Clinical Trial Details
— Status: Completed
Administrative data
| NCT number |
NCT05377008 |
| Other study ID # |
HM20024159 |
| Secondary ID |
|
| Status |
Completed |
| Phase |
N/A
|
| First received |
|
| Last updated |
|
| Start date |
May 13, 2022 |
| Est. completion date |
May 5, 2023 |
Study information
| Verified date |
September 2023 |
| Source |
Virginia Commonwealth University |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Interventional
|
Clinical Trial Summary
Children with neurofibromatosis are more likely to have difficulties related to their
psychological and neurocognitive functioning (e.g., more likely to have depression, have
social difficulties, be diagnosed with ADHD). The purpose of this randomized control study is
to determine how effective and useful this study's single session intervention can be in
improving psychological and neurocognitive functioning. Enrolled families will consist of one
parent/guardian and child. Parents and patients will complete questionnaires and objective
tests at baseline, 3 months, and 6 months. Families randomized to the intervention arm will
be provided with one single session intervention at Month 1 to learn about their child's
testing results and receive psychoeducation and recommendations related to psychological and
neurocognitive functioning.
Description:
Neurofibromatosis type 1 (NF1) is a chronic illness that is typically diagnosed during
childhood and is associated with a variety of medical, psychological, and neurocognitive
symptoms. NF1 is a genetic disorder, affecting about 1 in 3,000 people throughout the world.
Diagnosis usually occurs in childhood and is characterized by the presence of several
markers, including, but not limited to: multiple café au lait (light brown) skin spots,
neurofibromas (small benign growths) on or under the skin, optic pathway gliomas, bone
dysplasia, and freckling in the armpits or groin. Aside from its medical manifestations, NF1
also is associated with psychological impairment and a wide variety of neurocognitive
deficits. In comparison to healthy peers and the general population, children and adolescents
with NF1 have been found to have more social difficulties (e.g., more peer conflicts, fewer
friends), internalizing symptoms (e.g., anxiety, depression, withdrawal), externalizing
symptoms (e.g., sub-threshold or clinical symptoms consistent with ADHD such as inattention,
hyperactivity, impulsivity), sleep difficulties; and worse quality of life. Additionally,
while the vast majority of children with NF1 have intelligence in the average to low average
range, neurocognitive deficits have been found in a range of domains including: linguistic
development, verbal concept formation, reasoning, visuospatial skills, motor skills,
executive function (e.g., cognitive flexibility, working memory, inhibitory control),
academic achievement (e.g., reading/spelling, math), and attention. There is further support
that early onset of mental health problems are associated with both future mental health
problems and poorer functional outcomes in adulthood (e.g., unemployment, lower earnings,
lower academic attainment).
Children with NF are more likely to be diagnosed with Attention-Deficit/Hyperactivity
Disorder (ADHD) than their healthy counterparts. Early detection, prevention, and
intervention of psychological and neurocognitive risk may serve as a protective factor for
later adverse outcomes. A meta-analysis of 175 research studies worldwide on ADHD in children
ages 18 and younger found an overall pooled estimate of 7.2% of children and adolescents
diagnosed with ADHD within the general population; however, ADHD is over-represented within
the NF1 population, with an estimated 31 to 50% of children meeting diagnostic criteria for
ADHD. Although there is limited research investigating the longitudinal impact of ADHD and NF
on pediatric patients, longitudinal studies of children with ADHD document deficits in
multiple domains including lower levels of education, poorer social functioning, and worse
occupational outcomes. Therefore, children with comorbid diagnoses of both NF1 and ADHD or
even those NF patients with sub-threshold symptoms of ADHD are likely to be at an increased
risk for functional deficits later in life.
Given the general current demand for psychological services, there is a great need for brief,
problem-focused psychological interventions targeting psychological and neurocognitive
functioning in NF. Emerging data indicate a significant increase in mental health needs as a
result of the COVID-19 pandemic, which is consistent with previous large-scale disasters and
pandemics. This is a significant healthcare crisis given that prior to the pandemic in the
United States, more than 11.8 million people reported having unmet mental health needs. This
translates to more individuals needing mental health intervention without access; and,
subsequently challenges mental health providers to expand services through novel models of
intervention delivery (e.g., delivering brief problem-focused interventions based on findings
from standardized assessments in unconventional settings, such as, during multidisciplinary
medical visits for their medical condition). One novel delivery method of psychological
services is the single-session intervention (SSI), which is defined as the intentional
delivery of one visit or encounter with a clinic, provider, or program. Although there is no
current research investigating the feasibility and clinical effectiveness of SSI's for
pediatric patients diagnosed with NF, a meta-analysis of 50 randomized control trials of
SSI's for youth with psychological problems showed a significant beneficial effect (g=0.32),
with greatest effect sizes for reducing anxiety (g=0.56) and conduct problems (g=0.54).
Although psychoeducation-based SSI's often were found to have a small effect size, a
systematic review of 13 studies conducted between 1990 and 2018 that investigated the effects
of psychoeducation-based interventions on youth with ADHD showed moderate to large effects on
ADHD symptom improvement as reported by parents/teachers (g=0.787), and parent/teacher and
child knowledge about ADHD (g=1.037 and g=0.721, respectively).
This is a pilot feasibility study comprising a randomized controlled trial involving
patients' ages 6 to 16 years (n=50) with NF. Patients will complete standardized assessment
batteries and questionnaires assessing their neurocognitive, executive, and psychological
functioning. The present study will yield longitudinal patterns of neurocognitive and
psychosocial outcomes across the six month period, and, collect data for preliminary
development of a single session intervention. In order to gather information on how patients
perform and behave in different settings, patients, parents, and teachers will also complete
standardized questionnaires to gather information on psychological functioning over a six
month period. Given NF is associated with psychological impairment and a wide variety of
neurocognitive deficits, as well as poorer functional outcomes in adulthood, there is a great
need for early detection, prevention, and intervention for this vulnerable patient
population.
The proposed study has the following specific aims: Aim 1) Examine the preliminary
feasibility and acceptability of using a single-session intervention focused on
psychoeducation and cognitive behavioral-based recommendations related to psychological,
neurocognitive, and executive functioning over six months. Hypothesis 1a: We hypothesize that
families will report high acceptability for the intervention. Hypothesis 1b: It is
hypothesized that families assigned to the intervention group will demonstrate improvements
in both objective and subjective assessments of psychological and neurocognitive symptoms,
e.g., inattention and impulsivity, as well as, executive functioning when compared to those
in the control group. Aim 2) Examine descriptive and longitudinal patterns of psychological
and neurocognitive factors (e.g., intellectual functioning, executive functioning, attention)
across six months for the sample overall, as well as, for those in the intervention and
control groups. Hypothesis 2a: It is hypothesized that a similar prevalence of ADHD for
patients with NF that is seen in the literature (i.e., between 31-50%) will also be observed
in our sample. Hypothesis 2b: It is hypothesized that there will be individual differences in
neurocognitive patterns (e.g., executive functioning, attention) across six months in
pediatric patients with neurofibromatosis (NF) with significant intra-sample variability,
including differences observed between those who participated in the intervention versus
those who did not. We suspect that there will be subgroups of patients who have increases in
attention difficulties and executive function deficits over time, another subgroup will have
decreases in attention difficulties and executive function deficits over time, and a subgroup
who will have stable patterns across six months.
STUDY DESIGN
Approach:
Overview of Study Design: The current design is a pilot feasibility study comprising a
randomized controlled trial involving patients ages 6 to 16 years (n=50) who are diagnosed
with NF and followed by the comprehensive multidisciplinary NF clinic (Dr. Wang, Director;
Dr. Rohan, Co-Director). The proposed study will contribute to new knowledge by describing
trajectories of neurocognitive factors over time; as well as, examining the preliminary
feasibility and effectiveness of a single-session intervention on improving psychological and
neurocognitive factors such as attention and executive functioning.
This comprehensive measurement approach provides a powerful tool to address important
scientific and clinically relevant questions: 1) What are the patterns of neurocognitive
symptom presentation in pediatric NF?; and, how do these symptoms relate to psychosocial and
health outcomes over time?; 2) How might patients and families benefit from receiving
psychoeducation about neurocognitive, executive, and psychological functioning in patients
with NF; as well as receiving tailored recommendations based on results of neurocognitive
assessments? The use of multiple measures of neurocognitive functioning (multiple reporters
and objective and subjective assessment methods), standardized questionnaires, and a
single-session intervention in a pilot feasibility study of patients' ages 6 to 16 years is
an innovative approach that has potential clinical utility and impact. For example,
identification of neurocognitive and executive functioning deficits from multiple informants
may allow development of more precise interventions in different settings for children (e.g.,
school, home). This approach also affords the opportunity to identify risk and protective
factors in living with a chronic illness and to develop preventative and therapeutic
interventions that can be delivered at the point of care to minimize barriers and maximize
facilitators associated across the illness trajectory for this vulnerable patient population.
Study Procedures Recruitment and Data Collection: Eligible families seen in the Pediatric NF
Clinic at VCU will be invited to participate via telephone or in person (depending on timing
of clinic visit). Interested patients and parents/caregivers will provide informed consent,
parental permission, and assent prior to data collection. Consented participants will be
randomly allocated to the intervention (n=25) or control (n=25) groups via a computerized
randomization program.
Data collection will occur at four timepoints: baseline (T0), 3-months (T1), 6-months (T2),
and 7-months (T3). At T0 through T2, patients will complete standardized assessment batteries
for neurocognitive functioning; and, patients, parents, and teachers will complete
standardized questionnaires. All measures used within the present study are well validated
and can be administered longitudinally within these time intervals with acceptable
test-retest reliability. At T3, families in the intervention group will complete
semi-structured qualitative interviews about the feasibility, acceptability, and desirability
of the intervention.
