<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT03838016</url>
  </required_header>
  <id_info>
    <org_study_id>STUDY00004969</org_study_id>
    <nct_id>NCT03838016</nct_id>
  </id_info>
  <brief_title>Preventing Speech and Language Disorders in Children With Classic Galactosemia</brief_title>
  <official_title>Babble Boot Camp: Preventing Speech and Language Disorders in Children With Classic Galactosemia</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Arizona State University</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Washington State University</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Arizona State University</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      A critical knowledge gap is whether proactive intervention can improve speech and language&#xD;
      outcomes in infants at known risk for communication disorders. Speech and language&#xD;
      assessments and treatments are usually not initiated until deficits can be diagnosed, no&#xD;
      earlier than age 2-3 years. Preventive services are not available. Children with classic&#xD;
      galactosemia (CG) hold the keys towards investigating whether proactive services are more&#xD;
      effective than conventional management. CG is a recessively inherited inborn error of&#xD;
      metabolism characterized by defective conversion of galactose. Despite early detection and&#xD;
      strict adherence to lactose-restricted diets, children with CG are at very high risk not only&#xD;
      for motor and learning disabilities but also for severe speech sound disorder and language&#xD;
      impairment. Delays are evident from earliest signals of communication and persist into&#xD;
      adulthood in many cases but speech/language assessment and treatment are usually not&#xD;
      initiated until deficits manifest. However, because CG is diagnosed via newborn screening,&#xD;
      the known genotype-phenotype association can be leveraged to investigate the efficacy of&#xD;
      proactive interventions during the acquisition of prespeech (2 to 12 months) and early&#xD;
      communication skills (13 to 24 months). If this proactive intervention is more effective than&#xD;
      standard care regarding speech and language outcomes in children with CG, this will change&#xD;
      their clinical management from deficit-based to proactive services. It will also motivate&#xD;
      investigating this approach in infants with other types of known risk factors, e.g., various&#xD;
      genetic causes and very low birth weight.&#xD;
&#xD;
      The Babble Boot Camp is a program for children with CG, ages 2 to 24 months. The intervention&#xD;
      is implemented by a pediatric speech-language pathologist (SLP) via parent training.&#xD;
      Activities and routines are designed to foster earliest signals of communication, increase&#xD;
      coo and babble behaviors, support the emergence of first words and word combinations, and&#xD;
      expand syntactic complexity. The SLP meets with parents online every week for 10 to 15&#xD;
      minutes to provide instruction, feedback, and guidance. Close monitoring of progress is&#xD;
      achieved via regularly administered questionnaires, a monthly day-long audio recording, and&#xD;
      the SLPs weekly progress notes. At age 24 months, the active phase of the Babble Boot Camp&#xD;
      ends. The children receive a professional speech/language assessment at ages 2 1/2, 3 1/2,&#xD;
      and 4 1/2 years.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Speech and language assessments and treatments are usually not initiated until deficits can&#xD;
      be diagnosed, which occurs at age 2-3 years at the earliest. Preventive services are not&#xD;
      available. Children with classic galactosemia (CG) hold the keys towards investigating&#xD;
      whether proactive services are more effective than conventional management. CG is a&#xD;
      recessively inherited inborn error of metabolism characterized by defective conversion of&#xD;
      galactose. Despite early detection and strict adherence to lactose-restricted diets, children&#xD;
      with CG are at very high risk not only for motor and learning disabilities but also for&#xD;
      severe speech sound disorder and language impairment. Delays are evident from earliest&#xD;
      signals of communication and persist into adulthood in many cases. As with most other&#xD;
      children, speech and language assessments and treatment are usually not initiated until&#xD;
      deficits manifest. However, because CG is diagnosed via newborn screening, the known&#xD;
      genotype-phenotype association can be leveraged to investigate whether proactive&#xD;
      interventions during the acquisition of prespeech (2 to 12 months) and early communication&#xD;
      skills (13 to 24 months) can mitigate the speech and language deficits. If the investigators&#xD;
      show that interventions during the first two years of life are more effecting in improving&#xD;
      speech and language outcomes in children with CG, compared to traditional care, this will&#xD;
      change their clinical management from deficit-based to proactive services. It will also&#xD;
      motivate investigating this approach in infants with other types of known risk factors for&#xD;
      communication disorders, e.g., various genetic causes and very low birth weight.&#xD;
&#xD;
      The investigators created an intervention program designed to support communication abilities&#xD;
      during the prespeech and early speech and language stages for ages 2 to 24 months. The Babble&#xD;
      Boot Camp (BBC) is implemented via parent training by a speech-language pathologist (SLP)&#xD;
      with expertise in early childhood using Zoom, a HIPAA-compliant telepractice software&#xD;
      provided for free by the PI's institution, to connect with the families. Zoom runs on&#xD;
      computers, tablets, and smartphones. Parents learn about the typical milestones of prespeech,&#xD;
      speech, and language development, potential red flags for delays, and importantly, activities&#xD;
      that support typical development for all stages of the program. Following an orientation to&#xD;
      the program, the SLP meets with each family once per week for training and consultation the&#xD;
      relevant activities given the child's current speech/language status. Examples of activities&#xD;
      are stimulating and reinforcing coos and babble, enriching the child's linguistic environment&#xD;
      with joint book reading and pointing out the names of objects, and expanding child utterances&#xD;
      to provide slightly more complex model sentences. The key principle underlying all activities&#xD;
      is the zone of proximal development, also referred to as scaffolding, where parents provide&#xD;
      speech and language models that bridge what the child can already to and what is slightly&#xD;
      beyond the child's skill set: the model is in the zone of skills that the child can do with&#xD;
      help. One key skill that is targeted throughout the program is imitation. The program&#xD;
      brochure includes the rationale, instructions, and examples for each activity, such as&#xD;
      (direct quote from the brochure): &quot;Expanding on your child's utterance will provide a model&#xD;
      for more complex sentence structures as well as increase her/his vocabulary. An added benefit&#xD;
      is to let your child hear words in their correctly produced forms when her/his own&#xD;
      productions are showing some incorrect speech sounds. This will build your child's awareness&#xD;
      of what the word should sound like and get her/him ready to try the correct form. Throughout&#xD;
      your daily routine, listen to your child's utterances and expand upon them slightly. You can&#xD;
      add descriptive words or fill in some missing words to make a more complete sentence without&#xD;
      overwhelming your child's ability to comprehend your sentence.&#xD;
&#xD;
      Examples: Child: &quot;Goggie bye-bye.&quot; Parent: &quot;Yeah, that doggie is going bye-bye!&quot; - Child:&#xD;
&#xD;
      &quot;Mommy doing?&quot; Parent: &quot;What is Mommy doing? She is taking Sammie outside.&quot;&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Enrolling by invitation</overall_status>
  <start_date type="Actual">May 1, 2019</start_date>
  <completion_date type="Anticipated">August 1, 2025</completion_date>
  <primary_completion_date type="Anticipated">August 1, 2024</primary_completion_date>
  <phase>N/A</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <intervention_model_description>The investigators created an intervention program, Babble Boot Camp (BBC), designed to support communication skills during the prespeech and early speech and language stages. One cohort with classic galactosemia (CG) enters this treatment at age 2-5 months and is monitored closely using audio and video recordings and questionnaires. A second cohort with CG enters close monitoring at age 2-5 months but starts treatment at age 15 months. A typical cohort enters close monitoring at age 2-5 months. Treatment and close monitoring ends at age 24 months. Follow-up testing of speech and language skills and questionnaire data about child development and parent well-being is collected once a year at ages 2 ½, 3 ½, and 4 ½ years. A cohort with CG, age 6 months to 4 ½ years, too old for any treatment and close monitoring, provides the same data as the other cohorts at ages 2 ½, 3 ½, and 4 ½ years. Results will show whether the BBC is effective and, if yes, which start age is best.</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Single (Outcomes Assessor)</masking>
    <masking_description>Team members who conduct assessments or analyze data do not have access to a child's group assignment.</masking_description>
  </study_design_info>
  <primary_outcome>
    <measure>Speech sound production accuracy</measure>
    <time_frame>Through study completion, an average of 4 years 2 months</time_frame>
    <description>Standardized testing of speech sounds using a published test of articulation</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Expressive language skills</measure>
    <time_frame>Through study completion, an average of 4 years 2 months</time_frame>
    <description>Standardized testing of expressive language ability, using a published test of child language</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Cognitive development</measure>
    <time_frame>Through study completion, an average of 4 years 2 months</time_frame>
    <description>Standardized testing of cognitive development, using a published test</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Quality of life using the PedsQL questionnaires</measure>
    <time_frame>Through study completion, an average of 4 years 2 months</time_frame>
    <description>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.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Child health and development using the Ages and Stages Questionnaires 3</measure>
    <time_frame>Through study completion, an average of 4 years 2 months</time_frame>
    <description>Questionnaire-based assessment of child development in the areas of communication, gross motor, fine motor, problem solving, and personal-social development for children.</description>
  </secondary_outcome>
  <number_of_arms>4</number_of_arms>
  <enrollment type="Anticipated">309</enrollment>
  <condition>Classic Galactosemia</condition>
  <condition>Speech Disorders in Children</condition>
  <condition>Language Disorders in Children</condition>
  <arm_group>
    <arm_group_label>Treatment cohort with classic galactosemia</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>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 1/2, 3 1/2, and 4 1/2 years).</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Treatment cohort with classic galactosemia, delayed start</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>The children in the control cohort enter the study when they are younger than 5 months old and participate in the close monitoring until they are 24 months old. They start getting the same treatment type and intensity as the treatment cohort but at a delayed age, when they turn 15 months.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Older control cohort with classic galactosemia</arm_group_label>
    <arm_group_type>No Intervention</arm_group_type>
    <description>The children in the older control cohort are 6 months to 4 1/2 years old and provide standardized test results in the area of speech and language development at child ages 2 1/2, 3 1/2, and 4 1/2 years. They receive no treatment and no close monitoring. These families provide questionnaire information every three months until child age 24 months.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Typical controls</arm_group_label>
    <arm_group_type>No Intervention</arm_group_type>
    <description>These children are free of any medical or developmental diagnosis. They enter the study at ages 2 to 5 months and provide close monitoring data until they are 24 months old, then they receive standardized speech and language testing at ages 2 1/2, 3 1/2, and 4 1/2 years, just like the treatment cohort, but the typical controls receive no treatment under this study.</description>
  </arm_group>
  <intervention>
    <intervention_type>Behavioral</intervention_type>
    <intervention_name>Babble Boot Camp</intervention_name>
    <description>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.</description>
    <arm_group_label>Treatment cohort with classic galactosemia</arm_group_label>
    <arm_group_label>Treatment cohort with classic galactosemia, delayed start</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Newborn diagnosis of classic galactosemia&#xD;
&#xD;
          -  Any ethnic or racial background&#xD;
&#xD;
          -  Primary language in the home is English&#xD;
&#xD;
          -  Any geographic region in the US and other countries because the intervention is done&#xD;
             online&#xD;
&#xD;
          -  Computer and internet access (we can help if a family wants to participate but doesn't&#xD;
             have this access)&#xD;
&#xD;
          -  At least one parent must have at least an 8th grade education to be able to fill out&#xD;
             the questionnaires&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Other forms of galactosemia outside of classic galactosemia&#xD;
&#xD;
          -  Medical, sensory, or psychiatric condition that could introduce confounding, e.g.,&#xD;
             Trisomy 21 or deafness&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>2 Months</minimum_age>
    <maximum_age>54 Months</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Arizona State University</name>
      <address>
        <city>Tempe</city>
        <state>Arizona</state>
        <zip>85287</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
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    <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.</citation>
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  </results_reference>
  <results_reference>
    <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.</citation>
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  </results_reference>
  <results_reference>
    <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.</citation>
    <PMID>37235746</PMID>
  </results_reference>
  <verification_date>July 2023</verification_date>
  <study_first_submitted>February 7, 2019</study_first_submitted>
  <study_first_submitted_qc>February 8, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">February 12, 2019</study_first_posted>
  <last_update_submitted>July 21, 2023</last_update_submitted>
  <last_update_submitted_qc>July 21, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">July 24, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Principal Investigator</responsible_party_type>
    <investigator_affiliation>Arizona State University</investigator_affiliation>
    <investigator_full_name>Beate Peter</investigator_full_name>
    <investigator_title>Associate Professor</investigator_title>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Language Disorders</mesh_term>
    <mesh_term>Speech Disorders</mesh_term>
    <mesh_term>Galactosemias</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>Data will be de-identified. Only data that are reported in publications will be shared.</ipd_description>
    <ipd_info_type>Study Protocol</ipd_info_type>
    <ipd_info_type>Statistical Analysis Plan (SAP)</ipd_info_type>
    <ipd_info_type>Informed Consent Form (ICF)</ipd_info_type>
    <ipd_time_frame>Data will be shared at the time of publication of results, no end date.</ipd_time_frame>
    <ipd_access_criteria>Qualified researchers will be given access to the de-identified data. These are researchers who provide a methodologically sound proposal</ipd_access_criteria>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT03838016
org study id: STUDY00004969
nct id: NCT03838016
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
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.
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 Zoom, a HIPAA-compliant telepractice software
      provided for free by the PI's institution, to connect with the families. Zoom 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 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."
allocation: Randomized
intervention model: Parallel Assignment
intervention model description: The investigators created an intervention program, Babble Boot Camp (BBC), designed to support communication skills during the prespeech and early speech and language stages. One cohort with classic galactosemia (CG) enters this treatment at age 2-5 months and is monitored closely using audio and video recordings and questionnaires. A second cohort with CG enters close monitoring at age 2-5 months but starts treatment at age 15 months. A typical cohort enters close monitoring at age 2-5 months. Treatment and close monitoring ends at age 24 months. Follow-up testing of speech and language skills and questionnaire data about child development and parent well-being is collected once a year at ages 2 ½, 3 ½, and 4 ½ years. A cohort with CG, age 6 months to 4 ½ years, too old for any treatment and close monitoring, provides the same data as the other cohorts at ages 2 ½, 3 ½, and 4 ½ years. Results will show whether the BBC is effective and, if yes, which start age is best.
primary purpose: Treatment
masking: Single (Outcomes Assessor)
masking description: Team members who conduct assessments or analyze data do not have access to a child's group assignment.
measure: Speech sound production accuracy
time frame: Through study completion, an average of 4 years 2 months
description: Standardized testing of speech sounds using a published test of articulation
measure: Expressive language skills
time frame: Through study completion, an average of 4 years 2 months
description: Standardized testing of expressive language ability, using a published test of child language
measure: Cognitive development
time frame: Through study completion, an average of 4 years 2 months
description: Standardized testing of cognitive development, using a published test
measure: Quality of life using the PedsQL questionnaires
time frame: Through study completion, an average of 4 years 2 months
description: 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.
measure: Child health and development using the Ages and Stages Questionnaires 3
time frame: Through study completion, an average of 4 years 2 months
description: Questionnaire-based assessment of child development in the areas of communication, gross motor, fine motor, problem solving, and personal-social development for children.
arm group label: Treatment cohort with classic galactosemia
arm group type: Experimental
description: 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 1/2, 3 1/2, and 4 1/2 years).
arm group label: Treatment cohort with classic galactosemia, delayed start
arm group type: Experimental
description: The children in the control cohort enter the study when they are younger than 5 months old and participate in the close monitoring until they are 24 months old. They start getting the same treatment type and intensity as the treatment cohort but at a delayed age, when they turn 15 months.
arm group label: Older control cohort with classic galactosemia
arm group type: No Intervention
description: The children in the older control cohort are 6 months to 4 1/2 years old and provide standardized test results in the area of speech and language development at child ages 2 1/2, 3 1/2, and 4 1/2 years. They receive no treatment and no close monitoring. These families provide questionnaire information every three months until child age 24 months.
arm group label: Typical controls
arm group type: No Intervention
description: These children are free of any medical or developmental diagnosis. They enter the study at ages 2 to 5 months and provide close monitoring data until they are 24 months old, then they receive standardized speech and language testing at ages 2 1/2, 3 1/2, and 4 1/2 years, just like the treatment cohort, but the typical controls receive no treatment under this study.
intervention type: Behavioral
intervention name: Babble Boot Camp
description: 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.
arm group label: Treatment cohort with classic galactosemia
arm group label: Treatment cohort with classic galactosemia, delayed start
criteria: 
gender: All
minimum age: 2 Months
maximum age: 54 Months
healthy volunteers: No
facility: 
country: United States
citation: Demirbas D, Coelho AI, Rubio-Gozalbo ME, Berry GT. Hereditary galactosemia. Metabolism. 2018 Jun;83:188-196. doi: 10.1016/j.metabol.2018.01.025. Epub 2018 Jan 31.
PMID: 29409891
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PMID: 15506833
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PMID: 37146603
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.
PMID: 37235746
responsible party type: Principal Investigator
investigator affiliation: Arizona State University
investigator full name: Beate Peter
investigator title: Associate Professor
mesh term: Language Disorders
mesh term: Speech Disorders
mesh term: Galactosemias
sharing ipd: Yes
ipd description: Data will be de-identified. Only data that are reported in publications will be shared.
ipd info type: Study Protocol
ipd info type: Statistical Analysis Plan (SAP)
ipd info type: Informed Consent Form (ICF)
ipd time frame: Data will be shared at the time of publication of results, no end date.
ipd access criteria: Qualified researchers will be given access to the de-identified data. These are researchers who provide a methodologically sound proposal