Babble Boot Camp for Speech and Language Disorders in Galactosemia

Overseen By Linda Eng, M.S.

Age: < 18

Sex: Any

Trial Phase: Phase < 1

Sponsor: Arizona State University

Disqualifiers: Other galactosemia, Trisomy 21, deafness, others

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

Compensation: Varies

Number of Visits: Unknown

285 Participants Needed

Trial Summary

What is the purpose of this trial?

A critical knowledge gap is whether proactive intervention can improve speech and language outcomes in infants at known risk for communication disorders. Speech and language assessments and treatments are usually not initiated until deficits can be diagnosed, no earlier than age 2-3 years. Preventive services are not available. Children with classic galactosemia (CG) hold the keys towards investigating whether proactive services are more effective than conventional management. CG is a recessively inherited inborn error of metabolism characterized by defective conversion of galactose. Despite early detection and strict adherence to lactose-restricted diets, children with CG are at very high risk not only for motor and learning disabilities but also for severe speech sound disorder and language impairment. Delays are evident from earliest signals of communication and persist into adulthood in many cases but speech/language assessment and treatment are usually not initiated until deficits manifest. However, because CG is diagnosed via newborn screening, the known genotype-phenotype association can be leveraged to investigate the efficacy of proactive interventions during the acquisition of prespeech (2 to 12 months) and early communication skills (13 to 24 months). If this proactive intervention is more effective than standard care regarding speech and language outcomes in children with CG, this will change their clinical management from deficit-based to proactive services. It will also motivate investigating this approach in infants with other types of known risk factors, e.g., various genetic causes and very low birth weight. The Babble Boot Camp is a program for children with CG, ages 2 to 24 months. The intervention is implemented by a pediatric speech-language pathologist (SLP) via parent training. Activities and routines are designed to foster earliest signals of communication, increase coo and babble behaviors, support the emergence of first words and word combinations, and expand syntactic complexity. The SLP meets with parents online every week for 10 to 15 minutes to provide instruction, feedback, and guidance. Close monitoring of progress is achieved via regularly administered questionnaires, a monthly day-long audio recording, and the SLPs weekly progress notes. At age 24 months, the active phase of the Babble Boot Camp ends. The children receive a professional speech/language assessment at ages 2 1/2, 3 1/2, and 4 1/2 years.

How is the Babble Boot Camp treatment different from other treatments for speech and language disorders in galactosemia?

Babble Boot Camp is unique because it is a proactive, parent-implemented program designed to prevent speech and language disorders in infants with classic galactosemia, starting from less than 6 months of age. Unlike traditional therapies that begin after delays are observed, this treatment involves early intervention through parent training and is delivered entirely via telepractice, making it more accessible. ^{1 2 3}

Eligibility Criteria

This trial is for children aged 2-24 months with classic galactosemia, from any ethnic or racial background and geographic region. Parents must speak English at home, have an 8th grade education level to complete questionnaires, and access to a computer with internet (assistance available). Children with other forms of galactosemia or conditions like Trisomy 21 or deafness are excluded.

Inclusion Criteria

Any geographic region in the US and other countries because the intervention is done online

At least one parent must have at least an 8th grade education to be able to fill out the questionnaires

Any ethnic or racial background

Exclusion Criteria

I do not have conditions like Down syndrome or deafness that could affect the study.

I have a type of galactosemia that is not the classic form.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Intervention

Babble Boot Camp intervention implemented by a pediatric speech-language pathologist via parent training, focusing on fostering communication skills in children with classic galactosemia.

22 months

Weekly online meetings with SLP

Follow-up

Participants receive professional speech/language assessments at ages 2 1/2, 3 1/2, and 4 1/2 years to monitor long-term outcomes.

3 years

Treatment Details

Interventions

• Babble Boot Camp

Trial Overview The Babble Boot Camp program is being tested on infants at risk for speech and language disorders due to classic galactosemia. It's an online intervention by speech-language pathologists through parent training aimed at improving early communication skills. Progress is monitored weekly until the child reaches age two.

Participant Groups

Group I: Treatment cohort with classic galactosemia, delayed start Experimental Treatment 1 Intervention

The children in the control cohort enter the study when they are younger than 5 months old and participate in the close monitoring until they are 24 months old. They start getting the same treatment type and intensity as the treatment cohort but at a delayed age, when they turn 15 months.

Group II: Treatment cohort with classic galactosemia Experimental Treatment 1 Intervention

These children and their parents receive the Babble Boot Camp intervention and also participate in the close monitoring activities (progress reports that the speech-language pathologist generates during the online meeting with the family; monthly daylong audio recording; questionnaires that are sent out every three to six months; formal speech and language testing at ages 2 1/2, 3 1/2, and 4 1/2 years).

Group III: Older control cohort with classic galactosemia Active Control 1 Intervention

The children in the older control cohort are 6 months to 4 1/2 years old and provide standardized test results in the area of speech and language development at child ages 2 1/2, 3 1/2, and 4 1/2 years. They receive no treatment and no close monitoring. These families provide questionnaire information every three months until child age 24 months.

Group IV: Typical controls Active Control 1 Intervention

These children are free of any medical or developmental diagnosis. They enter the study at ages 2 to 5 months and provide close monitoring data until they are 24 months old, then they receive standardized speech and language testing at ages 2 1/2, 3 1/2, and 4 1/2 years, just like the treatment cohort, but the typical controls receive no treatment under this study.

Who Is Running the Clinical Trial?

Arizona State University

Lead Sponsor

Trials

311

Recruited

109,000+

Washington State University

Collaborator

Trials

114

Recruited

58,800+

References

1. United States pubmed.ncbi.nlm.nih.gov

Toward Preventing Speech and Language Disorders of Known Genetic Origin: First Post-Intervention Results of Babble Boot Camp in Children With Classic Galactosemia. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Feasibility of a Proactive Parent-Implemented Communication Intervention Delivered via Telepractice for Children With Classic Galactosemia. [2023]

3. England pubmed.ncbi.nlm.nih.gov

Toward a paradigm shift from deficit-based to proactive speech and language treatment: Randomized pilot trial of the Babble Boot Camp in infants with classic galactosemia. [2020]