VAULT Phase 4 for Language Disorder

Overseen By Nora 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 Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 16

Duration: 8 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to help late-talking toddlers learn new words more effectively. Researchers are testing two methods: one teaches words that sound similar to those the child already knows, while the other uses words that sound less familiar. By comparing these approaches, they hope to determine which is more effective for language development. Children who speak English, have normal hearing, and are not enrolled in other treatments might be a good fit.

As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering a chance to contribute to early findings in language development.

Will I have to stop taking my current medications?

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

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that the VAULT Phase 4 treatment has been studied for its safety and effects on language development in toddlers who start talking later than usual. One study using a telehealth approach for the VAULT protocol found no major side effects, indicating the treatment is generally well-tolerated. Another study examined a similar treatment, VAULT Phase 5, and also found it to be safe, with no serious side effects reported.

Since the trial remains in an early stage, the treatment appears safe so far, but more research is needed to confirm its safety in larger groups. Overall, current evidence suggests that the treatment is generally safe and does not cause significant issues for participants. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about the VAULT Phase 4 approach for language disorders because it explores how the familiarity of sounds can impact language learning in children. Unlike current language therapies that might focus broadly on speech therapy techniques, this method specifically tests whether learning words with sounds that are more or less familiar helps children better understand and use new vocabulary. By tailoring word learning to sound familiarity, this approach could offer a more personalized and potentially more effective way to boost language development in children with language disorders.

What evidence suggests that this trial's treatments could be effective for language disorder?

Research has shown that the VAULT treatment helps children who are late talkers expand their vocabulary. Studies have found that children using VAULT learn to say words they didn't know before the treatment. Another study confirmed that VAULT helps children learn new words faster. In this trial, participants will be assigned to different treatment arms: one focusing on learning new words with more familiar sounds and another with less familiar sounds. Different versions of the treatment have shown better results than no treatment at all. Overall, VAULT appears promising for helping children with language development challenges. ^{4 5 6 7 8}

Who Is on the Research Team?

Mary Alt, PhD

Principal Investigator

University of Arizona

Are You a Good Fit for This Trial?

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.

Exclusion Criteria

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

Enrolled in concurrent treatment elsewhere

Information from parents about other medical conditions.

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

• VAULT Phase 4

Trial Overview The 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.

How Is the Trial Designed?

Group I: More Familiar Sounds Experimental Treatment 1 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 Sounds Active Control 1 Intervention

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

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+

Published Research Related to This Trial

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 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]

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]

Developing quantitative biomarkers as Drug Development Tools (DDTs) is crucial for improving drug approvals in Central Nervous System (CNS) diseases, which are notoriously difficult to treat.

The review emphasizes the need for validated and regulatory-accepted biomarkers across various CNS conditions, highlighting the importance of collaboration and data sharing to accelerate the development of effective therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Regulatory-accepted drug development tools are needed to accelerate innovative CNS disease treatments. Arnerić, SP., Kern, VD., Stephenson, DT.[2019]

A systematic assessment revealed that over half of the clinical outcomes for multiple sclerosis (55% from NINDS and 52% from ClinicalTrials.gov) are not covered by the CDISC Controlled Terminology, highlighting a significant gap in the terminology's comprehensiveness.

This study suggests that expanding the CDISC terminology is necessary to better align with established clinical standards and improve the representation of clinical trial data in multiple sclerosis research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparative Assessment of Completeness of CDISC Controlled Terminology. Elghafari, A., Finkelstein, J.[2022]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8608147/

A Vocabulary Acquisition and Usage for Late Talkers ... This study examined the efficacy of the Vocabulary Acquisition and Usage for Late Talkers (VAULT) treatment in a version that manipulated the length of ...

2. asha.figshare.com asha.figshare.com/articles/online_resource/VAULT_context_variability_Alt_et_al_2025_/28200074

VAULT context variability (Alt et al., 2025) - ASHA journals VAULT, with a focus on context variability, can be used efficaciously to teach children to say words they do not understand at the start of treatment.

3. ldbase.org ldbase.org/system/files/documents/2024-12/VAULT_TALK_Codebook_Guide_Wave1.pdf

Codebook Guide for A Longitudinal Assessment of Late- ... The primary goal of VAULT was to see if children increased their rate of expressive vocabulary acquisition following the VAULT treatment protocol. The primary ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7213481/

Exploring Input Parameters in an Expressive Vocabulary ... We would be able to replicate the findings from Alt et al. (2014) using the VAULT protocol and find evidence that treatment was more effective than no treatment ...

5. researchgate.net researchgate.net/publication/350511071_A_Vocabulary_Acquisition_and_Usage_for_Late_Talkers_Treatment_Efficacy_Study_The_Effect_of_Input_Utterance_Length_and_Identification_of_Responder_Profiles

(PDF) A Vocabulary Acquisition and Usage for Late Talkers ... This study examined the efficacy of the Vocabulary Acquisition and Usage for Late Talkers (VAULT) treatment in a version that manipulated the length of ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10023173/

Vocabulary Acquisition and Usage for Late Talkers This feasibility study examined a caregiver-implemented telehealth model of the Vocabulary Acquisition and Usage for Late Talkers (VAULT) protocol.

7. withpower.com withpower.com/trial/semantic-support-for-language-development-in-late-talking-toddlers-a200e

Semantic Support for Language Development in Late ... This N/A medical study run by University of Arizona is evaluating whether VAULT Phase 5 will have tolerable side effects & efficacy for patients with Speech ...

8. clinicaltrials.gov clinicaltrials.gov/study/NCT05921227

NCT05921227 | Addressing Treatment Nonresponders Once children are shown to not respond to the usual Vocabulary Acquisition and Usage for Late Talkers (VAULT) treatment, they will be placed in this arm, which ...