NCT03838016

Preventing Speech and Language Disorders in Children With Classic Galactosemia

Completed EARLY_PHASE1 Last updated 23 January 2026

What this trial tests

EARLY_PHASE1 trial testing Babble Boot Camp in Classic Galactosemia in 285 participants. Completed in 31 August 2025.

Timeline

1 May 2019

Primary endpoint
31 May 2025

31 August 2025

Quick facts

Lead sponsor	Arizona State University
Phase	EARLY_PHASE1
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	single
Primary purpose	treatment
Enrollment	285
Start date	1 May 2019
Primary completion	31 May 2025
Estimated completion	31 August 2025
Sites	1 location across United States

Drugs / interventions tested

• Babble Boot Camp

Conditions studied

• Classic Galactosemia — all drugs for Classic Galactosemia →
• Speech Disorders in Children — all drugs for Speech Disorders in Children →
• Language Disorders in Children — all drugs for Language Disorders in Children →

Sponsor

Arizona State University

Who can join

Adults 2 Months to 54 Months, any sex, with Classic Galactosemia or Speech Disorders in Children. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

1. Galactosemia: Biochemistry, Molecular Genetics, Newborn Screening, and Treatment.
   Succoio M, Sacchettini R, Rossi A, Parenti G, et al · · 2022 · cited 37× · PMID 35883524 · DOI 10.3390/biom12070968
2. 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.
   Peter B, Potter N, Davis J, Donenfeld-Peled I, et al · · 2019 · cited 19× · PMID 32566130 · DOI 10.12688/f1000research.18062.5
3. Translating principles of precision medicine into speech-language pathology: Clinical trial of a proactive speech and language intervention for infants with classic galactosemia.
   Peter B, Davis J, Finestack L, Stoel-Gammon C, et al · · 2022 · cited 17× · PMID 35677809 · DOI 10.1016/j.xhgg.2022.100119
4. Toward Preventing Speech and Language Disorders of Known Genetic Origin: First Post-Intervention Results of Babble Boot Camp in Children With Classic Galactosemia.
   Peter B, Davis J, Cotter S, Belter A, et al · · 2021 · cited 15× · PMID 34665663 · DOI 10.1044/2021_ajslp-21-00098
5. Translating the Power of Precision Medicine Into the World of Communication Disorders.
   Peter B, Davis J, Bruce L, Finestack L, et al · · 2025 · cited 3× · PMID 40990846 · DOI 10.1044/2025_jslhr-25-00077
6. Babble Boot Camp for Infants With Down Syndrome: Piloting a Proactive, Caregiver-Led Intervention Designed to Boost Earliest Speech and Language Skills.
   Peter B, Finestack L, Loveall S, Thompson L, et al · · 2025 · cited 1× · PMID 40153250 · DOI 10.1044/2024_ajslp-24-00271
7. 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
   Peter B, Potter N, Davis J, Donenfeld-Peled I, et al · · 2020 · DOI 10.12688/f1000research.18062.5