免费获得国外相关药品，最快 1 个工作日回馈药物信息
Healthy Sleep for Children With Down Syndrome (HELP-DS)
| Condition or disease | Intervention/treatment |
|---|---|
| Down Syndrome Sleep Disordered Breathing | Procedure: Adenotonsillectomy |
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:
- 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).
- 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.
- 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 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 |
| 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.
|
- % enrolled/approached [ Time Frame: 1 year ]Percentage of families approached for participation that sign informed consent to participate.
- % retained/enrolled [ Time Frame: 1 year ]Percentage of consented families that completed study participation.
| Ages Eligible for Study: | 3 Years to 13 Years (Child) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
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.
| Contact: Michael G Morrical | 8573070332 | mmorrical@partners.org | |
| Contact: Jessie P Bakker, PhD | 6177325987 | jpbakker@partners.org |
| United States, Michigan | |
| University of Michigan Health System | Not yet recruiting |
| Ann Arbor, Michigan, United States, 48109 | |
| Contact: Libby Hobson 734-936-4817 mehobson@med.umich.edu | |
| 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 suzanna.hicks@cchmc.org | |
| 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 heather.rogers@uhhospitals.org | |
| 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 karamessil@email.chop.edu | |
| 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 Lauren.Plummer@childrens.com | |
| 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 stonelj@evms.edu | |
| Principal Investigator: Christina M Baldassari, MD | |
| Sub-Investigator: Michael J Strunc, MD | |
| Sub-Investigator: Jennifer L Wiebke, MD | |
| 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 |
| Tracking Information | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| First Submitted Date | April 17, 2019 | |||||||||
| First Posted Date | April 19, 2019 | |||||||||
| Last Update Posted Date | January 26, 2021 | |||||||||
| Actual Study Start Date | May 22, 2019 | |||||||||
| Estimated Primary Completion Date | July 1, 2021 (Final data collection date for primary outcome measure) | |||||||||
| Current Primary Outcome Measures |
|
|||||||||
| Original Primary Outcome Measures | Same as current | |||||||||
| Change History | ||||||||||
| Current Secondary Outcome Measures | Not Provided | |||||||||
| Original Secondary Outcome Measures | Not Provided | |||||||||
| Current Other Pre-specified Outcome Measures | Not Provided | |||||||||
| Original Other Pre-specified Outcome Measures | Not Provided | |||||||||
| Descriptive Information | ||||||||||
| Brief Title | Healthy Sleep for Children With Down Syndrome | |||||||||
| Official Title | Healthy Sleep for Children With Down Syndrome | |||||||||
| 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. | |||||||||
| 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:
These aims have substantial public health significance given the high morbidity of Down syndrome and sleep-disordered breathing in children. |
|||||||||
| Study Type | Observational | |||||||||
| Study Design | Observational Model: Cohort Time Perspective: Prospective |
|||||||||
| Target Follow-Up Duration | Not Provided | |||||||||
| Biospecimen | Not Provided | |||||||||
| 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. | |||||||||
| Condition |
|
|||||||||
| Intervention | Procedure: Adenotonsillectomy
Routine AT per clinical care.
|
|||||||||
| Study Groups/Cohorts | 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.
Intervention: Procedure: Adenotonsillectomy
|
|||||||||
| Publications * |
|
|||||||||
|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
||||||||||
| Recruitment Information | ||||||||||
| Recruitment Status | Recruiting | |||||||||
| Estimated Enrollment |
50 | |||||||||
| Original Estimated Enrollment | Same as current | |||||||||
| Estimated Study Completion Date | December 1, 2021 | |||||||||
| Estimated Primary Completion Date | July 1, 2021 (Final data collection date for primary outcome measure) | |||||||||
| Eligibility Criteria |
Inclusion Criteria:
Exclusion Criteria:
|
|||||||||
| Sex/Gender |
|
|||||||||
| Ages | 3 Years to 13 Years (Child) | |||||||||
| Accepts Healthy Volunteers | No | |||||||||
| Contacts |
|
|||||||||
| Listed Location Countries | United States | |||||||||
| Removed Location Countries | ||||||||||
| Administrative Information | ||||||||||
| NCT Number | NCT03922165 | |||||||||
| Other Study ID Numbers | 18-015848 | |||||||||
| Has Data Monitoring Committee | Yes | |||||||||
| U.S. FDA-regulated Product |
|
|||||||||
| IPD Sharing Statement | Not Provided | |||||||||
| Responsible Party | Susan Redline, Brigham and Women's Hospital | |||||||||
| Study Sponsor | Brigham and Women's Hospital | |||||||||
| Collaborators |
|
|||||||||
| Investigators |
|
|||||||||
| PRS Account | Brigham and Women's Hospital | |||||||||
| Verification Date | January 2021 | |||||||||
