Tuberous SclerosisJETSNCT03422367
JASPER Early Intervention for Tuberous Sclerosis
A study testing JASPER in people with Tuberous Sclerosis, run by University of California, Los Angeles at 2 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 40 months
- Conditions the study is for
- Tuberous Sclerosis
- What is being tested?
- JASPER
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 64 participants
- Where
- 2 sitesUnited States
- Study dates
- 24 Oct 2017to 15 Jun 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 40 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- A confirmed diagnosis of Tuberous Sclerosis Complex
Who cannot join
- A mental age less than 6 months. A plan for epilepsy surgery during the study participation period.
Trial locations and contacts
2 sites in 1 country.
United States2 sites, 2 recruiting
- Recruiting
Boston Children's Hospital
Boston, Massachusetts, 02215
Jack Keller, 857-218-3010, Jack.Keller@childrens.harvard.edu
- Recruiting
University of California Los Angeles
Los Angeles, California, 90065
Carly Hyde, BS, 310-825-8738, chyde@mednet.ucla.edu
About this study
Official title: Mechanisms of Change With Early Intervention in Tuberous Sclerosis
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.
Read the 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.
- Conditions
- Tuberous Sclerosis
- Being tested
- JASPER
- Keywords
- autism, epilepsy, asd, TSC, UCLA, Los Angeles, JASPER, intervention, Boston, BCH, MGH
- Medical subject headings
- Tuberous Sclerosis, Autistic Disorder, Epilepsy, Child Development Disorders, Pervasive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
JASPERBehavioural
Also called Joint Attention; Symbolic Play and Engagement Regulation
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.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Group | Experimental | The Treatment Group begins the JASPER intervention immediately upon enrollment in the study. Receives: Behavioral: JASPER |
| Delayed Treatment Group | Active comparator | 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. Receives: Behavioral: JASPER |
| Control | No intervention | Participants who are unable to complete JASPER due to age or time/distance commitment can enroll for single-time point participation |
What is being measured
The primary outcome is the main question the study is designed to answer.
ESCS (Early Social Communication Scales) Assessment.
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
Measured: 3 months
ESCS (Early Social Communication Scales) Assessment.
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
Measured: 6 months
Parent Caregiver Interaction assesses the amount of time the parent and child spend in engaged in the same play routine.
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.
Measured: 3 months
Parent Caregiver Interaction assesses the amount of time the parent and child spend in engaged in the same play routine.
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.
Measured: 6 months
Structured Play Assessment (SPA)
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.
Measured: 3 months
SPA
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.
Measured: 6 months
Mullen Scales of Early Learning (MSEL)
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.
Measured: 3 months
Mullen
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.
Measured: 6 months
Social communication
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.
Measured: 3 months
Social communication
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.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2018
- Study start24 Oct 2017
- Primary completion15 Jun 2022 (estimated)
- Study completion15 Jun 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Tuberous SclerosisStudies of the same condition, open and under way.
- More trials run by University of California, Los AngelesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 64 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of California, Los Angeles
- Sponsor type
- University, hospital or other
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Boston Children's Hospital, Massachusetts General Hospital
- Responsible party
- Principal investigator, Shafali Jeste, MD, University of California, Los Angeles
- Investigators
- Shafali Jeste, Principal investigator, University of California, Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03422367
- Sponsor's protocol ID
- IRB #17-000262
- Other IDs
- 1R01HD090138-01A1
- Acronym
- JETS
- Status last verified
- Nov 2020
- First submitted
- 30 Jan 2018
- Last updated
- 6 Nov 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
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.
PubMed 31969108
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mechanisms of Change With Early Intervention in Tuberous Sclerosis
- ClinicalTrials.gov ID
- NCT03422367
- Acronym / sponsor ID
- JETS / IRB #17-000262
- Conditions
- Tuberous Sclerosis
- Keywords
- autism, epilepsy, asd, TSC, UCLA, Los Angeles, JASPER, intervention
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 64 participants (estimated)
- Lead sponsor
- University of California, Los AngelesUniversity, hospital or other
- Collaborators
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Boston Children's Hospital, Massachusetts General Hospital
- Study start
- 24 Oct 2017 (actual)
- Primary completion
- 15 Jun 2022 (estimated)
- Study completion
- 15 Jun 2022 (estimated)
- First posted
- 5 Feb 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Treatment Group | Experimental | Behavioral: JASPERThe Treatment Group begins the JASPER intervention immediately upon enrollment in the study. |
| Delayed Treatment Group | Active comparator | Behavioral: JASPERThe Delayed treatment group receives the same JASPER intervention as the Treatment Group, but after 6 months of receiving services in the community as usual. |
| Control | No intervention | –Participants who are unable to complete JASPER due to age or time/distance commitment can enroll for single-time point participation |
Who may qualify
- Ages
- 6 to 40 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- A confirmed diagnosis of Tuberous Sclerosis Complex
Key exclusion criteria (1 listed)
- A mental age less than 6 months. A plan for epilepsy surgery during the study participation period.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| JASPERAlso: Joint Attention; Symbolic Play and Engagement RegulationJASPER (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. | Behavioural | Delayed Treatment Group, Treatment Group |
Primary outcome
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.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2020.
- Derived from this study: PubMed 31969108Hyde 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.
- Related InfoLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Shafali Jeste principal investigator
University of California, Los Angeles
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing JASPER in people with Tuberous Sclerosis, run by University of California, Los Angeles at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 6 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Carly Hyde (3108258738, chyde@mednet.ucla.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ESCS (Early Social Communication Scales) Assessment. (time frame: 3 months). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03422367, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Nov 2020.