Clinical Trial Details
— Status: Active, not recruiting
Administrative data
| NCT number |
NCT04208685 |
| Other study ID # |
2018-0253 |
| Secondary ID |
|
| Status |
Active, not recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
June 18, 2018 |
| Est. completion date |
June 2026 |
Study information
| Verified date |
December 2023 |
| Source |
Children's Hospital Medical Center, Cincinnati |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial Summary
As basic and behavioral science identify new ways to improve cognition and behavior in
individuals with Down syndrome (DS), the lack of rigorous outcome measures represents an
important problem for interpreting findings. Null findings in clinical trials could result
from insensitive outcome measures, rather than ineffectiveness of treatment.
The long-term goal is to improve measurement of outcomes for children and adults with DS.
Towards that goal, the investigators propose to test and refine a battery of cognitive
measures that can be used in treatment studies focused on school-aged children and adults
with Down syndrome. The batteries are designed to assess key domains of the DS phenotype
where gaps remain in outcome measures, including attention, executive function, learning and
memory, processing speed, and social cognition.
The investigators will examine the psychometric properties of measures (test-retest,
validity, sensitivity to change), and to evaluate differences in the psychometric properties
of measures as a function of variations in participant age, gender, degree of ID, and the
participants' physical health and medical comorbidities. The investigators will evaluate at
least 80 children and 50 adults with Down syndrome, per site, at five time points to evaluate
key domains with a diverse and novel range of methods. This proposal aims to provide a
preliminary evaluation to support the enhancement of clinical outcome measures, which
ultimately will increase the accuracy in documenting improvements in the lives of children
and young adults with Down syndrome.
Description:
The purpose of the proposed project is to test and refine a battery of cognitive measures
that can be used in treatment studies focused on Down syndrome. The battery is designed to
assess several key domains of the Down syndrome cognitive phenotype where gaps remain in
outcome measures, including attention, executive function, learning and memory, processing
speed, and social cognition, considering comorbid conditions that can potentially interfere
with the development within these cognitive domains. Although a single battery for the entire
lifespan of the individual with Down syndrome has obvious appeal, the investigators have
focused their effort on the school-age years so that the investigators can capture a key time
point in development of these cognitive domains. Moreover, new pharmaceutical treatments are
transitioning from adults to the school-age population, making the need greatest for this
portion of the lifespan.
However, based on preliminary research from the ongoing study with children, a need was
identified to evaluate measures with young adults with DS (ages 18-39). Supplementary funding
will support the expansion of the project to include young adults with DS. Results will
provide empirical guidance for refining a battery and form the foundation for a larger-scale
study evaluating outcome measures.
For participants 6-17 years, at least 160 children with Down syndrome will be recruited
across two sites for the study, and participants will be enrolled over four (4) years.
Following pre-screening and the consent visit to determine study eligibility, children will
be assessed at 6 time points. The first assessment will be the baseline testing/Time 0 visit
to assess IQ. At Time 1 baseline assessments for other functioning will be completed. A
two-week interval will separate Time 1 and Time 2, at which time test-retest reliability
assessments will be collected. Time 3 will occur three months following Time 2 to evaluate
test sensitivity to change. Time 4 will occur 3 months following Time 3 and Time 5 will occur
6 months after Time 4 to assess longitudinal changes. The time interval was selected to
replicate the duration of many clinical trials.
For participants 18-35 years, at least one hundred young adults aged 18-29 years with DS will
be recruited across two sites to participate in the study. Adults will participate in
neuropsychology assessments at 5 time points. Parents/Caregivers will complete behavioral
measures at all time points. At Time 1 baseline assessments will be completed. A two-week
interval (+/- 3 days) will separate Time 1 and Time 2, when test-retest reliability
assessments will be collected. Time 3 will occur three months following Time 2 (+/- 2 weeks),
Time 4 will occur three months following Time 3 (+/- 2 weeks), and Time 5 will occur 6 months
following Time 4 (+/- 2 weeks) to evaluate sensitivity to change, Time 6 will occur 24 months
following Time 1 (+/- 4 weeks), Time 7 will occur 36 months following Time 1 (+/- 4 weeks),
and Time 8 will occur 48 months following Time 1 (+/- 4 weeks) to evaluate sensitivity to
change. The three and six-month time intervals were selected to replicate the duration of
many clinical trials. The 12-month interval was selected to provide a time interval in which
natural development should occur and allow the investigators to evaluate sensitivity to
change. In this study, change will occur due to natural development, while in a clinical
trial, change would be expected due to treatment. The ability to detect relatively small
developmentally-based change over 12 months will allow the investigators to evaluate if the
measures could detect similarly small changes in trials. Once recruitment goals are met for
NIH standards for 100 young adults, aged 18-29, the study team may begin recruiting adults
who are 29-35, as well.
