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Healthy Sleep for Children With Down Syndrome (HELP-DS)

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. Identifier: NCT03922165
Recruitment Status : Recruiting
First Posted : April 19, 2019
Last Update Posted : January 26, 2021
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
Information provided by (Responsible Party):
Susan Redline, Brigham and Women's Hospital

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
Down Syndrome Sleep Disordered Breathing 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.

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Study Type : Observational
Estimated Enrollment : 50 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Healthy Sleep for Children With Down Syndrome
Actual Study Start Date : May 22, 2019
Estimated Primary Completion Date : July 1, 2021
Estimated Study Completion Date : December 1, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Down Syndrome

Group/Cohort 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.

Primary Outcome Measures :
  1. % enrolled/approached [ Time Frame: 1 year ]
    Percentage of families approached for participation that sign informed consent to participate.

  2. % retained/enrolled [ Time Frame: 1 year ]
    Percentage of consented families that completed study participation.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   3 Years to 13 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Dyads of caregivers and children with DS aged 3-13 years diagnosed with SDB and referred for treatment with adenotonsillectomy (AT) will be recruited.

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03922165

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Contact: Michael G Morrical 8573070332
Contact: Jessie P Bakker, PhD 6177325987

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United States, Michigan
University of Michigan Health System Not yet recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Libby Hobson    734-936-4817   
Principal Investigator: Ronald D Chervin, MD         
Sub-Investigator: Fauziya Hassan, MBBS, MS         
Sub-Investigator: Susan L Garetz, MD         
Sub-Investigator: David A Zopf, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Not yet recruiting
Cincinnati, Ohio, United States, 45229
Contact: Suzanna Hicks    513-636-4944   
Principal Investigator: Raouf S Amin, MD         
Sub-Investigator: Stacey L Ishman, MD, MPH         
University Hospitals-Case Medical Center Not yet recruiting
Cleveland, Ohio, United States, 44106
Contact: Heather Rogers    216-368-0475   
Principal Investigator: Carol L Rosen, MD         
Sub-Investigator: Kristie R Ross, MD         
Sub-Investigator: Hudson G Taylor, PhD         
Sub-Investigator: Jay Shah, MD         
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Laurie Rosenthal    215-590-9184   
Principal Investigator: Ignacio E Tapia, MD         
Sub-Investigator: Jerilynn Radcliffe, PhD         
Sub-Investigator: Lisa M Elden, MD MS FRCSC         
United States, Texas
University of Texas Southwestern Medical Center Not yet recruiting
Dallas, Texas, United States, 75390
Contact: Lauren Plummer    214-456-2269   
Principal Investigator: Ronald B Mitchell, MD         
Sub-Investigator: Kamal M Naqvi, MD         
United States, Virginia
Children's Hospital of the King's Daughters Not yet recruiting
Norfolk, Virginia, United States, 23507
Contact: Laura Stone    757-388-6238   
Principal Investigator: Christina M Baldassari, MD         
Sub-Investigator: Michael J Strunc, MD         
Sub-Investigator: Jennifer L Wiebke, MD         
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
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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
Additional Information:

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Responsible Party: Susan Redline, Professor, Brigham and Women's Hospital Identifier: NCT03922165    
Other Study ID Numbers: 18-015848
First Posted: April 19, 2019    Key Record Dates
Last Update Posted: January 26, 2021
Last Verified: January 2021

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Susan Redline, Brigham and Women's Hospital:
Additional relevant MeSH terms:
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Sleep Apnea Syndromes
Down Syndrome
Pathologic Processes
Respiration Disorders
Respiratory Tract Diseases
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Sleep Disorders, Intrinsic
Sleep Wake Disorders