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JASPER Early Intervention for Tuberous Sclerosis (JETS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03422367
Recruitment Status : Recruiting
First Posted : February 5, 2018
Last Update Posted : October 18, 2019
Sponsor:
Collaborators:
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Boston Children's Hospital
Massachusetts General Hospital
Information provided by (Responsible Party):
Shafali Jeste, MD, University of California, Los Angeles

Tracking Information
First Submitted Date  ICMJE January 30, 2018
First Posted Date  ICMJE February 5, 2018
Last Update Posted Date October 18, 2019
Actual Study Start Date  ICMJE October 24, 2017
Estimated Primary Completion Date June 15, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 21, 2018)
  • ESCS (Early Social Communication Scales) Assessment. [ Time Frame: 3 months ]
    Change from baseline in the amount of time spent in a Joint Attention (the coordination of attention between objects and people for the purpose of sharing) state at 3 months; ESCS is an experimental measure reported as frequency for requesting and joint attention in 15-minute intervals
  • ESCS (Early Social Communication Scales) Assessment. [ Time Frame: 6 months ]
    Change from baseline in the amount of time spent in a Joint Attention (the coordination of attention between objects and people for the purpose of sharing) state at 6 months; ESCS is an experimental measure reported as frequency for requesting and joint attention in 15-minute intervals
  • Parent Caregiver Interaction assesses the amount of time the parent and child spend in engaged in the same play routine. [ Time Frame: 3 months ]
    change from baseline in Joint Engagement (shared engagement in a play routine) at 3 months into study participation, according to the parent caregiver interaction assessment.
  • Parent Caregiver Interaction assesses the amount of time the parent and child spend in engaged in the same play routine. [ Time Frame: 6 months ]
    change from baseline in Joint Engagement (shared engagement in a play routine) at 6 months into study participation, according to the parent caregiver interaction assessment.
  • Structured Play Assessment (SPA) [ Time Frame: 3 months ]
    Change from baseline in Symbolic Play (types of play and level of play) at 3 months; this is an experimental measure reported as types of behaviors reflecting flexibility and diversity of the child's play level, based on distinct sets of toys that target specific developmental markers of play.
  • SPA [ Time Frame: 6 months ]
    Change from baseline in Symbolic Play (types of play and level of play) at 6 months; this is an experimental measure reported as types of behaviors reflecting flexibility and diversity of the child's play level, based on distinct sets of toys that target specific developmental markers of play.
  • Mullen Scales of Early Learning (MSEL) [ Time Frame: 3 months ]
    Measures cognitive ability and language development (baseline to 3 months). The test has five scales: gross motor, visual reception, fine motor, receptive language, and expressive language. The test identifies the subject's strengths and weaknesses and assesses early intellectual development.
  • Mullen [ Time Frame: 6 months ]
    Measures cognitive ability and language development (baseline to 6 months). The test has five scales: gross motor, visual reception, fine motor, receptive language, and expressive language. The test identifies the subject's strengths and weaknesses and assesses early intellectual development.
  • Social communication [ Time Frame: 3 months ]
    Change from baseline at 3 months in the amount of Parent use of social communication support strategies Parent use of social communication support strategies as taught by JASPER will be measured by a parent-child interaction assessment.
  • Social communication [ Time Frame: 6 months ]
    Change from baseline at 3 months in the amount of Parent use of social communication support strategies Parent use of social communication support strategies as taught by JASPER will be measured by a parent-child interaction assessment.
Original Primary Outcome Measures  ICMJE
 (submitted: January 30, 2018)
  • ESCS (Early Social Communication Scales) Assessment. [ Time Frame: 3 months ]
    Change from baseline in the amount of time spent in a Joint Attention (the coordination of attention between objects and people for the purpose of sharing) state at 3 months
  • ESCS (Early Social Communication Scales) Assessment. [ Time Frame: 6 months ]
    Change from baseline in the amount of time spent in a Joint Attention (the coordination of attention between objects and people for the purpose of sharing) state at 6 months
  • Parent Caregiver Interaction assesses the amount of time the parent and child spend in engaged in the same play routine. [ Time Frame: 3 months ]
    change from baseline in Joint Engagement (shared engagement in a play routine) at 3 months into study participation, according to the parent caregiver interaction assessment.
  • Parent Caregiver Interaction assesses the amount of time the parent and child spend in engaged in the same play routine. [ Time Frame: 6 months ]
    change from baseline in Joint Engagement (shared engagement in a play routine) at 6 months into study participation, according to the parent caregiver interaction assessment.
  • SPA [ Time Frame: 3 months ]
    Change from baseline in Symbolic Play (types of play and level of play) at 3 months, according to the Structured Play Assessment (SPA) The Structured Play Assessment measures the child's play level, based on distinct sets of toys that target specific developmental markers of play.
  • SPA [ Time Frame: 6 months ]
    Change from baseline in Symbolic Play (types of play and level of play) at 3 months, according to the Structured Play Assessment (SPA) The Structured Play Assessment measures the child's play level, based on distinct sets of toys that target specific developmental markers of play.
  • Mullen [ Time Frame: 3 months ]
    Change from baseline at 3 months in developmental quotient, as determined by the Mullen Scales of Early Learning Cognition will be determined by a Developmental Quotient on a traditional cognitive measure, the Mullen Scales of Early Learning
  • Mullen [ Time Frame: 6 months ]
    Change from baseline at 3 months in developmental quotient, as determined by the Mullen Scales of Early Learning Cognition will be determined by a Developmental Quotient on a traditional cognitive measure, the Mullen Scales of Early Learning
  • Social communication [ Time Frame: 3 months ]
    Change from baseline at 3 months in the amount of Parent use of social communication support strategies Parent use of social communication support strategies as taught by JASPER will be measured by a parent-child interaction assessment.
  • Social communication [ Time Frame: 6 months ]
    Change from baseline at 3 months in the amount of Parent use of social communication support strategies Parent use of social communication support strategies as taught by JASPER will be measured by a parent-child interaction assessment.
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE JASPER Early Intervention for Tuberous Sclerosis
Official Title  ICMJE Mechanisms of Change With Early Intervention in Tuberous Sclerosis
Brief Summary

The investigators are running an intervention study for young children with Tuberous Sclerosis Complex (TSC). The study will include free play-based behavioral intervention administered remotely that may improve social and communication skills in children with TSC. Eligible families will have a child in the age range of 12-36 months, with a diagnosis of TSC. Children with TSC below 12 months may be eligible for an early markers study prior to enrollment in the intervention trial.

The intervention will focus on teaching caregivers skills to improve the social and communication outcomes of their children. The content of the intervention will be individually tailored to the child's developmental level. The intervention involves 4 on-site assessment visits, and 12 weekly intervention sessions, administered in-person and remotely. The intervention focuses on improving social-communication and play skills.

Detailed Description

Tuberous Sclerosis Complex (TSC) is a genetic disorder caused by mutations in either the TSC1 or TSC2 genes. The genetic variation results in the growth of non-malignant tumors throughout the body. Neurodevelopmental disorders, including global developmental delay, intellectual disability, and autism spectrum disorder (ASD) are very common in children with TSC, although developmental outcomes can vary widely. Study investigators, over the past several years, has been studying infants and young children with TSC, and investigators have found that developmental delays can be identified in the first year of life, and that these delays and differences can predict a diagnosis of ASD. Currently, there is no specific treatment for the neurodevelopmental disorders associated with TSC. Based on this information, researchers now want to know if early intervention can help to improve development and prevent ASD in children with TSC.

Investigators are studying the effects of early behavioral intervention on developmental outcomes in infants and toddlers with TSC. The study focuses on social-communication skills, as these are tightly linked to the development of ASD. The overarching goal of this research is to improve outcomes in infants with TSC by conducting rigorous, innovative research in treatment, using both brain and behavioral measures to study the effects of treatment.

Participation requires four on-site assessment visits at UCLA or Boston Children's Hospital, and 12 weekly behavioral intervention sessions, administered in-person and remotely. Because this behavioral intervention is parent-mediated, a parent must be available to attend these sessions. Behavioral assessments generally take up to 4 hours to complete. This study also uses research EEG. This non-invasive assessment generally takes 30-45 minutes to complete. After the participant's first assessment, he/she will be randomly assigned to receive treatment either immediately or in 6 months, with behavioral assessments throughout. Participants will be involved in the study for a duration of 15-21 months, depending on randomization.

There are no anticipated risks from this study, although it is possible that the participant may react negatively to some of the assessment measures or intervention sessions. For example, at the most extreme, the participant may be fearful of an age-appropriate toy and may cry or physically pull back from the toy. If this should occur, that particular toy presentation, assessment or intervention will be stopped. Sometimes the EEG net can feel uncomfortable, especially if children do not like wearing hats or having anything touch their heads. Because of this, the study team will provide families with the demonstration ("practice") net to use with the participant before his/her session. At any time during the session if the participant becomes too upset or agitated, researchers will stop the session. Loss of confidentiality is a risk of participating in this research study. As described below, researchers will take every measure to keep participation in this research study confidential. Participants may benefit from the intervention by receiving detailed information about your child's cognitive, language, and communication skills both before the intervention begins and after the intervention is completed. Participants also may benefit from the intervention itself, which is aimed at improving social communication skills in infants and toddlers at high risk for neurodevelopmental disorders. Families will receive written feedback on the participants's performance on the behavioral assessments administered during assessment time point 4. These assessments are used for research purposes and therefore are not comprehensive clinical evaluations. Some of the assessments administered will measure social communication skills relevant to Autism Spectrum Disorder (ASD), and the feedback letter will report whether the assessment indicated the possible presence of ASD. The results of the research may also contribute to the broader TSC and autism fields by enhancing the knowledge of the effects of early intervention on social communication skills in TSC. Benefits that participating children may derive from the intervention may lead to greater benefits for all children with autism and/or TSC.

Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Tuberous Sclerosis
Intervention  ICMJE Behavioral: JASPER
JASPER (Joint Attention, Symbolic Play, Engagement, and Regulation) is a treatment approach based on a combination of developmental and behavioral principles developed by Dr. Connie Kasari and Dr. Amanda Gulsrud at UCLA. It targets the foundations of social communication (joint attention, imitation, play) and uses naturalistic strategies to increase the rate and complexity of social communication.
Other Name: Joint Attention; Symbolic Play and Engagement Regulation
Study Arms  ICMJE
  • Experimental: Treatment Group
    The Treatment Group begins the JASPER intervention immediately upon enrollment in the study.
    Intervention: Behavioral: JASPER
  • Active Comparator: Delayed Treatment Group
    The Delayed treatment group receives the same JASPER intervention as the Treatment Group, but after 6 months of receiving services in the community as usual.
    Intervention: Behavioral: JASPER
  • No Intervention: Control
    Participants who are unable to complete JASPER due to age or time/distance commitment can enroll for single-time point participation
Publications * Hyde C, Pizzano M, McDonald NM, Nelson CA 3rd, Kasari C, Thiele EA, Jeste SS. A telehealth approach to improving clinical trial access for infants with tuberous sclerosis complex. J Neurodev Disord. 2020 Jan 22;12(1):3. doi: 10.1186/s11689-019-9302-0.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: January 30, 2018)
64
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 15, 2022
Estimated Primary Completion Date June 15, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • A confirmed diagnosis of Tuberous Sclerosis Complex

Exclusion Criteria:

  • A mental age less than 6 months. A plan for epilepsy surgery during the study participation period.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Months to 40 Months   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Carly Hyde 3108258738 chyde@mednet.ucla.edu
Contact: Web: www.JETSstudy.org JETSstudy@gmail.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03422367
Other Study ID Numbers  ICMJE IRB #17-000262
1R01HD090138-01A1 ( U.S. NIH Grant/Contract )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Shafali Jeste, MD, University of California, Los Angeles
Study Sponsor  ICMJE University of California, Los Angeles
Collaborators  ICMJE
  • Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
  • Boston Children's Hospital
  • Massachusetts General Hospital
Investigators  ICMJE
Principal Investigator: Shafali Jeste University of California, Los Angeles
PRS Account University of California, Los Angeles
Verification Date October 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP