Published onTrisomy 21 Update
HEalthy sLeeP for Children with Down Syndrome (HELP-DS), a new multicenter study funded by the National Institutes of Health, could help clinicians and researchers develop new treatments for children who have Down syndrome and obstructive sleep apnea.
Children's Hospital of Philadelphia (CHOP) is the clinical coordinating center, while the data coordinating center is located at Brigham and Women’s Hospital in Boston. Participating clinical sites for the study include CHOP, Cincinnati Children's Hospital Medical Center, UT Southwestern Children’s Medical Center, Cleveland Clinic Children's, King’s Daughters Medical Center, and Children's Hospital of Michigan.
The goal of this study is to collect the necessary data to plan and execute a future pivotal randomized controlled clinical trial (RCT) of the role of obstructive sleep apnea (OSA) treatment in the care of children with Down syndrome (trisomy 21).
This study will provide critical information on:
- The methodological approaches for conducting such an RCT
- Informing the appropriate recruitment strategies
- Patient population
- Outcome measures for a future clinical trial
We will leverage expertise in pediatric sleep medicine, clinical trials, neuropsychology, and Down syndrome, as well as the infrastructure at the six clinical trial sites to catalyze a new Down syndrome clinical research initiative.
Our proposed next steps are to enroll in an observational study at these six centers with 50 children with Down syndrome who have been referred for clinically indicated evaluation and treatment of OSA. Children will undergo systematic assessments of sleep, behavior, cognition, daytime functioning, and quality of life at the beginning of the study, and again three and six months after adenotonsillectomy. This will generate key data to inform the design of a later pivotal RCT.