscles responsible for voice control. The goal is to see if this new treatment can improve voice quality longer than current options like onabotulinumtoxinA (Botox).

How Is the Trial Designed?

Group I: Administration of Daxi Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

University of California, San Francisco

Lead Sponsor

Trials

2,636

Recruited

19,080,000+

Citations

1. movementdisorders.onlinelibrary.wiley.com movementdisorders.onlinelibrary.wiley.com/doi/10.1002/mdc3.70104

Long‐Term Safety and Efficacy of Repeat Treatments with ... DaxibotulinumtoxinA (DAXI), a novel botulinum neurotoxin (BoNT) formulation, was shown to be safe, effective, and long-lasting in the ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40439027/

Long-Term Safety and Efficacy of Repeat Treatments with ... DAXI was safe and efficacious over repeated treatments in adults with CD. Adverse event rates were similar to or potentially lower compared with conventional ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11946516/

Efficacy Remaining at Time of Requested Re-Treatment ... The median percent efficacy remaining at the time of requested re-injection was 45.5%, which corresponded to a median of 16.0 weeks (range 10.9–40.3) post- ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT05158166

Study Details | NCT05158166 | DaxibotulinumtoxinA ... This study aims to assess the efficacy of DAXI for transcervical laryngeal injection in patients with adductor spasmodic dysphonia. Official Title.

5. movementdisorders.onlinelibrary.wiley.com movementdisorders.onlinelibrary.wiley.com/doi/abs/10.1002/mdc3.70104

Long‐Term Safety and Efficacy of Repeat Treatments with ... DAXI was safe and efficacious over repeated treatments in adults with CD. Adverse event rates were similar to or potentially lower compared with conventional ...

6. researchgate.net researchgate.net/publication/392202595_Long-Term_Safety_and_Efficacy_of_Repeat_Treatments_with_DaxibotulinumtoxinA_in_Cervical_Dystonia_Results_from_the_ASPEN-Open-Label_Study

Results from the ASPEN‐Open‐Label Study The most frequently reported treatment‐related adverse events per treatment were muscular weakness (4.9%), injection‐site pain (4.2%), and dysphagia (3.9%).

7. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40961718/

Efficacy and safety of DaxibotulinumtoxinA for injection in ... Conclusions: This pooled analysis of two phase 3 trials demonstrates that DAXI is an effective, safe, and long-acting treatment for CD. Key ...