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HELP-DS: Healthy Sleep for Children With Down Syndrome

Sponsor
Brigham and Women's Hospital (Other)
Overall Status
Completed
CT.gov ID
NCT03922165
Collaborator
Children's Hospital of Philadelphia (Other), University Hospitals Cleveland Medical Center (Other), Children's Hospital Medical Center, Cincinnati (Other), University of Michigan (Other), University of Texas Southwestern Medical Center (Other), University of Rochester (Other), Children's Hospital of The King's Daughters (Other)
26
Enrollment
6
Locations
31.3
Actual Duration (Months)
4.3
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this multi-center observational study (utilizing the sites enrolling patients for the Pediatric Adenotonsillectomy Trial for Snoring (PATS) [1U011HL125307-O1A1]) is to gather data regarding children with Down syndrome (DS) and Sleep Disordered Breathing (SDB) referred for treatment with adenotonsillectomy to inform a future randomized controlled trial in this population.

Condition or Disease Intervention/Treatment Phase
  • Procedure: Adenotonsillectomy

Detailed Description

Down syndrome (DS) is associated with a wide range of neurobehavioral and physical morbidities. Obstructive sleep apnea (OSA) is prevalent in children with DS and causes a myriad of physiological stresses that may exacerbate neurobehavioral and cardiac morbidities. Improving sleep health thus provides a modifiable intervention target for improving behavior, cognition, quality of life, and physical health of children with DS. However, critical knowledge gaps limit the adoption and implementation of effective OSA interventions, resulting in practice variability and suboptimal treatment of OSA across the spectrum of DS. In particular, it is in unclear how to select candidates likely to benefit from adenotonsillectomy (AT) and how to utilize information from polysomnographic, clinical, and demographic parameters to predict children most likely to benefit from AT compared to alternative treatments or to watchful waiting. The goal of this study is to collect the necessary data to plan and execute a future pivotal RCT of the role of OSA treatment in the care of children with DS. This study will provide critical information on the methodological approaches for conducting such a randomized controlled clinical trial (RCT), informing the appropriate recruitment strategies, patient population and outcome measures for a future clinical trial. The investigators will leverage expertise in pediatric sleep medicine, clinical trials, neuropsychology, and DS as well as the PATS clinical trials infrastructure to catalyze a new DS clinical research initiative. Our proposed next steps are to enroll in an observational study at active PATS centers 50 children with DS who are referred for clinically indicated evaluation and treatment of OSA. At baseline and 6 months following AT, these children will undergo systematic assessments of sleep, behavior, cognition, daytime functioning, and quality of life, generating key data to inform the design of a later pivotal RCT.

Healthy Sleep for Children with Down Syndrome (HELP-DS) intends to take advantage of a successful collaboration of leaders in sleep medicine, otolaryngology and clinical trials to efficiently leverage experiences from the The Childhood Adenotonsillectomy Trial (CHAT) as well as the current resources of the PATS trial to collect data for the evaluation of key questions needed to design a future RCT, including approaches related to the selection of participating sites, the patient population to receive the intervention, and outcome measurements.

The primary objective of HELP-DS to determine the feasibility of recruiting and retaining individuals in an observational study of AT across multiple clinical sites.

Secondary objectives are:

  1. Evaluate the feasibility of collecting a range of baseline and outcome data, and specifically the ability of children to perform neurobehavioral assessments (GoNoGo, pegboard task).

  2. Understand issues related to patient preference, equipoise, and barriers to treatment. The investigators will track how many children referred for AT actually undergo surgery, the time interval between referral and surgery, other interventions pursued, and factors such as insurance, socio-economic factors, and cultural norms, that associate with family-decision making.

  3. Examine the consistency and completeness of Healthcare Utilization (HCU) data extracted across our sites, describing differences by recruitment site, health, system characteristics, insurance-related factors, etc. The investigators will estimate incidence rates of HCU events for hospital admissions, emergency department/unscheduled office visits, specialty consultations and medication use during the 6 months following surgery and compare rates to those for the year prior to surgery.

These aims have substantial public health significance given the high morbidity of Down syndrome and sleep-disordered breathing in children.

Study Design

Study Type:
Observational
Actual Enrollment :
26 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
Healthy Sleep for Children With Down Syndrome
Actual Study Start Date :
May 22, 2019
Actual Primary Completion Date :
Dec 20, 2021
Actual Study Completion Date :
Dec 31, 2021

Arms and Interventions

Arm Intervention/Treatment
DS peds eligible for Adenotonsillectomy

Dyads of caregivers and children with DS aged 3-13 years diagnosed with SDB and referred for treatment with adenotonsillectomy.

Procedure: Adenotonsillectomy
Routine AT per clinical care.

Outcome Measures

Primary Outcome Measures

  1. % enrolled/approached [1 year]

    Percentage of families approached for participation that sign informed consent to participate.

  2. % retained/enrolled [1 year]

    Percentage of consented families that completed study participation.

Eligibility Criteria

Criteria

Ages Eligible for Study:
3 Years to 13 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:

  • DS diagnosis regardless of genetic status (e.g., mosaicism or translocation).

  • Aged >3 to <13 years at the date of consent.

  • Primary indication for AT is nocturnal obstructive symptoms (i.e., not recurrent infections or other indications).

  • Deemed to be a candidate for AT by Ear, Nose and Throat (ENT) evaluation; that is, no technical issues that would be a contraindication for surgery such as submucous cleft palate.

Exclusion Criteria:

  • Prior tonsillectomy (partial or complete).

  • Severe chronic health conditions that would contradict surgery (severe morbid obesity, unrepaired cyanotic congenital heart disease, bleeding disorders).

  • Severe behavioral problems that would preclude participation in the study's testing procedures (PSG, actigraphy).

  • Severe OSA with respiratory failure needing urgent/emergent management

  • Plan to undergo additional airway surgery at the time of AT.

  • Caregiver/child planning to move out of the area within 6 months.

  • Caregiver/child does not speak English or Spanish well enough to complete the behavioral and performance measures.

  • Child in foster care.

Contacts and Locations

Locations

Site City State Country Postal Code
1 University of Michigan Health System Ann Arbor Michigan United States 48109
2 Cincinnati Children's Hospital Medical Center Cincinnati Ohio United States 45229
3 University Hospitals-Case Medical Center Cleveland Ohio United States 44106
4 Children's Hospital of Philadelphia Philadelphia Pennsylvania United States 19104
5 University of Texas Southwestern Medical Center Dallas Texas United States 75390
6 Children's Hospital of the King's Daughters Norfolk Virginia United States 23507

Sponsors and Collaborators

  • Brigham and Women's Hospital
  • Children's Hospital of Philadelphia
  • University Hospitals Cleveland Medical Center
  • Children's Hospital Medical Center, Cincinnati
  • University of Michigan
  • University of Texas Southwestern Medical Center
  • University of Rochester
  • Children's Hospital of The King's Daughters

Investigators

  • Principal Investigator: Susan S Redline, MD, MPH, Brigham and Women's Hospital
  • Principal Investigator: Rui Wang, PhD, Brigham and Women's Hospital
  • Principal Investigator: Susan L Furth, Md, PhD, Children's Hospital of Philadelphia

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

Publications

Responsible Party:
Susan Redline, Professor, Brigham and Women's Hospital
ClinicalTrials.gov Identifier:
NCT03922165
Other Study ID Numbers:
  • 18-015848
First Posted:
Apr 19, 2019
Last Update Posted:
Mar 2, 2022
Last Verified:
Mar 1, 2022
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Keywords provided by Susan Redline, Professor, Brigham and Women's Hospital
Pediatrics
Adenotonsillectomy
Additional relevant MeSH terms:
Sleep Apnea Syndromes
Down Syndrome
Syndrome

Study Results

No Results Posted as of Mar 2, 2022