Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT06278961 |
| Other study ID # |
IRB 2021-4329 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
January 28, 2021 |
| Est. completion date |
September 30, 2024 |
Study information
| Verified date |
February 2024 |
| Source |
Ann & Robert H Lurie Children's Hospital of Chicago |
| Contact |
Ryan Millman, B.S. |
| Phone |
847-530-2319 |
| Email |
ryan.millman[@]northwestern.edu |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational
|
Clinical Trial Summary
The study objective is to improve accuracy in the early detection of neurodevelopmental
impairment, especially CP, by evaluating the timepoint (in weeks post term age) that the
Prechtl GMA is most useful for prediction of neurodevelopmental impairment at two years of
age in children with and without medical complexity. The study team plans to recruit 100
healthy, term-born infants and 250 infants at risk of developing CP for a total of 350
enrolled infants.
Description:
Research teams at Northwestern (Prentice), University of Sydney, Fondazione IRCCS Stella
Maris, Cerebral Palsy Alliance (CPA) and Murdoch Children's Research Institute (MCRI) will be
collecting data in the same manner using the Baby Moves app with the inclusion and exclusion
criteria above. The data from the Baby Moves app will be temporarily stored in a common
REDcap database at the Murdoch Children's Research Institute (MCRI) with all sites included.
The MCRI has all necessary approvals in place to host the videos. Video files from Prentice
and Lurie will be transferred to a common REDCap Database at Northwestern. Other sites
(University of Sydney, MCRI, Stella Maris, CPA), will have their own REDCap databases where
the data is stored and managed. The research teams at these sites will be responsible for
recruiting and consenting subjects. All sites (except Prentice) will seek IRB approval from
their respective sites.
Study design: Prospectively recruited cohort study.
Study procedures:
Eligible families will be identified using the Electronic Data Warehouse (Prentice), through
Data Analytics and Reporting (DAR) Lurie, the electronic medical record platform EPIC, or
through recruitment on social media websites and family advocacy websites. Families will be
contacted via email and/or text message and will have the option to enroll through electronic
consent (econsent) in order to minimize in-person contact. Participating families will be
able to enroll online using REDCap. If the family has questions about enrollment or the
consent form, they will be able to notify a member of the study team, using a survey form on
REDCap. A member of the research team can contact the family by phone to provide information
and answer questions about participating in the study. Participating families will then
receive directions on the phone on how to use and download the Baby Moves app on their
smartphones.
Participating families will download the Baby Moves app on their smartphones after
enrollment. A member of the research team will register the infant into the study database,
using a study ID, infant birth date, and infant due date. The participating families may
watch a 3-minute demonstration video on use of the app and proper filming technique.
A member of the research team will also record the subject's pertinent medical information
(neurological findings, gestational age at birth, birthweight and neonatal medical
complications, severity of ROP, and treatment for ROP, if SMA: SMA type, SMN2 copy number if
known, date of dx, and pharmacologic treatment, treatment for SMA and date of 1st treatment)
into a REDcap database.
Parents will receive a reminder on their smartphone through both e-mail and push notification
at required filming time points (10, 12, 14, 16, 18 and 20 weeks of corrected age). Families
will be reminded to film the infant. Parents will receive the following family-friendly and
standardized instructions for filming the baby:
All participants will be asked to upload video recordings using the Baby Moves app outside of
their standard of care.
Infant videos will be uploaded into a HIPAA-compliant REDcap database and the research team
will be notified when the family has taken the video using the app.
Two advanced-trained general movement assessors will independently review the video within
3-5 days of receiving it, using Prechtl methodology (see below). These assessors will be part
of the study team from any of the participating sites but will be blinded to infant data and
PHI.
General Movement Assessment: General movements are a developmentally regulated pattern of
spontaneous motor activity, appearing in the embryo by 9 weeks post-conceptional age. By
50-55 weeks post-conceptional age (10-15 weeks post-term), the predominant general movements
seen are termed Fidgety Movements - a pattern of continuous, small amplitude movements of the
neck, trunk and limbs during wakefulness that disappear with agitation. These patterns are
identifiable and are classified as normal if present (intermittent or continual), and
abnormal if exaggerated (with respect to speed and amplitude), sporadic (interspersed with
long pauses) or absent. Importantly, absence of these fidgety movements at 10-15 weeks
post-term accurately predicts the development of cerebral palsy(4). Certified raters will use
the 3-minute video filmed by families using the Baby Moves app to categorize infant general
movements according to the Prechtl methodology(5) and to quantify movement patterns.
The family will be notified of the results within one week of uploading the final video at 20
weeks corrected age.
If the videos are deemed by raters to be normal, the family will receive notification by
email.
If the infant's general movements are deemed to be high-risk, the participating family will
receive a phone call and will be scheduled for a follow-up visit (either as an outpatient or
in their home).
The families will not receive a formal diagnosis but will be triaged to a virtual visit from
a physician or physical therapist. At this time they will receive 2 standardized
developmental assessments: the Alberta Infant Motor Scale (AIMS) and a modified version of
the Hammersmith Infant Neurological Examination (HINE). These videos will be completed using
the videoconferencing software Zoom (hosted through. The study team will use the waiting room
feature to minimize loss of confidentiality risk. The files from these encounters will be
stored in FSM resfiles and/or on encrypted devices.
Infants with high-risk general movements will also be referred to local Early Intervention
Services.
For the very few cases of enrolled participants who take videos from Prentice Women's
Hospital or Lurie Children's Hospital and who are deemed high-risk. we will share the risk
level based on the study's screening with a clinician in charge of developmental care (such
as Dr. Raye-Anne deRegnier, who also happens to be a co-investigator on this study). We can
communicate with the clinical care team so that the team can accurately and unambiguously
share the participants study screening results with the family. In this special case, we will
send a follow up email to families thanking them for participating and letting the families
know that we have shared the result with the developmental team at their respective Hospital.
Video data from the Baby Moves app will be securely stored in the REDCap database.
Duration of the Study: Infants will be followed for 5 years. Data will be collected until the
target of 450 subjects is achieved for all sites.
For follow-up the infants will be grouped into two categories:
1. Children who are enrolled in a high-risk follow-up clinic
2. Children who are not enrolled in a high-risk follow-up clinic
Infants in group 1 (enrolled in high-risk follow-up clinic) Infants from all sites who are
already enrolled in a high-risk follow-up clinic, will be followed for five years, using
site-specific standardized neurodevelopmental outcome measures, which are routinely done in
their respective follow-up clinics. If enrolled in an SMA follow up clinic a similar follow
up protocol will take place, using site specific SMA, motor function outcome measures, which
are routinely done in their respective follow-up clinics. Data will be collected from
standard visit data and entered into REDCap.
Infants in group 2: Will be asked to complete surveys at 1 and 2 years of corrected age
called the Ages and Stages questionnaire. They will send this survey back electronically or
by mail.
The difference between groups 1 and 2 is that group 1 will not be asked to complete the Ages
and Stages questionnaires at 1 and 2 years of age.
All families from groups 1 and 2 will fill out a survey about the Baby Moves app that will be
completed electronically. Each questionnaire will take approximately 5-15 minutes to
complete. Infants from all sites may also receive follow-up phone calls that will be
completed by the parents at 12 and 24 months. These phone surveys will require 1-3 minutes of
the parents time.
Data including names, addresses, names, dates, telephone numbers, addresses, email, infant's
medical history, video data, questionnaire results and neurodevelopmental tests will be
collected prospectively.
Researchers from all sites will have access to the REDcap database. This REDCap database will
be managed by Northwestern University. Data use agreements are being established among sites.
Additionally, individual expert raters in the general movement assessment from additional
institutions, working as content experts will also have access to de-identified and coded
data
While they will not have personal health identifiers, these raters will be able to see the
face of the infants in the videos. The facial expressions and movements of these infants are
required in rating the videos. The observation of faces are not considered to be identifiers
due to the young age of the subjects and their quickly changing appearance.
ONE YEAR FOLLOW-UP Infants recruited will receive a one-year-old birthday card from the study
team, and their families will complete a 12-month Ages and Stages Questionnaire at their
child's 12 month corrected age as a global developmental screening with a member of the study
team.
TWO-YEAR FOLLOW-UP, NEURODEVELOPMENTAL ASSESSMENTS Participating families will return for a
two-year follow-up visit and will receive the following batteries of standardized
assessments.
Table 1. Two-year neurodevelopmental assessment battery
Assessment name
Assessment requirements
Constructs assessed
Bayley Scales of Infant and Toddler Development, version 4 (Bayley-4)
Trained assessor, standardized test kit with manipulatives, stimulus book, manual
Cognitive, language, motor skills, norm-referenced
Infant Toddler Quality of Life Questionnaire (ITQOL)
Parent questionnaire
Quality of Life
Child Behavior Checklist (CBCL)
Parent questionnaire
Socioemotional
Gross motor functional measure (GMFM)-66*
Trained assessor, manual, standardized equipment
Motor ability in children diagnosed with cerebral palsy
*Only infants diagnosed with CP will be tested with the GMFM-66
We will also record whether a child has received a diagnosis of CP. If they have not received
a diagnosis but have high-risk based on two-year presentation, we will refer patients for
diagnostic follow-up and then record outcome of that diagnosis.
