Intervention Effectiveness on the Neurocognitive Functioning of Children and Adolescents With Neurofibromatosis Type 1

NCT05377008 | Completed

• 1 other identifier: HM20024159
• Study Type: interventional
• Target: 38
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Neurocognitive Deficit; Mental Health Impairment; Neurofibromatosis 1

Keywords

Neurofibromatosis; Neurocognitive; Intervention

Outcome Measures

Primary Outcomes (2)

• Change in intellectual function

  Intellectual function will be assessed using the Wechsler Intelligence Scale for Children (WISC) adapted to the age of the child. The child is asked to perform a variety of intellectual tasks. Overall and subscale scores are calculated by summing the number of correct answers. Higher scores indicate better intellectual function.

  Baseline, 3 months, 6 months

• Change in neurocognitive function

  Neurocognitive function will be assessed using the NIH Toolbox Cognitive Test Battery (NCTB). The NCTB is a standardized set of brief measures. Tasks are completed on a tablet and scores can be evaluated separately or as composite scores. Scores are normalized with a score of 100 being average, lower scores indicating lower neurocognitive function, and higher scores indicating higher cognitive function.

  Baseline, 3 months, 6 months

Secondary Outcomes (1)

• Satisfaction with intervention

  7 months

Study Arms (2)

Intervention

EXPERIMENTAL

Parents in the intervention arm will receive one single-session intervention one month after completion of baseline measures. They will also complete a feasibility exit interview one month after completion of their data collection period (i.e., at 7 months).

Behavioral: OPTIMAL-SSI

Control

NO INTERVENTION

Families in the control arm will complete measures at the exact same timepoints as those in the intervention arm but will not receive the single session intervention and will not complete the feasibility exit interview. They will receive the same information as the intervention arm via an education manual at the completion of their data collection period.

Interventions

OPTIMAL-SSI BEHAVIORAL

The purpose of the intervention feedback session will be twofold: 1) to provide the results of the previous assessment, and 2) to provide general psychoeducation on psychological and neurocognitive functioning and tailored cognitive and behavioral interventions based on the results in order to reduce the impact of psychological and neurocognitive deficits.

Intervention

Eligibility Criteria

• Age: 6 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Patients are eligible if they are English-speaking children and adolescents between the ages of 6 to 16 years diagnosed with NF who are currently followed by the Pediatric NF Clinic at VCU. Patients are eligible if they currently have a diagnosis of ADHD or do not have a diagnosis of ADHD.
• At least one parent/caregiver must be actively involved in the patient's care and able to complete study-related questionnaires at baseline, 3-, and 6-months; and the exit interview at 7-months. Parents are eligible if they if they are English-speaking adults 18+ years old and self-identify as the parent or guardian of the patient.

You may not qualify if:

• No subject will be excluded on the basis of gender or ethnicity.

Sponsors & Collaborators

• Virginia Commonwealth University: lead

Study Sites (1)

Children's Hospital of Richmond at Virginia Commonwealth University

Richmond, Virginia, 23219, United States

Location

MeSH Terms

Conditions

Neurofibromatosis 1; Neurofibromatoses

Condition Hierarchy (Ancestors)

Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Jennifer M Rohan, PhD

  Virginia Commonwealth University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, OUTCOMES ASSESSOR
• Masking Details: Patient randomization is assigned after baseline is completed (i.e., the patient and assessor are not aware of random assignment until after the baseline is completed). The Principal Investigator and Clinical Research Coordinator will be the only ones with access to random assignments.
• Purpose: HEALTH SERVICES RESEARCH
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: This is a pilot feasibility study comprising a randomized controlled trial involving 50 families (i.e., parent/guardian AND child ages 6 to 16 years with NF). Consented participants will be randomly allocated to the intervention (n=25) or control (n=25) arms.

Study Record Dates

• First Submitted: May 5, 2022
• First Posted: May 17, 2022
• Study Start: May 13, 2022
• Primary Completion: May 5, 2023
• Study Completion: May 5, 2023
• Last Updated: September 22, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)