Down Syndrome Clinical Trial
— BBC-DSOfficial title:
Babble Boot Camp for Infants With Down Syndrome: Improving Speech and Language Outcomes Via a Proactive, Parent-led Intervention
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.
Status | Recruiting |
Enrollment | 60 |
Est. completion date | February 27, 2025 |
Est. primary completion date | February 27, 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 0 Months to 12 Months |
Eligibility | Inclusion Criteria: - Full trisomy 21 - English is the main language spoken in the home Exclusion Criteria: - Partial or mosaic trisomy 21 - Any additional condition that could confound the findings - Awaiting heart surgery - Born prematurely before 34 weeks gestation |
Country | Name | City | State |
---|---|---|---|
United States | Arizona State University | Tempe | Arizona |
Lead Sponsor | Collaborator |
---|---|
Arizona State University | American Speech-Language-Hearing Foundation |
United States,
Finestack LH, Potter N, VanDam M, Davis J, Bruce L, Scherer N, Eng L, Peter B. Feasibility of a Proactive Parent-Implemented Communication Intervention Delivered via Telepractice for Children With Classic Galactosemia. Am J Speech Lang Pathol. 2022 Nov 16;31(6):2527-2538. doi: 10.1044/2022_AJSLP-22-00107. Epub 2022 Oct 14. — View Citation
Peter B, Bruce L, Finestack L, Dinu V, Wilson M, Klein-Seetharaman J, Lewis CR, Braden BB, Tang YY, Scherer N, VanDam M, Potter N. Precision Medicine as a New Frontier in Speech-Language Pathology: How Applying Insights From Behavior Genomics Can Improve Outcomes in Communication Disorders. Am J Speech Lang Pathol. 2023 Jul 10;32(4):1397-1412. doi: 10.1044/2023_AJSLP-22-00205. Epub 2023 May 5. — View Citation
Peter B, Davis J, Cotter S, Belter A, Williams E, Stumpf M, Bruce L, Eng L, Kim Y, Finestack L, Stoel-Gammon C, Williams D, Scherer N, VanDam M, Potter N. Toward Preventing Speech and Language Disorders of Known Genetic Origin: First Post-Intervention Results of Babble Boot Camp in Children With Classic Galactosemia. Am J Speech Lang Pathol. 2021 Nov 4;30(6):2616-2634. doi: 10.1044/2021_AJSLP-21-00098. Epub 2021 Oct 19. — View Citation
Peter B, Davis J, Finestack L, Stoel-Gammon C, VanDam M, Bruce L, Kim Y, Eng L, Cotter S, Landis E, Beames S, Scherer N, Knerr I, Williams D, Schrock C, Potter N. Translating principles of precision medicine into speech-language pathology: Clinical trial of a proactive speech and language intervention for infants with classic galactosemia. HGG Adv. 2022 May 20;3(3):100119. doi: 10.1016/j.xhgg.2022.100119. eCollection 2022 Jul 14. — View Citation
Peter B, Potter N, Davis J, Donenfeld-Peled I, Finestack L, Stoel-Gammon C, Lien K, Bruce L, Vose C, Eng L, Yokoyama H, Olds D, VanDam M. 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. F1000Res. 2019 Mar 11;8:271. doi: 10.12688/f1000research.18062.5. eCollection 2019. — View Citation
Potter NL, VanDam M, Bruce L, Davis J, Eng L, Finestack L, Heinlen V, Scherer N, Schrock C, Seltzer R, Stoel-Gammon C, Thompson L, Peter B. Virtual Post-Intervention Speech and Language Assessment of Toddler and Preschool Participants in Babble Boot Camp. J Speech Lang Hear Res. 2023 May 26:1-13. doi: 10.1044/2023_JSLHR-22-00687. Online ahead of print. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Linguistic environment | Using day-long audio recordings, the investigators will obtain adult word counts, child utterance counts, and conversational turn rates. | Families will provide a set of three day-long audio recordings at the beginning of the intervention (baseline) and again at the end of the intervention (10 months later), each time within a three-week window. | |
Primary | Child utterance complexity | For children age 6 months and up, the investigators will mine the day-long audio recordings for those 5-minute segments with the highest numbers of child utterances. These utterances will be transcribed into the International Phonetic Alphabet. The utterances will be scored for phonetic complexity based on the consonant and vowel content. For babbled utterances, the investigators will compute the Mean Babbling Level (scores range from 1 to 3), and for meaningful utterances, the investigators will compute the Syllable Structure Level (scores range from 1 to 4). For both of these measures, higher scores reflect greater utterance complexity. | Child utterance complexity will be based on the six day-long audio recordings that will be provided by the families in sets of three each at the beginning (baseline) and end (10 months later) of the intervention. | |
Primary | MacArthur-Bates Communicative Inventories 3: Comprehensive Parent | In this checklist-based questionnaires, caregivers provide information about their child's language skills, for instance how many words the child understands and how many words the child produces. Point scores are converted into percentiles, where higher numbers reflect better outcomes. | Collected at the beginning of the intervention (baseline) and again at the end of the intervention (10 months later) | |
Primary | Vineland Adaptive Behavior Scales 3: Comprehensive Parent/Caregiver Form | Parents complete this questionnaire. Areas included in it are Communication, Daily Living Skills, Socialization, and Motor Skills. Point scores from the questionnaire are converted into standard scores and percentiles, where higher numbers reflect better outcomes. | Collected at the beginning of the intervention (baseline) and again at the end of the intervention (10 months later) | |
Primary | Age at first words | For each child, the age at which she/he started to produce words is recorded. | This milestone may occur at any point through the 10 months of intervention, or not at all. | |
Secondary | Consonant and vowel inventory | From the phonetic transcriptions of the day-long audio recordings, an inventory of consonant and vowel sounds will be made. | Collected at the beginning of the intervention (baseline) and again at the end of the intervention (10 months later) | |
Secondary | Parenting Stress Index 4 Short Form | In three blocks of 12 questions each, parents report on their own personal stress, stress derived from stressful interactions with the child, and stress derived from the child's difficult behaviors. Raw scores are converted into percentiles. Higher percentiles indicate higher levels of perceived stress. | Collected at the beginning of the intervention (baseline) and again at the end of the intervention (10 months later) |
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT04854122 -
Blood Flow Regulation in Individuals With Down Syndrome - Training Study
|
N/A | |
Completed |
NCT04020302 -
Self-Monitoring Shopping Intervention
|
N/A | |
Recruiting |
NCT01950624 -
DS-Connect {TM}: The Down Syndrome Registry
|
||
Completed |
NCT04751136 -
the Effect of Cerebrolysin on Physical and Mental Functions of Down Syndrome
|
Phase 2 | |
Completed |
NCT04767412 -
Inspiratory Muscle Training and Physical Fitness in Children With Down Syndrome Randomized Control Trial
|
N/A | |
Completed |
NCT04536506 -
Bobath and Vojta Therapy for DS
|
N/A | |
Not yet recruiting |
NCT04037579 -
Protocol for a Non-randomized Survey in Down Syndrome People Who Practice Sports. Self and Observers´ Perception.
|
||
Completed |
NCT02882698 -
Performance Analysis in Down Syndrome on Mobile Phone
|
N/A | |
Completed |
NCT01791725 -
A 4-Week Safety Study of Oral ELND005 in Young Adults With Down Syndrome Without Dementia
|
Phase 2 | |
Unknown status |
NCT01975545 -
Fluor Varnish With Silver Nanoparticles for Dental Remineralization in Patients With Trisomy 21
|
Phase 2 | |
Completed |
NCT01808508 -
Obstructive Sleep Apnea and Neurocognitive and Cardiovascular Function in Children With Down Syndrome
|
N/A | |
Terminated |
NCT00754052 -
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 11 To 17
|
Phase 3 | |
Terminated |
NCT00754013 -
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 6 To 10
|
Phase 3 | |
Completed |
NCT01256112 -
Parent Supported Weight Reduction in Down Syndrome
|
N/A | |
Completed |
NCT01313325 -
Hippotherapy to Improve the Balance of Children With Movement Disorders
|
N/A | |
Completed |
NCT01594346 -
Multicenter Vitamin E Trial in Aging Persons With Down Syndrome
|
Phase 3 | |
Completed |
NCT05343468 -
Life Skills Improved in Children With Down Syndrome After Using Assistive Technology
|
N/A | |
Suspended |
NCT05755464 -
Evaluation of Patients With Down Syndrome Compliance to Dental Therapy
|
||
Recruiting |
NCT04022460 -
Using Personal Mobile Technology to Identify Obstructive Sleep Apnea in Children With Down Syndrome (UPLOAD)
|
||
Completed |
NCT04818437 -
Effect of Core Stability Exercises and Balance Training in Postural Control Among Down Syndrome
|
N/A |