Babble Boot Camp for Down Syndrome

(BBC-DS Trial)

Overseen By Beate Peter, Ph.D.

Age: < 18

Sex: Any

Trial Phase: Academic

Sponsor: Arizona State University

Disqualifiers: Partial trisomy 21, Awaiting heart surgery, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 10 months

60 Participants Needed

Trial Summary

What is the purpose of this trial?

Children with Down syndrome (DS) face life-long struggles with verbal communication. Babble and speech sound development is delayed, and speech can be difficult to understand. Words emerge late, at 21 months on average, compared to 12 months for typical peers, and vocabulary and grammar can remain limited throughout adulthood. Because DS is diagnosed at or even before birth, these difficulties are predictable; yet despite this prognostic knowledge, systematic and sustained proactive interventions have not yet been developed: Most children with DS are not assessed and treated for speech and language delays until age 2 to 4 years. This presents an untapped opportunity space to conduct a clinical trial of a proactive intervention in earliest infancy with the goal of building resilience against the anticipated difficulties. The intervention trialed here is a modified version of Babble Boot Camp (BBC), a proactive speech and language intervention originally developed for young infants with classic galactosemia (CG) (NIH 5R01HD098253). CG is a metabolic disease that, similar to DS, is diagnosed at birth and poses risks for severe speech and language delays. BBC is implemented by a speech-language pathologist who, via telehealth, trains parents to incorporate skill-building activities and routines into their daily lives at home. For the present study, 20 children with DS age birth to 12 months will be recruited and randomized into two treatment arms. One group will receive weekly individualized parent sessions and close monitoring of the child's progress. The second group will receive the same content but at a lower intensity and dosage, via monthly parent group meetings. Both groups will receive their intervention for 10 months. Specific aims are to quantify benefits for babble, speech production, and receptive and expressive language and to investigate associations between conversational dynamics in child-adult interactions and the children's speech and language. Outcomes in speech and language skills will show relative feasibility and benefits for each of these treatment modalities and motivate a larger clinical trial, with the ultimate goal of changing the way infants with DS receive support in their speech and language development, from a deficit-based, remedial model to a proactive one.

Do I need to stop my current medications for this trial?

The trial protocol does not specify whether you need to stop taking your current medications.

What data supports the idea that Babble Boot Camp for Down Syndrome is an effective treatment?

The available research does not provide specific data on the effectiveness of Babble Boot Camp for Down Syndrome. However, it does mention other interventions for Down Syndrome, such as early intervention programs and computer-based phonics training, which show mixed results. Early intervention programs show short-term benefits in motor skills and social abilities but have inconsistent results in language and cognitive areas. Computer-based phonics training shows some individual improvements in reading skills, but overall benefits are limited. Therefore, while there is some evidence of benefits from other treatments, there is no direct data supporting the effectiveness of Babble Boot Camp for Down Syndrome. ^{1 2 3 4 5}

What safety data exists for Babble Boot Camp for Down Syndrome?

The provided research does not contain specific safety data for Babble Boot Camp, Modified Babble Boot Camp, or BBC for Down Syndrome. The articles focus on general pediatric drug safety, adverse drug reactions, and regulatory requirements, but do not mention this specific treatment. ^{6 7 8 9 10}

Is the Babble Boot Camp treatment promising for Down Syndrome?

The Babble Boot Camp treatment is promising because it aims to improve communication skills in children with Down Syndrome, which can help them express themselves better and interact more effectively with others. ^{2 11 12 13 14}

Research Team

Beate Peter

Principal Investigator

Arizona State University

Eligibility Criteria

This trial is for infants with Down syndrome, from birth to 12 months old. It aims to test a proactive speech and language intervention called Babble Boot Camp (BBC). The goal is to help these children develop better communication skills early on.

Inclusion Criteria

English is the primary language spoken in my home.

I have Down syndrome.

Exclusion Criteria

Any additional condition that could confound the findings

I am waiting for heart surgery.

I was born before reaching 34 weeks of pregnancy.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a proactive speech and language intervention via telehealth, with weekly individualized sessions or monthly group sessions for 10 months

10 months

Weekly or monthly telehealth sessions

Follow-up

Participants are monitored for speech and language development outcomes and parental well-being

4 weeks

Treatment Details

Interventions

• Babble Boot Camp

Trial Overview The study tests the effectiveness of BBC in improving babble, speech production, and receptive and expressive language in infants with Down syndrome. Participants are split into two groups: one receives weekly sessions; the other has monthly group meetings over ten months.

Participant Groups

Group I: Individual intervention Experimental Treatment 1 Intervention

Individual families will meet weekly with a pediatric speech-language pathologist for approximately 20 minutes. The speech-language pathologist will coach parents in strategies designed to boost speech and language skills, individually tailored to the child's current skillset.

Group II: Group intervention Active Control 1 Intervention

Caregivers will meet in monthly 1-hour group sessions with a pediatric speech-language pathologist. The speech-language pathologist will describe strategies that families can use at home to boost their child's speech and language skills.

Who Is Running the Clinical Trial?

Arizona State University

Lead Sponsor

Trials

311

Recruited

109,000+

American Speech-Language-Hearing Foundation

Collaborator

Trials

2

Recruited

90+

American Speech-Language-Hearing Foundation

Collaborator

Trials

2

Recruited

90+

Findings from Research

A review of 21 studies on early interventions for infants and children with Down's syndrome showed consistent short-term improvements in fine motor skills, social skills, and developmental quotients (DQ/IQ), but mixed results in gross motor, language, and cognitive skills.

Long-term follow-up indicated that the initial gains from these interventions did not persist into the early years of primary schooling, suggesting a need for more effective and tailored intervention strategies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Aggregated early intervention effects for Down's syndrome persons: patterning and longevity of benefits. Gibson, D., Harris, A.[2019]

In a study involving 17 children with Down syndrome over 8 weeks, the use of the computer-based phonics program GraphoGame showed limited overall benefits at the group level, but some individual children demonstrated improved word decoding skills.

The results suggest that while the program may not be universally effective, it can be beneficial for certain children with Down syndrome when their individual learning challenges are taken into account.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intensive computer-based phonics training in the educational setting of children with Down syndrome: An explorative study. Nakeva von Mentzer, C., Kalnak, N., Jennische, M.[2021]

A group of 20 adolescents with Down's syndrome participated in a structured athletics training program twice a week, which began in January 1994, and underwent complete medical examinations before starting.

The program led to significant improvements in strength, speed, and endurance test scores, suggesting that such training can positively influence physical fitness and promote a more athletic body type in these individuals.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Development of physical response after athletics training in adolescents with Down's syndrome. Perán, S., Gil, JL., Ruiz, F., et al.[2019]

A study involving 95 school-aged children with Down syndrome (mean age 10.9 years) highlighted that while they generally received appropriate multidisciplinary care, access to medical specialists and rehabilitation services was often challenging due to long waiting lists and limited availability.

Despite 94% of children under six being integrated into typical classes, by age 15, 75% were placed in medico-social institutions, indicating a shift in educational settings as they age, which may reflect the need for more tailored support as their needs evolve.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Care management in a French cohort with Down syndrome from the AnDDI-Rares/CNSA study. Roux-Levy, PH., Sanlaville, D., De Freminville, B., et al.[2021]

An abbreviated cognitive remediation programme was tested on 18 children aged 6-15 with neurological and attention issues, showing improvements in both parent-reported attention and children's performance on attention tasks.

The results suggest that this shorter version of the established programme could be an effective outpatient treatment for attention problems, but further research with control groups and longer follow-up is needed to confirm its efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A pilot study evaluating an abbreviated version of the cognitive remediation programme for youth with neurocognitive deficits. Luton, LM., Reed-Knight, B., Loiselle, K., et al.[2019]

Approximately 80% of drugs approved by the FDA in the last 30 years have been labeled with disclaimers for use in children, highlighting a significant gap in pediatric drug safety and efficacy studies.

Recent initiatives by the FDA and the National Institute of Child Health and Human Development aim to increase the number of drugs studied and labeled for children, potentially improving access to safe and effective medications for pediatric patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Status of drug approval processes and regulation of medications for children. Kauffman, RE.[2019]

Out of 1787 reported adverse drug reactions (ADRs) over 11 years, 103 (5.85%) were in pediatric patients, with serious ADRs being more common in younger children, particularly newborns and infants.

The most frequent ADRs were nervous system disorders, primarily linked to antineoplastics and immunodulators, highlighting the need for careful monitoring and reporting, especially since medication errors were noted in 7.8% of cases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Spontaneous Reporting of Adverse Drug Reactions in a Pediatric Population in a Tertiary Hospital. López-Valverde, L., Domènech, È., Roguera, M., et al.[2021]

A total of 78,623 adverse event reports were analyzed from the FDA database, with serious outcomes occurring in 40% of children and 43% of adolescents, highlighting the significant risk associated with medications in these age groups.

Central nervous system stimulants and analgesics were the most commonly reported drugs in children, while adolescents reported more cases involving tumor necrosis factor blockers and atypical antipsychotics, indicating different medication safety profiles and adverse event types based on age.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Drugs associated with adverse events in children and adolescents. Lee, WJ., Lee, TA., Pickard, AS., et al.[2014]

The FDA has implemented a Pediatric Study Plan (PSP) requirement for all new drugs to ensure that safety and efficacy data for pediatric patients is systematically collected, addressing a significant gap in drug approval processes.

Incentives for conducting pediatric clinical trials, such as marketing exclusivity and priority review vouchers, encourage pharmaceutical companies to study drugs in various pediatric age groups, ensuring that medications are safe and effective for children.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Food and Drug Administration Requirements for Clinical Studies in Pediatric Patients. Tabor, E.[2020]

A review of 311 individual case safety reports (ICSRs) from the WHO database revealed that musculoskeletal disorders were the most common adverse drug reactions (ADRs) in children and adolescents taking statins, accounting for 27.3% of reported cases.

The study highlighted serious safety concerns, including 42.8% of reports being classified as 'serious' with 11 deaths primarily due to intentional overdoses in adolescents, indicating a need for increased monitoring and reporting of statin ADRs in pediatric populations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adverse drug reactions of statins in children and adolescents: a descriptive analysis from VigiBase, the WHO global database of individual case safety reports. Conte, C., Rousseau, V., Vert, C., et al.[2021]

Treadmill training is feasible for infants at high risk for neuromotor dysfunction, as demonstrated in a case report of a male infant who began training at 5 months corrected age after experiencing a grade III intraventricular hemorrhage.

The treadmill training led to stepping patterns that were similar to those of typically developing infants, suggesting that it may promote more mature stepping characteristics despite the child's high-risk status.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treadmill training for an infant born preterm with a grade III intraventricular hemorrhage. Bodkin, AW., Baxter, RS., Heriza, CB.[2009]

Physical activity offers numerous benefits for children, including enjoyment, social interaction, health maintenance, and a sense of achievement.

For children with Down syndrome, establishing a strong foundation for physical activity early on is crucial for maintaining an active lifestyle throughout school and into adulthood, highlighting the need for increased support from professionals for parents.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Increasing opportunities for physical activity. Buckley, S.[2016]

Wobble board training significantly improved static balance in adolescents with Down syndrome, as indicated by a decrease in the center of pressure (COP) area after training.

The training was effective regardless of whether the participants had their eyes open or closed, suggesting that it enhances balance control mechanisms in these adolescents.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The effects of wobble board training on the eyes open and closed static balance ability of adolescents with down syndrome. Park, TJ.[2021]

The DSFit exercise program, designed for adolescents with Down syndrome, was found to be feasible and engaging, with most participants showing improvements in physical fitness assessments after the intervention.

Feedback from participants and parents indicated high enjoyment and appreciation for the program, highlighting its potential to help develop sustainable exercise habits among adolescents with Down syndrome.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

DSFit: a feasibility pilot study of a group exercise programme for adolescents with Down syndrome. Hojlo, MA., Milliken, AL., Baumer, NT., et al.[2023]

References

1. England pubmed.ncbi.nlm.nih.gov

Aggregated early intervention effects for Down's syndrome persons: patterning and longevity of benefits. [2019]

2. England pubmed.ncbi.nlm.nih.gov

Intensive computer-based phonics training in the educational setting of children with Down syndrome: An explorative study. [2021]

3. Denmark pubmed.ncbi.nlm.nih.gov

Development of physical response after athletics training in adolescents with Down's syndrome. [2019]

4. Netherlands pubmed.ncbi.nlm.nih.gov

Care management in a French cohort with Down syndrome from the AnDDI-Rares/CNSA study. [2021]

5. England pubmed.ncbi.nlm.nih.gov

A pilot study evaluating an abbreviated version of the cognitive remediation programme for youth with neurocognitive deficits. [2019]

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

Status of drug approval processes and regulation of medications for children. [2019]

7. Switzerland pubmed.ncbi.nlm.nih.gov

Spontaneous Reporting of Adverse Drug Reactions in a Pediatric Population in a Tertiary Hospital. [2021]

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

Drugs associated with adverse events in children and adolescents. [2014]

9. Switzerland pubmed.ncbi.nlm.nih.gov

Food and Drug Administration Requirements for Clinical Studies in Pediatric Patients. [2020]

10. England pubmed.ncbi.nlm.nih.gov

Adverse drug reactions of statins in children and adolescents: a descriptive analysis from VigiBase, the WHO global database of individual case safety reports. [2021]

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

Treadmill training for an infant born preterm with a grade III intraventricular hemorrhage. [2009]

12. England pubmed.ncbi.nlm.nih.gov

Increasing opportunities for physical activity. [2016]

13. Japan pubmed.ncbi.nlm.nih.gov

The effects of wobble board training on the eyes open and closed static balance ability of adolescents with down syndrome. [2021]

14. England pubmed.ncbi.nlm.nih.gov

DSFit: a feasibility pilot study of a group exercise programme for adolescents with Down syndrome. [2023]