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.12345
What safety data exists for Babble Boot Camp for Down Syndrome?
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.
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Treatment Details
Interventions
- Babble Boot Camp (Behavioural Intervention)
Trial OverviewThe 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
2Treatment groups
Experimental Treatment
Active Control
Group I: Individual interventionExperimental Treatment1 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 interventionActive Control1 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.
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Arizona State UniversityTempe, AZ
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Who Is Running the Clinical Trial?
Arizona State UniversityLead Sponsor
American Speech-Language-Hearing FoundationCollaborator
American Speech-Language-Hearing FoundationCollaborator
References
Aggregated early intervention effects for Down's syndrome persons: patterning and longevity of benefits. [2019]Pooled findings from 21 early intervention demonstration studies for Down's syndrome infants and children yield consistency of short-term benefits in the growth of finer motor skills, simple social repertoire and DQ/IQ scores, but conflicting evidence in support or not of benefits in the gross motor, linguistic and cognitive/academic domains. Support for the tenacity of gains, on follow-up to the early years of primary schooling, is disappointing. It is recommended that: (1) intervention programmers view the key working assumptions and ideological positions governing present practices more critically; (2) intervention curricula reflect the unique biological and behavioural properties of the syndrome, taking into account individual differences which are independent of etiological label; and (3) care delivery systems be based more fully on multidisciplinary collaboration, especially between the health sciences and education fields.
Intensive computer-based phonics training in the educational setting of children with Down syndrome: An explorative study. [2021]Children with Down syndrome (DS) using intensive computer-based phonics (GraphoGame, GG) were studied. The children's independence and improvement in phonological processing, letter knowledge, word decoding, and reading strategies were investigated. Seventeen children (5-16 years) with DS participated in a crossover design through 8 weeks (one period), with three test sessions separated by 4 weeks. Children were randomly assigned to GG intervention or regular schooling (RS). All children completed one period and eight children completed two periods. A majority gradually became independent in managing GG. At the group level, very little benefit was found from working with GG. At the individual level, several children with mild to severe intellectual disabilities showed increased decoding of trained words. After one period of GG and RS, an increase in alphabetically decoded words was found. The finding suggests that when individual challenges are considered, computer-based phonics may be beneficial for children with DS in their educational setting.
Development of physical response after athletics training in adolescents with Down's syndrome. [2019]Starting in January 1994, a group of 20 adolescents with Down's syndrome began a competition-oriented athletics training program at a public sports stadium twice a week as part of their education towards integration. They were given complete medical examinations beforehand and followed medically and professionally throughout the program. The results showed a significant improvement in the test scores measuring strength, speed and endurance and a tendency towards an athletic morphotype.
Care management in a French cohort with Down syndrome from the AnDDI-Rares/CNSA study. [2021]Down syndrome (DS) is a genetic neurodevelopmental disorder. In individuals with DS, a multidisciplinary approach to care is required to prevent multiple medical complications. The aim of this study was to describe the rehabilitation, medical care, and educational and social support provided to school-aged French DS patients with varying neuropsychological profiles. A mixed study was conducted. Quantitative data were obtained from a French multicentre study that included patients aged 4-20 years with diverse genetic syndromes. Qualitative data were collected by semi-structured face-to-face interviews and focus groups. Ninety-five DS subjects with a mean age of 10.9 years were included. Sixty-six per cent had a moderate intellectual disability (ID) and 18.9% had a severe ID. Medical supervision was generally multidisciplinary but access to medical specialists was often difficult. In terms of education, 94% of children under the age of six were in typical classes. After the age of 15, 75% were in medico-social institutions. Analysis of multidisciplinary rehabilitation conducted in the public and private sectors revealed failure to access physiotherapy, psychomotor therapy and occupational therapy, but not speech therapy. The main barrier encountered by patients was the difficulty accessing appropriate facilities due to a lack of space and long waiting lists. In conclusion, children and adolescents with DS generally received appropriate care. Though the management of children with DS has been improved considerably, access to health facilities remains inadequate.
A pilot study evaluating an abbreviated version of the cognitive remediation programme for youth with neurocognitive deficits. [2019]To determine the effectiveness of an abbreviated version of an established cognitive remediation programme for children with neurological disorders and attention problems in an outpatient setting.
Status of drug approval processes and regulation of medications for children. [2019]The term therapeutic orphan was coined in 1968 to describe the exclusion of infants and children from approved indications for use of the majority of drugs approved by the US Food and Drug Administration (FDA). Although the 1962 Kefauver-Harris amendments to the Food, Drug, and Cosmetic Act were designed to insure the efficacy and safety of drugs approved for human use, infants and children have largely been excluded from the protection of the law. Approximately 80% of the drugs approved by the FDA during the past 30 years have been approved with a labeling disclaimer for use by children. This high percentage is due largely to the lack of studies in children to document safety and efficacy or a failure to use available data to amend labeling to include pediatric indications. Recently, several initiatives by the FDA and the National Institute of Child Health and Human Development have been implemented that promise to increase the number of drugs studied and labeled for children. These initiatives may introduce a new era of drug development for children in which pediatric investigators, the FDA, the National Institutes of Health, and the pharmaceutical industry join together to bring the same level of pharmacotherapeutic safety and efficacy to children that adult patients enjoy.
Spontaneous Reporting of Adverse Drug Reactions in a Pediatric Population in a Tertiary Hospital. [2021]The pediatric population is a vulnerable group for adverse drug reactions (ADRs), and data on spontaneous reporting of ADRs in the hospital setting are scarce. We conducted a retrospective analysis of ADRs in pediatric patients spontaneously reported by health care professionals to a Pharmacovigilance Program in a tertiary hospital between 2010 and 2020, and we compared characteristics of ADRs between pediatric age subgroups. From 1787 spontaneously reported ADRs in an 11-year period, 103 (5.85%) were pediatric ADRs. The median age of patients with ADRs was 8.4 years (range 1 day-17 years) and 57.3% were male. The most frequent ADRs reported were nervous system disorders (13.6%) and the most frequently involved drugs were antineoplastics and immunodulators (32.4%). A 59.2% of the ADRs were serious and 55.3% were classified as being type B reactions. Medication errors were involved in 7.8% of the ADRs and 11.9% of the suspected drugs were used off-label. Spontaneous reports of ADRs in newborns, infants, and toddlers were more serious and less often described in the product data sheet than in children and adolescents (p < 0.001 and p = 0.004 respectively). Medication errors were more frequent in patients under two years of age. These results should be interpreted with caution due to under-reporting and biases in spontaneous reporting of ADRs.
Drugs associated with adverse events in children and adolescents. [2014]To describe the suspected medications, types of reactions, and outcomes of adverse events (AEs) most commonly reported to the United States Food and Drug Administration (FDA) in children by age group.
Food and Drug Administration Requirements for Clinical Studies in Pediatric Patients. [2020]Many drugs approved by the US Food and Drug Administration (FDA) for use in adults lack adequate data on safety and efficacy in pediatric patients, a potential source of unintended harm to pediatric patients. Through a series of laws, regulations, and guidance documents, the US Congress and FDA have created a program both to encourage and mandate clinical studies in pediatric patients to develop evidence-based dosing, safety, and efficacy information. A "Pediatric Study Plan" (PSP) is required for every new drug. FDA provides incentives for the voluntary conduct of clinical trials in pediatric patients, including opportunities for added marketing exclusivity and for obtaining a "priority review voucher." FDA also mandates that clinical studies for new drugs be conducted in each pediatric age group (newborns, infants, children, and adolescents), except in circumstances where a waiver or a deferral of studies can be justified. Sometimes this mandate can be met by extrapolation from studies in adults, or from patients in one pediatric age group to another, for evidence of efficacy. However, separate studies of safety and dosing are usually required for each pediatric age group. The package insert for each new drug now must address the use in pediatric patients. In addition, the FDA website displays all changes in drug labeling related to pediatric patients (excerpted from the labels for easy access), summaries of all pediatric studies that have led to labeling changes, links to FDA medical reviews of pediatric studies, summaries of all pediatric safety issues presented to the FDA Pediatric Advisory Committee (with links to the meeting materials and transcripts), and details of deferred pediatric studies with their timelines and progress. These measures reflect the increasing attention by FDA and the medical community to the importance of clinical studies in pediatric patients.
Adverse drug reactions of statins in children and adolescents: a descriptive analysis from VigiBase, the WHO global database of individual case safety reports. [2021]In adults, statins safety profile is well known. However, literature data on their adverse drug reactions (ADRs) remain scarce in children in real-life setting. In order to better characterize ADRs related to 'real-life' use of statins in children, we reviewed statin-related ADRs recorded in the World Health Organization (WHO) global database of individual case safety reports (ICSRs), VigiBase. Methods. Individual case safety reports (ICSRs) in children (2-11 years) and adolescents (12-17 years) associated with statins from January 1, 1987, to July 18, 2017, were extracted from VigiBase. Characteristics of ICSRs, type of ADRs according to MedDRA classification (SOC and PT), and ICSR seriousness were described using SAS 9.4. A total of 311 ICSRs were identified for 8 statins with 712 ADRs. Musculoskeletal disorders (n = 85, 27.3%) were the first registered ADRs followed by general disorders (n = 67, 21.5%; mainly asthenia and pain). More than 1 out of 5 ADRs were 'injury, poisoning and procedural complications' (n = 67), mainly accidental or intentional exposures (n = 44, 14.1%), overdoses (n = 14, 4.5%), or off-label use (n = 11, 3.5%). Overall, 133 (42.8%) reports were 'serious', including 11 deaths. Deaths mainly involved adolescents with intentional overdose and completed suicide with other associated drugs in 75% of reports. Our study identified rare but serious safety issues (rhabdomyolysis, myalgia, and hepatocellular injury). These ADRs can impact quality of life or lead to life-threatening complications in children. Our results should be supplemented with other data sources. Spontaneous statin ADR reports in children to pharmacovigilance networks must be promoted.
Treadmill training for an infant born preterm with a grade III intraventricular hemorrhage. [2009]Research has documented the feasibility and benefit of treadmill training in children with cerebral palsy and Down syndrome. The purposes of this case report are: (1) to determine the feasibility of treadmill training in an infant at high risk for neuromotor dysfunction and (2) to describe the child's treadmill stepping patterns following treadmill training.
Increasing opportunities for physical activity. [2016]Being physically active can have a number of benefits - having fun, meeting with friends, keeping healthy and experiencing success. For children with Down syndrome the foundations need to be laid early if they are to keep active in school, teenage and adult years and parents ask for more help in this area from professionals.
The effects of wobble board training on the eyes open and closed static balance ability of adolescents with down syndrome. [2021][Purpose] The aim of the present study was to examine the influence of wobble board training on static balance, with and without vision, of adolescents with Down syndrome (DS). [Subjects] Ten adolescents with DS were recruited for this study. [Methods] Participants performed quiet standing with their eyes open and closed, pre- and post-wobble board training. During quiet standing, the center of pressure (COP) data was recorded using a force plate. To assess the static balance ability of the participants, the 95% confidence ellipse area of COP was calculated. The paired t-test was used to compare the 95% confidence ellipse area of COP between the eyes open and closed conditions, and between pre- and post-training. [Results] Although there was no significant difference in the 95% confidence ellipse area of COP between with and without vision, the 95% confidence ellipse area of COP decreased significantly after wobble board training. [Conclusion] These findings suggest that wobble board training is an effective at improving the static balance ability of adolescents with DS.
DSFit: a feasibility pilot study of a group exercise programme for adolescents with Down syndrome. [2023]While past research has underscored the benefits of physical activity for people with Down syndrome (DS), exercise programming that is customised to and/or accessible for children and adolescents with DS is limited. The objectives of this pilot were to (1) develop and refine an engaging exercise programme for adolescents with DS, called DSFit; (2) assess feasibility over the course of two pilot iterations; and (3) examine participant and parent feedback regarding exercise priorities and the DSFit exercise programme.