Clinical Trial Details
— Status: Terminated
Administrative data
| NCT number |
NCT04056650 |
| Other study ID # |
GCO 18-0756 |
| Secondary ID |
|
| Status |
Terminated |
| Phase |
Phase 4
|
| First received |
|
| Last updated |
|
| Start date |
October 18, 2019 |
| Est. completion date |
December 22, 2020 |
Study information
| Verified date |
March 2021 |
| Source |
Icahn School of Medicine at Mount Sinai |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Interventional
|
Clinical Trial Summary
The growing consumer-grade molecular and digital wellness market is generating unprecedented
volumes of information to support decision-making around individual health. Current trends
suggest the demand for personalized health information, tools, and services will continue to
rise in the next decade. What is missing is a reliable, individualized way to turn this data
into action. Dialogue around consumer health often ignores the disconnect between
measurements and goals. For example, monitoring one's weight is not the same as losing
weight, and counting steps is not the same as lowering blood pressure. If individuals are to
benefit from data, they must be able to relate changes in their personal data to targeted
changes in actions and outcomes. There is a great need and opportunity to adapt the tools and
capabilities of modern computer science, statistics, and clinical trial design to the needs
of individual patients and consumers. The team at the Institute for Next Generation
Healthcare (INGH) has created a smartphone-based app ("N1 app") and study platform that
together allow individuals to design, implement, and analyze methodologically sound,
statistically robust studies of their personal health data. The focus of the platform will be
the creation of single-participant randomized crossover studies, known as n-of-1 trials. The
platform employs informatics-based intelligence that automates study design and analysis
while simultaneously maintaining high standards of statistical rigor and reproducibility.
These novel methods and tools are designed to empower individuals to make rational,
data-driven choices about their own health, maximizing the benefit all will receive from new
and existing sources of personal health data.
Description:
The growing burden of chronic disease in the U.S. and the economics of accountable care are
driving a shift toward proactive approaches to disease prevention and health maintenance. At
the same time, precision medicine studies continue to reveal substantial heterogeneity in the
manifestations of even the most common chronic diseases. The bulk of morbidity and mortality
in the U.S. arises from conditions with a significant lifestyle component (e.g. type II
diabetes), and responsibility for monitoring and maintaining health largely falls on
individuals.
Recent advances in molecular biology, sensors, and digital health technology underlie rapidly
growing market availability of products and devices for measuring and monitoring individual
health. A vast array of wearable devices, smart home monitors, and health tracking apps
provide an unprecedented view of individuals "in the wild" and provide customers with health
information once accessible only to researchers. The growing digital health market is
generating unprecedented volumes of information to support decision making around individual
health, and current trends suggest the demand for personalized health information, tools, and
services will continue to grow in the next decade.
What is missing from this technological and scientific growth is a reliable, individualized
way to translate data into action. If society wants to prevent diabetes, heart disease, and
other chronic illnesses that kill millions of Americans each year, individuals must be
empowered to address precursor conditions like obesity, hypertension, and depression.
Dialogue around consumer health often fails to address the profound disconnect between
measurements and outcomes/goals; e.g. monitoring one's weight is not the same as losing
weight, and counting steps is not the same as lowering blood pressure. Data are only useful
if they can help individuals identify interventions that work for them. The combination of
diet, exercise, drugs/supplements, activities, and lifestyle changes that targets an
individual's particular set of health problems is unique to him or her, and it is dependent
on a complex web of factors including genetics, environment, and personal lifestyle. If
individuals are to benefit from data, they must be able to relate changes in their personal
data to targeted adjustments in actions and outcomes. This effectively necessitates
conducting a robust trial at the level of the individual to determine the most promising
recipe of personal lifestyle adjustments to effect change.
To address these challenges, the researchers have developed a unified statistical framework
for producing consistent, interpretable study results from diverse n-of-1 study designs. The
analysis framework is the backbone of the initial software platform, which includes modules
for study design, e-consent, data ingestion, data analysis, and visualization of results.
To test this platform, the researchers plan to deploy a prototype study that allows
individuals to test the cognitive effects of two commonly consumed substances: caffeine and
caffeine in combination with a safe, prevalent compound, L-theanine. Each enrolled individual
will participate in his/her own n-of-1 trial. After a baseline period where neither treatment
is taken, participants will alternate between the two treatments ("caffeine alone" and
"caffeine + L-theanine") according to a predefined schedule. Participants will complete a
daily cognitive assessment composed of 3 validated cognitive tests administered via the N1
app. The platform will analyze the cognitive assessment data and determine whether there is a
statistically meaningful treatment effect for either treatment compared to baseline for any
of the 3 cognitive tests for each individual that completes the study.
It is important to state explicitly that the research objectives for this protocol are not
related to the efficacy of L-theanine and caffeine. This specific study is designed to allow
the researchers to efficiently recruit and enroll subjects so that the underlying statistical
methods and software platform for executing n-of-1 trials may be evaluated.
