Sleep Intervention for Duchenne Muscular Dystrophy

(DMD Trial)

Overseen By Roxanna M Bendixen, PhD

Age: < 65

Sex: Any

Trial Phase: Academic

Sponsor: University of Pittsburgh

Disqualifiers: Cognitive, Behavioral concerns, Sleep intervention

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 11

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new sleep intervention designed to help children and teens with Duchenne muscular dystrophy (DMD) improve their sleep. The study focuses on teaching parents how to implement a sleep routine at home through weekly online sessions. Researchers aim to determine if this approach, called the Transdiagnostic Behavioral Sleep Intervention, improves sleep patterns and other related issues. Families with children aged 6-17 who have DMD and aren't currently receiving sleep treatments are suitable for this trial. Participants need access to a smartphone or computer with internet for the sessions. As an unphased trial, this study offers families the chance to contribute to innovative research that could enhance sleep for children with DMD.

Do I have to stop my current medications for this trial?

The trial protocol does not specify whether you need to stop taking your current medications. However, if your child is currently receiving an intervention for a sleep-related disorder, they cannot participate in the trial.

What prior data suggests that the Transdiagnostic Behavioral Sleep Intervention is safe for youth with Duchenne Muscular Dystrophy?

Research has shown that the Transdiagnostic Sleep and Circadian Intervention (TranS-C) is generally safe. Studies have found that TranS-C does not cause more negative side effects than basic sleep information, indicating that participants usually tolerate it well. In one study, young people using TranS-C reported better sleep without any significant negative effects.

The program primarily involves parents leading training sessions over video calls. These sessions focus on simple methods like setting sleep routines and solving sleep-related problems. Since it emphasizes behavior rather than medication, it typically carries fewer risks. Although the study did not specifically address safety, the absence of reported negative effects is encouraging for prospective participants. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Unlike standard treatments for Duchenne Muscular Dystrophy that focus primarily on managing physical symptoms, the Transdiagnostic Behavioral Sleep Intervention targets sleep issues that often accompany the condition. Researchers are excited about this approach because it involves a 10-week, parent-mediated program with weekly educational sessions, which is a novel method for addressing sleep disturbances holistically. By engaging parents directly in the intervention, it also empowers families to play an active role in improving their child's sleep, potentially leading to better overall quality of life.

What evidence suggests that the Transdiagnostic Behavioral Sleep Intervention is effective for Duchenne Muscular Dystrophy?

Research has shown that the Transdiagnostic Sleep and Circadian Intervention (TranS-C) can improve sleep and body clock rhythms in young people. Studies have found that it significantly enhances sleep habits and daily life for teenagers with the eveningness chronotype over a year. Another study found that TranS-C improved sleep quality and body clock function over six months. In this trial, participants will engage in a 10-week, parent-mediated sleep intervention with weekly education sessions. These findings suggest that this intervention could help manage sleep problems in young people with conditions like Duchenne Muscular Dystrophy. ^{1 2 3 4 6}

Who Is on the Research Team?

Angela Caldwell, PhD

Principal Investigator

University of Pittsburgh

Are You a Good Fit for This Trial?

This trial is for English-speaking parents or caregivers of boys aged 6-17 with Duchenne Muscular Dystrophy (DMD) who live at home and have access to a smartphone or computer with internet. It's not suitable for those whose children have cognitive/behavioral issues that would limit participation, or are currently receiving sleep disorder treatments.

Inclusion Criteria

I am a parent/caregiver of a child aged 6-17 with Duchenne muscular dystrophy living at home.

I have a smartphone or computer with internet for weekly sessions and data uploads.

English speaking

Exclusion Criteria

My child with DMD is being treated for a sleep disorder.

Unable to speak or read English

Your child with DMD has thinking or behavior problems that would make it hard for them to take part in the study or stick to the treatment plan.

Timeline for a Trial Participant

Pre-phase

Initial interview and ActiGraph data collection to characterize the sample and gather baseline data

2 weeks

1 remote interview (video web conferencing)

Intervention Implementation

Weekly video-conference meetings for parent training and education on sleep health, with data collection on parent attendance and feedback

10 weeks

10 remote sessions (video web conferencing)

Post-Intervention

ActiGraph data collection to assess changes in sleep efficiency after the intervention

2 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Transdiagnostic Behavioral Sleep Intervention

Trial Overview The study tests the Transdiagnostic Sleep and Circadian Intervention (TranS-CY), delivered through weekly remote parent training sessions via video web conferencing. The goal is to see if parents can learn and apply strategies like motivational interviewing, goal setting, problem solving, and sleep routine scheduling at home.

How Is the Trial Designed?

Group I: Sleep Intervention Experimental Treatment 1 Intervention

All participants will engage in a 10-week, parent mediated sleep intervention with weekly education sessions.

Who Is Running the Clinical Trial?

University of Pittsburgh

Lead Sponsor

Trials

1,820

Recruited

16,360,000+

Published Research Related to This Trial

The TranS-C intervention significantly improved sleep and circadian functioning in adolescents with an eveningness chronotype over a 12-month period compared to psychoeducation, with a notable reduction in eveningness.

Text messaging reminders enhanced the effectiveness of the TranS-C intervention, helping to maintain treatment benefits throughout the follow-up period.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Transdiagnostic Sleep and Circadian Intervention for Adolescents Plus Text Messaging: Randomized Controlled Trial 12-month Follow-up. Dolsen, EA., Dong, L., Harvey, AG.[2023]

The TranS-C cognitive behavioral sleep intervention significantly reduced the severity of Major Depressive Disorder (MDD) in adolescents with evening circadian preference, showing lasting effects up to 12 months after treatment.

Improved alignment between circadian biology and sleep-wake behavior was identified as a key mechanism for the reduction in depression symptoms, suggesting that addressing sleep patterns can be crucial in treating depression in adolescents.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Report from a randomized control trial: improved alignment between circadian biology and sleep-wake behavior as a mechanism of depression symptom improvement in evening-type adolescents with depressive symptoms. Asarnow, LD., Soehner, A., Dolsen, E., et al.[2023]

In a study of nine non-ambulatory Duchenne muscular dystrophy patients aged 10 to 20, nocturnal sleep was found to negatively impact oxygen levels, particularly during REM sleep, despite normal daytime blood gas levels.

Over a 2-year period, the oxygen imbalance during sleep worsened significantly, correlating with the severity of restrictive thoracic syndrome experienced while awake, indicating a need for monitoring sleep-related respiratory issues in these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Nocturnal sleep and oxygen balance in Duchenne muscular dystrophy. A clinical and polygraphic 2-year follow-up study. Manni, R., Zucca, C., Galimberti, CA., et al.[2019]

Boys with Duchenne muscular dystrophy (n=19) experience significantly worse sleep quality and quantity compared to unaffected peers (n=17), as indicated by both parent-reported questionnaires and objective actigraphy measurements over a 30-day period.

The study found that boys with Duchenne muscular dystrophy had lower sleep efficiency, more frequent awakenings, and longer durations of awakenings, highlighting the need for increased attention to sleep health in this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Uncovering sleep in young males with Duchenne muscular dystrophy. Hartman, AG., Terhorst, L., Little, N., et al.[2020]

The TranS-C intervention led to significant improvements in total sleep time on weeknights and weekends for youth aged 10-18 with evening circadian preference, compared to those receiving psychoeducation.

Despite these improvements in sleep duration, the changes did not reach statistical significance when compared to the psychoeducation group, indicating that while TranS-C shows promise, further research is needed to confirm its efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The effect of the transdiagnostic sleep and circadian intervention (TranS-C) on actigraphic estimates of sleep and rest-activity rhythms in adolescents with an evening circadian preference. Gasperetti, CE., Dong, L., Harvey, AG.[2022]

In a study of 63 boys with Duchenne Muscular Dystrophy (DMD), 25.4% experienced significant sleep disturbances, which is much higher than the 3% prevalence in the general population.

The most common sleep issues included difficulties in initiating and maintaining sleep, and these disturbances were strongly linked to factors like needing assistance from a caregiver and being from a single-parent family, highlighting the importance of assessing sleep in DMD patients for better management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sleep disorders in boys with Duchenne muscular dystrophy. Bloetzer, C., Jeannet, PY., Lynch, B., et al.[2015]

In a study of 21 non-ambulant patients with Duchenne muscular dystrophy aged 13-23, 13 patients experienced sleep hypoxaemia, indicating that this condition is common among these individuals during sleep.

The study found that sleep hypoxaemia was often associated with obstructive apnoeas, and the frequency of hypoxaemic episodes increased with age, suggesting a need for careful monitoring and management of sleep-related breathing issues in these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Obstructive apnoeas in Duchenne muscular dystrophy. Khan, Y., Heckmatt, JZ.[2019]

The TranS-C intervention, when combined with usual care, significantly reduced functional impairment, psychiatric symptoms, and sleep disturbances in adults with serious mental illness, as shown in a study of 121 participants over 8 weeks.

These improvements were not only significant immediately after treatment but were also largely maintained at a 6-month follow-up, indicating the potential long-term benefits of the TranS-C approach for enhancing sleep and overall functioning.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A randomized controlled trial of the Transdiagnostic Intervention for Sleep and Circadian Dysfunction (TranS-C) to improve serious mental illness outcomes in a community setting. Harvey, AG., Dong, L., Hein, K., et al.[2022]

In a study of 54 children with Duchenne muscular dystrophy (DMD), 20% reported significant sleep impairment, which was linked to lower quality of life, highlighting the importance of addressing sleep issues in this population.

Actigraphy, a method of monitoring rest-activity patterns, showed that fragmented sleep rhythms in ambulatory children correlated with their subjective sleep quality, suggesting it could be a useful tool for assessing both sleep and motor function in DMD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Use of a wearable device to assess sleep and motor function in Duchenne muscular dystrophy. Siegel, BI., Cakmak, A., Reinertsen, E., et al.[2020]

The TranS-C intervention significantly improved sleep and circadian issues in 176 adolescents, which in turn reduced their risk in five health domains: emotional, cognitive, behavioral, social, and physical.

Key mediators of these improvements included reduced eveningness and daytime sleepiness, indicating that addressing sleep patterns can have broad benefits for at-risk adolescents.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Is improving sleep and circadian problems in adolescence a pathway to improved health? A mediation analysis. Dong, L., Gumport, NB., Martinez, AJ., et al.[2021]

Citations

1. centerwatch.com centerwatch.com/clinical-trials/listings/NCT04529707/sleep-intervention-in-young-boys-with-duchenne-muscular-dystrophy

Sleep Intervention in Young Boys with Duchenne Muscular ... This project will systematically plan and evaluate the implementation of the Transdiagnostic Sleep and Circadian Intervention for youth ...

2. withpower.com withpower.com/trial/phase-muscular-dystrophy-duchenne-1-2021-9b506

Sleep Intervention for Duchenne Muscular Dystrophy · Info ... The TranS-C intervention significantly improved sleep and circadian functioning in adolescents with an eveningness chronotype over a 12-month period compared to ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7242125/

A transdiagnostic sleep and circadian intervention for ... The evidence supports the TranS-C treatment effects over six months on improving sleep and circadian functioning on selected outcomes but not on reducing risk ...

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/38729603/

Transdiagnostic Sleep and Circadian Intervention in Youth This preregistered study compared the effects of the Transdiagnostic Sleep and Circadian Intervention (TranS-C) with psychoeducation (PE) about sleep, health, ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT06963463?intr=trans-c%2Bbl&rank=1

Transdiagnostic Sleep and Circadian Intervention + Bright ... This study will adapt TranS-C+BL for at-risk adolescents using health equity-informed implementation science methods. This randomized trial will enroll 75 youth ...

6. researchgate.net researchgate.net/publication/282563678_A_Transdiagnostic_Intervention_for_Youth_Sleep_and_Circadian_Problems

A Transdiagnostic Intervention for Youth Sleep and ... As a transdiagnostic treatment, TranS-C addresses a wide range of sleep and circadian problems that occur during adolescence instead of focusing ...