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Compensation: Varies

Number of Visits: 16

Duration: 8 weeks

32 Participants Needed

VAULT Phase 4 for Language Disorder

Overseen ByNora Evans-Reitz, MS

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: University of Arizona

Disqualifiers: Other diagnoses, Concurrent treatment, Low IQ, others

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

Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

What is the purpose of this trial?

The goal of this clinical trial is to compare two treatment conditions for late talking toddlers. In 8 weeks of word learning treatment, toddlers will be taught words that sound more like the words they already know or sound less like the words they already know. The investigators see which group learns more, to understand if choosing words that let children use the knowledge they already have helps with learning.

Will I have to stop taking my current medications?

The trial information does not specify whether participants must stop taking their current medications.

What safety data exists for the treatment evaluated in VAULT Phase 4 for Language Disorder?

The treatment deutetrabenazine, which may be related to VAULT Phase 4, has been evaluated for Huntington disease chorea and showed a relatively low rate of adverse events (side effects) such as sedation, insomnia, and diarrhea. This suggests it is generally well-tolerated in humans.¹ ² ³ ⁴ ⁵

Research Team

Mary Alt, PhD

Principal Investigator

University of Arizona

Eligibility Criteria

This trial is for English-speaking toddlers aged 2-3 who are late talkers, scoring below the 10th percentile on expressive language scales. They must have normal hearing but can't be in another treatment or have other diagnoses affecting speech, and their nonverbal IQ should be above 75.

Inclusion Criteria

Native English Speaking

My hearing is normal, as confirmed by a test or doctor's report.

I am between 2 and 3 years old.

See 1 more

Exclusion Criteria

My parents can't regularly take me to treatment sessions.

Enrolled in concurrent treatment elsewhere

Information from parents about other medical conditions.

See 1 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 8 weeks of Vocabulary Acquisition and Usage for Late Talkers (VAULT) word learning treatment, with sessions 2 times per week for 30 minutes each

8 weeks

16 visits (in-person)

Follow-up

Participants are monitored for retention and effectiveness of word learning after treatment

4-6 weeks

Treatment Details

Interventions

VAULT Phase 4

Trial OverviewThe study tests a word learning treatment over 8 weeks where one group learns words similar to what they know, while another learns different sounding words. The aim is to see if using familiar sounds helps toddlers learn more effectively.

Participant Groups

2Treatment groups

Experimental Treatment

Active Control

Group I: More Familiar SoundsExperimental Treatment1 Intervention

Children in this condition will learn new words whose sounds are more like the sounds in the words the child already understands.

Group II: Less Familiar SoundsActive Control1 Intervention

Children in this condition will learn new words whose sounds are less like the sounds in the words the child already understands.

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of Arizona

Lead Sponsor

Trials

545

Recruited

161,000+

National Institute on Deafness and Other Communication Disorders (NIDCD)

Collaborator

Trials

377

Recruited

190,000+

Findings from Research

Drugs for neurological diseases without orphan indications consistently required at least two randomized, double-blind, placebo-controlled trials for FDA approval, while only 32% of orphan drugs met this standard, highlighting a significant difference in trial rigor.

Orphan drugs had fewer pivotal trials per drug and smaller trial sizes compared to non-orphan drugs, indicating a need for improved study designs to ensure adequate evidence of efficacy as orphan drug development increases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pivotal studies of orphan drugs approved for neurological diseases.Mitsumoto, J., Dorsey, ER., Beck, CA., et al.[2022]

In a retrospective study of 58 Huntington disease patients treated with deutetrabenazine over an average of 476 days, there was a significant reduction in chorea scores, indicating its effectiveness in managing HD-associated chorea.

The treatment was generally well-tolerated, with a low adverse event rate of 32.8%, and the most common side effects included sedation, insomnia, and diarrhea, supporting its safety profile compared to tetrabenazine.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Real-World Experience With Deutetrabenazine for Huntington Disease Chorea.Curtis, K., Sung, V.[2023]

In a systematic review of 113 trials involving 39,875 patients, there was no significant efficacy advantage for unapproved, disease-modifying interventions compared to placebo in treating neurodegenerative diseases like Alzheimer's, Parkinson's, ALS, and Huntington's disease.

Patients with Alzheimer's disease who received active treatment had a higher risk of serious adverse events and withdrawals due to adverse events, suggesting that these interventions may not only be ineffective but could also pose additional risks.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Risks and benefits of unapproved disease-modifying treatments for neurodegenerative disease.Feustel, AC., MacPherson, A., Fergusson, DA., et al.[2023]