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Preventing Speech and Language Disorders in Children With Classic Galactosemia (BBC)

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ClinicalTrials.gov Identifier: NCT03838016
Recruitment Status : Recruiting
First Posted : February 12, 2019
Last Update Posted : February 12, 2019
Sponsor:
Collaborator:
Washington State University
Information provided by (Responsible Party):
Beate Peter, Arizona State University

Brief Summary:

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 and the children receive a professional speech/language assessment, to be repeated at ages 36 and 48 months.


Condition or disease Intervention/treatment Phase
Classic Galactosemia Speech Disorders in Children Language Disorders in Children Behavioral: Babble Boot Camp Not Applicable

Detailed Description:

Speech and language assessments and treatments are usually not initiated until deficits can be diagnosed, which occurs at age 2-3 years at the earliest. 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. As with most other children, speech and language assessments 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 whether proactive interventions during the acquisition of prespeech (2 to 12 months) and early communication skills (13 to 24 months) can mitigate the speech and language deficits. If the investigators show that interventions during the first two years of life are more effecting in improving speech and language outcomes in children with CG, compared to traditional care, 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 for communication disorders, e.g., various genetic causes and very low birth weight.

The investigators created an intervention program designed to support communication abilities during the prespeech and early speech and language stages for ages 2 to 24 months. The Babble Boot Camp (BBC) is implemented via parent training by a speech-language pathologist (SLP) with expertise in early childhood using Vidyo, a HIPAA-compliant telepractice software provided for free by the PI's institution, to connect with the families. Vidyo runs on computers, tablets, and smartphones. Parents learn about the typical milestones of prespeech, speech, and language development, potential red flags for delays, and importantly, activities that support typical development for all stages of the program. Following an orientation to the program, the SLP meets with each family once per week for training and consultation on the relevant activities given the child's current speech/language status. Examples of activities are stimulating and reinforcing coos and babble, enriching the child's linguistic environment with joint book reading and pointing out the names of objects , and expanding child utterances to provide slightly more complex model sentences. The key principle underlying all activities is the zone of proximal development, also referred to as scaffolding, where parents provide speech and language models that bridge what the child can already to and what is slightly beyond the child's skill set: the model is in the zone of skills that the child can do with help. One key skill that is targeted throughout the program is imitation. The program brochure includes the rationale, instructions, and examples for each activity, such as (direct quote from the brochure): "Expanding on your child's utterance will provide a model for more complex sentence structures as well as increase her/his vocabulary. An added benefit is to let your child hear words in their correctly produced forms when her/his own productions are showing some incorrect speech sounds. This will build your child's awareness of what the word should sound like and get her/him ready to try the correct form. Throughout your daily routine, listen to your child's utterances and expand upon them slightly. You can add descriptive words or fill in some missing words to make a more complete sentence without overwhelming your child's ability to comprehend your sentence. Examples: Child: "Goggie bye-bye." Parent: "Yeah, that doggie is going bye-bye!" - Child: "Mommy doing?" Parent: "What is Mommy doing? She is taking Sammie outside."


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 225 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Infants with Classic Galactosemia and their parents enroll themselves in the study when the infants are approximately 2 months old. In the current pilot phase, all but one family were assigned to the treatment cohort to observe the effects of the treatment longitudinally. Initial results of outcome measures have been consistent with a beneficial effect. A larger clinical trial with randomization is scheduled to start in April 2019, pending final notification of grant award. In that future model, there will be three arms, 25 infants with classic galactosemia who undergo the Babble Boot Camp treatment plus the close monitoring, 25 infants with classic galactosemia who only undergo the close monitoring, and 25 typically developing infants who also only undergo the close monitoring.
Masking: None (Open Label)
Masking Description: At this early phase, there is no masking. Once the larger clinical begins, there will be masking for the outcomes assessors who analyze the outcome variables of interest.
Primary Purpose: Treatment
Official Title: Babble Boot Camp: Preventing Speech and Language Disorders in Children With Classic Galactosemia
Actual Study Start Date : January 31, 2017
Estimated Primary Completion Date : March 30, 2023
Estimated Study Completion Date : March 30, 2039


Arm Intervention/treatment
Experimental: Treatment cohort
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, 3, and 4 years).
Behavioral: Babble Boot Camp
The Babble Boot Camp is an experimental study to investigate whether earliest and proactive activities and routines can positively influence the speech and language development of children who were diagnosed with classic galactosemia at birth. A speech-language pathologists implements the intervention by teaching parents to foster and expand earliest signals of communication, prespeech activities such as coo and babble, vocabulary growth, sentence complexity, and use of language to communicate. Examples are intentional eye contact, reinforcing babble with rewarding play activities, and repeating a child's rudimentary sentence with slight expansions to scaffold longer sentences.

No Intervention: Control cohort
Currently, there is only one child plus parents in the control cohort. This control family participates only 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, 3, and 4 years).



Primary Outcome Measures :
  1. Speech sound production accuracy [ Time Frame: Through study completion, an average of 3 years 8 months ]
    Standardized testing of speech sounds using a published test of articulation

  2. Expressive language skills [ Time Frame: Through study completion, an average of 3 years 8 months ]
    Standardized testing of expressive language ability, using a published test of child language


Secondary Outcome Measures :
  1. Cognitive development [ Time Frame: Through study completion, an average of 3 years 8 months ]
    Standardized testing of cognitive development, using a published test

  2. Quality of life using the PedsQL questionnaires [ Time Frame: Through study completion, an average of 3 years 8 months ]
    Questionnaire-based assessment of quality of life for the child and parent(s). Captures physical functioning, physical symptoms, emotional functioning, social functioning, and cognitive functioning for children. Captures physical functioning, emotional functioning, social functioning, and work/school function for adults.

  3. Ages and Stages Questionnaires 3 [ Time Frame: Through study completion, an average of 3 years 8 months ]
    Questionnaire-based assessment of child development in the areas of communication, gross motor, fine motor, problem solving, and personal-social development for children.



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Months to 6 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Newborn diagnosis of classic galactosemia
  • Any ethnic or racial background
  • Primary language in the home is English
  • Any geographic region in the US and other countries because the intervention is done online
  • Computer and internet access (we can help if a family wants to participate but doesn't have this access)
  • At least one parent must have at least an 8th grade education to be able to fill out the questionnaires

Exclusion Criteria:

  • Other forms of galactosemia outside of classic galactosemia
  • Medical, sensory, or psychiatric condition that could introduce confounding, e.g., Trisomy 21 or deafness

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03838016


Contacts
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Contact: Beate Peter 4809654737 Beate.Peter@asu.edu
Contact: Hanako Yokoyama 480 727 0425 hanakoyok@hotmail.com

Locations
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United States, Arizona
Arizona State University Recruiting
Tempe, Arizona, United States, 85287
Contact: Beate Peter    480-965-4737    Beate.Peter@asu.edu   
Contact: Hanako Yokoyama    4807270425    hanakoyok@hotmail.com   
Sponsors and Collaborators
Arizona State University
Washington State University

Publications:

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Responsible Party: Beate Peter, Assistant Professor, Arizona State University
ClinicalTrials.gov Identifier: NCT03838016     History of Changes
Other Study ID Numbers: STUDY00004969
First Posted: February 12, 2019    Key Record Dates
Last Update Posted: February 12, 2019
Last Verified: February 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Data will be de-identified. Only data that are reported in publications will be shared.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Time Frame: Data will be shared at the time of publication of results, no end date.
Access Criteria: Qualified researchers will be given access to the de-identified data. These are researchers who provide a methodologically sound proposal

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Galactosemias
Language Disorders
Speech Disorders
Disease
Pathologic Processes
Communication Disorders
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
Metabolic Diseases
8-chloro-cyclic adenosine monophosphate
Antineoplastic Agents
Immunologic Factors
Physiological Effects of Drugs