Duchenne Muscular DystrophyDMDNCT04529707
Sleep Intervention in Young Boys with Duchenne Muscular Dystrophy
A study testing Transdiagnostic Behavioral Sleep Intervention in people with Duchenne Muscular Dystrophy, run by University of Pittsburgh at 1 site in United States.
Trial summary
This project will systematically plan and evaluate the implementation of the Transdiagnostic Sleep and Circadian Intervention for youth (TranS-CY). As an early stage study, investigators will focus on recruitment strategies to reach the target population and collection of preliminary data on primary and secondary… Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Transdiagnostic Behavioral Sleep Intervention
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 38 participants
- Where
- 1 siteUnited States
- Study dates
- 17 Feb 2021to 31 Dec 2024
- 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 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Parents/caregivers of youth between ages 6 and 17 with a primary diagnosis of Duchenne muscular dystrophy (DMD) who lives at home .
- Access to a smart-phone or computer and internet for the weekly web-based sessions, as well as uploading of the Actigraph data;
- English speaking.
Who cannot join
- Unable to speak or read English
- Their child with DMD has cognitive or behavioral concerns that would limit participation and follow-through of intervention;
- Their child/youth with DMD is currently receiving an intervention for a sleep related disorder
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Pittsburgh
Pittsburgh, Pennsylvania, 15219
About this study
Official title: Sleep Health Dysfunction and the Use of a Transdiagnostic Sleep Intervention in Duchenne Muscular Dystrophy
This project will systematically plan and evaluate the implementation of the Transdiagnostic Sleep and Circadian Intervention for youth (TranS-CY). As an early stage study, investigators will focus on recruitment strategies to reach the target population and collection of preliminary data on primary and secondary effects of the TranS-CY. Weekly remote (video web conferencing) parent training sessions will allow investigators to explore adoption through parent adherence and examine whether the essential elements of the TranS-CY intervention (e.g., motivational interviewing, goal setting, problem solving, sleep routine scheduling, monitoring) can be consistently taught by clinicians and implemented by parents into the home setting.
Read the detailed description
Pre-phase (2 weeks in duration):
Initial Interview: Following informed consent and enrollment, participants will complete a 45-60 minute interview with research investigators using video web conferencing (e.g., Zoom). If the participant agrees, this interview will be recorded. During this interview, investigators will gather descriptive data to characterize the sample including information regarding: 1) the child (age, disease severity for DMD, weight/ height, race/ethnicity, location (rural/suburban/urban), grade in school, medication use); 2) the parent (race/ethnicity, marital status, work status, education level, income); 3) the child's sleep environment (technology/telephone access, darkness, temperature, bed/bedroom sharing); 4) the child's daily activities and routines (current wake-up time \& routines; activities in the home; activities at school and after school); and 5) the child's nighttime routines (activities prior to bedtime; current bedtime routine; technology use prior to bedtime).
ActiGraph: Each parent-child dyad will be mailed a package containing the Actigraph GT9x with wristband, charging device, a manual of operating procedures (MOP) providing all technical and protocol instructions, and phone numbers for research team support. Throughout the 2-week pre-phase data collection period, parents will be required to log and push data from their child's Actigraph to a secure cloud service, CentrePoint, via Wi-Fi each day. This will be accomplished through an app and all data will be de-identified.
Intervention Implementation Phase (10-week duration):
Training Modules: During the intervention phase, weekly video-conference meetings will be held where trained clinicians will guide parents through approximately 60-minute web-based modules using a video-conferencing platform (e.g., Zoom). These sessions will focus on education, dynamic parent training (child-related goal-setting and problem-solving), assessment of sleep health dimensions, and development of a weekly sleep and activity diary. Parents will be provided with a personalized TranS-CY Parent Workbook, which contains information regarding each of the 10 weekly modules. Each weekly session will be agenda driven with the option to revise the agenda based on family needs. Children/youth with DMD will be invited to attend the sessions and provide as much feedback as possible. Data will be collected on parent attendance to assess intervention adoption. Each week, clinicians will request feedback on the previous week's session and provide additional educational support for the previous content should it be required.
Intervention Questionnaires: During the 10-week intervention phase, parents will be asked to complete knowledge checks every 2-3 weeks in order to confirm understanding of the module information (primary outcome). These questionnaires will either be asked verbally during the session or questions will be sent to the parent's email through Qualtrics, a HIPAA compliant survey software platform. Hard copies of the knowledge checks will be sent if the parent prefers.
Sleep and Activity Log: Throughout the intervention phase, parents will be asked to complete a daily sleep and activity log covering their child's day. This log will be sent to their emails using Qualtrics. Each log will take approximately 3-5 minutes to complete.
Post-Intervention Phase (2 weeks):
ActiGraph: At the end of the 10-week intervention, each participant (parent-child dyad) will again be mailed an Actigraph GT9x, all accessories and protocols. Throughout the 2-week post-phase data collection period, parents will again use their personal smart-phone app to log and push data from the Actigraph to CentrePoint, via Wi-Fi each day. Data from the Actigraph GT9x will be provide the secondary outcome of percent sleep efficiency. Investigators will explore change in percent sleep efficiency at pre-post 10 week TranS-CY intervention.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Transdiagnostic Behavioral Sleep Intervention
- Keywords
- Duchenne Muscular Dystrophy, Sleep, Intervention
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Transdiagnostic Behavioral Sleep InterventionBehavioural
The Transdiagnostic Sleep and Circadian Intervention for Youth (TranS-CY) uses a comprehensive sleep health framework along with robust evidence for promoting healthy sleep patterns in youth. The TranS-CY incorporates best practices from numerous efficacious behavioral sleep interventions including 1) cognitive behavioral therapy, 2) interpersonal and social rhythm therapy, and 3) behavioral treatment of circadian rhythm sleep-wake disorders. The "traditional" TranS-CY is clinician-led and provides a robust sleep treatment framework for youth with a wide range of disorders and sleep health concerns. For this study, investigators are using the basic framework of the TranS-CY, but clinicians will train parents to incorporate the intervention into the home for their young child with DMD. Trained clinicians will work with parents to ensure the basic components of the TranS-CY are incorporated into the home-based intervention.
| Study group | Role | What they receive |
|---|---|---|
| Sleep Intervention | Experimental | All participants will engage in a 10-week, parent mediated sleep intervention with weekly education sessions. Receives: Behavioral: Transdiagnostic Behavioral Sleep Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Parent Mastery Questionnaire
100% of parents who are retained throughout the study will attain ≥80% knowledge accuracy at each knowledge check time point and will accurately answer 8 of the 10 questions provided.
Measured: A 10-item questionnaire focused on intervention modules will be delivered every 2-3 weeks during the 10 week intervention. Parents will be required to answer questions that address their knowledge of the information provided in each module.
Secondary outcomes (2)
Child intradaily variability from Actigraphy
Vector magnitude scores from Actigraphy for each 2-week data collection period (pre and post intervention) are analyzed through nParAct software. Analyses provide us with our secondary outcome of intradaily variability. Intradaily variability is the frequency and extent of transitions between periods of rest and activity on an hourly basis over 24 hours. Relative amplitude is how active a child is during a 24 hour period.
Measured: 2 weeks pre-intervention and 2 weeks post-intervention
Child relative amplitude from Actigraph
Vector magnitude scores from Actigraphy for each 2-week data collection period (pre and post intervention) are analyzed through nParAct software. Analyses provide us with our secondary outcome of relative amplitude. Relative amplitude is how active a child is during a 24 hour period.
Measured: 2 weeks pre-intervention and 2 weeks post-intervention.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2020
- Study start17 Feb 2021
- Primary completion31 Oct 2024
- Study completion31 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by University of PittsburghThe 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
- Not applicable
- Intervention model
- Single group
- Model description
- The overall objective of this project is to conduct an early phase single-group repeated measures design project examining the implementation and within-subjects effects of a 10-week parent-mediated TranS-CY program to improve sleep health in young boys with DMD.
- Masking
- None (open label)
- Enrolment
- 38 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Pittsburgh
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Angela Caldwell, University of Pittsburgh
- Investigators
- Angela Caldwell, PhD, Principal investigator, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04529707
- Sponsor's protocol ID
- STUDY20030019
- Acronym
- DMD
- Status last verified
- Feb 2025
- First submitted
- 17 Aug 2020
- Last updated
- 7 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: This is a preliminary study focused on determining if the TranS-CY can be modified and implemented into the home by parents of young boys with DMD. The primary and secondary outcomes are focused on collecting data regarding parent knowledge of the intervention components and change in intradaily variability and relative amplitude (through Actigraphy) of the child with DMD pre and post intervention.
Study at a glance
From the registry record, last updated by the sponsor on 7 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sleep Health Dysfunction and the Use of a Transdiagnostic Sleep Intervention in Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT04529707
- Acronym / sponsor ID
- DMD / STUDY20030019
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy, Sleep, Intervention
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 38 participants (actual)
- Lead sponsor
- University of PittsburghUniversity, hospital or other
- Study start
- 17 Feb 2021 (actual)
- Primary completion
- 31 Oct 2024 (actual)
- Study completion
- 31 Dec 2024 (actual)
- First posted
- 28 Aug 2020 (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.
This project will systematically plan and evaluate the implementation of the Transdiagnostic Sleep and Circadian Intervention for youth (TranS-CY). As an early stage study, investigators will focus on recruitment strategies to reach the target population and collection of preliminary data on primary and secondary effects of the TranS-CY. Weekly remote (video web conferencing) parent training sessions will allow investigators to explore adoption through parent adherence and examine whether the essential elements of the TranS-CY intervention (e.g., motivational interviewing, goal setting, problem solving, sleep routine scheduling, monitoring) can be consistently taught by clinicians and implemented by parents into the home setting.
Detailed description
Pre-phase (2 weeks in duration):
Initial Interview: Following informed consent and enrollment, participants will complete a 45-60 minute interview with research investigators using video web conferencing (e.g., Zoom). If the participant agrees, this interview will be recorded. During this interview, investigators will gather descriptive data to characterize the sample including information regarding: 1) the child (age, disease severity for DMD, weight/ height, race/ethnicity, location (rural/suburban/urban), grade in school, medication use); 2) the parent (race/ethnicity, marital status, work status, education level, income); 3) the child's sleep environment (technology/telephone access, darkness, temperature, bed/bedroom sharing); 4) the child's daily activities and routines (current wake-up time \& routines; activities in the home; activities at school and after school); and 5) the child's nighttime routines (activities prior to bedtime; current bedtime routine; technology use prior to bedtime).
ActiGraph: Each parent-child dyad will be mailed a package containing the Actigraph GT9x with wristband, charging device, a manual of operating procedures (MOP) providing all technical and protocol instructions, and phone numbers for research team support. Throughout the 2-week pre-phase data collection period, parents will be required to log and push data from their child's Actigraph to a secure cloud service, CentrePoint, via Wi-Fi each day. This will be accomplished through an app and all data will be de-identified.
Intervention Implementation Phase (10-week duration):
Training Modules: During the intervention phase, weekly video-conference meetings will be held where trained clinicians will guide parents through approximately 60-minute web-based modules using a video-conferencing platform (e.g., Zoom). These sessions will focus on education, dynamic parent training (child-related goal-setting and problem-solving), assessment of sleep health dimensions, and development of a weekly sleep and activity diary. Parents will be provided with a personalized TranS-CY Parent Workbook, which contains information regarding each of the 10 weekly modules. Each weekly session will be agenda driven with the option to revise the agenda based on family needs. Children/youth with DMD will be invited to attend the sessions and provide as much feedback as possible. Data will be collected on parent attendance to assess intervention adoption. Each week, clinicians will request feedback on the previous week's session and provide additional educational support for the previous content should it be required.
Intervention Questionnaires: During the 10-week intervention phase, parents will be asked to complete knowledge checks every 2-3 weeks in order to confirm understanding of the module information (primary outcome). These questionnaires will either be asked verbally during the session or questions will be sent to the parent's email through Qualtrics, a HIPAA compliant survey software platform. Hard copies of the knowledge checks will be sent if the parent prefers.
Sleep and Activity Log: Throughout the intervention phase, parents will be asked to complete a daily sleep and activity log covering their child's day. This log will be sent to their emails using Qualtrics. Each log will take approximately 3-5 minutes to complete.
Post-Intervention Phase (2 weeks):
ActiGraph: At the end of the 10-week intervention, each participant (parent-child dyad) will again be mailed an Actigraph GT9x, all accessories and protocols. Throughout the 2-week post-phase data collection period, parents will again use their personal smart-phone app to log and push data from the Actigraph to CentrePoint, via Wi-Fi each day. Data from the Actigraph GT9x will be provide the secondary outcome of percent sleep efficiency. Investigators will explore change in percent sleep efficiency at pre-post 10 week TranS-CY intervention.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- The overall objective of this project is to conduct an early phase single-group repeated measures design project examining the implementation and within-subjects effects of a 10-week parent-mediated TranS-CY program to improve sleep health in young boys with DMD.
| Arm | Type | Receives |
|---|---|---|
| Sleep Intervention | Experimental | Behavioral: Transdiagnostic Behavioral Sleep InterventionAll participants will engage in a 10-week, parent mediated sleep intervention with weekly education sessions. |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Parents/caregivers of youth between ages 6 and 17 with a primary diagnosis of Duchenne muscular dystrophy (DMD) who lives at home .
- Access to a smart-phone or computer and internet for the weekly web-based sessions, as well as uploading of the Actigraph data;
- English speaking.
Key exclusion criteria (3 listed)
- Unable to speak or read English
- Their child with DMD has cognitive or behavioral concerns that would limit participation and follow-through of intervention;
- Their child/youth with DMD is currently receiving an intervention for a sleep related disorder
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 |
|---|---|---|
| Transdiagnostic Behavioral Sleep InterventionThe Transdiagnostic Sleep and Circadian Intervention for Youth (TranS-CY) uses a comprehensive sleep health framework along with robust evidence for promoting healthy sleep patterns in youth. The TranS-CY incorporates best practices from numerous efficacious behavioral sleep interventions including 1) cognitive behavioral therapy, 2) interpersonal and social rhythm therapy, and 3) behavioral treatment of circadian rhythm sleep-wake disorders. The "traditional" TranS-CY is clinician-led and provides a robust sleep treatment framework for youth with a wide range of disorders and sleep health concerns. For this study, investigators are using the basic framework of the TranS-CY, but clinicians will train parents to incorporate the intervention into the home for their young child with DMD. Trained clinicians will work with parents to ensure the basic components of the TranS-CY are incorporated into the home-based intervention. | Behavioural | Sleep Intervention |
Primary outcome
Parent Mastery Questionnaire
Time frame: A 10-item questionnaire focused on intervention modules will be delivered every 2-3 weeks during the 10 week intervention. Parents will be required to answer questions that address their knowledge of the information provided in each module.
100% of parents who are retained throughout the study will attain ≥80% knowledge accuracy at each knowledge check time point and will accurately answer 8 of the 10 questions provided.
2 secondary outcomes
Child intradaily variability from Actigraphy
Time frame: 2 weeks pre-intervention and 2 weeks post-intervention
Child relative amplitude from Actigraph
Time frame: 2 weeks pre-intervention and 2 weeks post-intervention.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Mar 2025.
- Individual participant dataSharing planned: Yes. This is a preliminary study focused on determining if the TranS-CY can be modified and implemented into the home by parents of young boys with DMD. The primary and secondary outcomes are focused on collecting data regarding parent knowledge of the intervention components and change in intradaily variability and relative amplitude (through Actigraphy) of the child with DMD pre and post intervention.
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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.
Angela Caldwell, PhD principal investigator
University of Pittsburgh
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Transdiagnostic Behavioral Sleep Intervention in people with Duchenne Muscular Dystrophy, run by University of Pittsburgh at 1 site 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 3 inclusion and 3 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: finished, as of the sponsor's last update on 7 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Parent Mastery Questionnaire (time frame: A 10-item questionnaire focused on intervention modules will be delivered every 2-3 weeks during the 10 week intervention. Parents will be required to answer questions that address their knowledge of the information provided in each module.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 7 Mar 2025.