Clinical trial NCT06262113: A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1
Official title: A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Neurofibromatosis 1
Keywords: Neurofibromatosis
NF1
Guideline
Healthcare
PCP
Parent
Pediatric
Primary Care
Annual Care
Physical Exam
Well-Child
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: Patient-Centered Outcomes Research Institute
First posted: 2024-02-15
Enrollment: 360
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: HEALTH_SERVICES_RESEARCH

Brief summary: The goal of this fully decentralized, randomized controlled trial is to compare the efficacy of two educational interventions for individuals with Neurofibromatosis 1 (NF1). The primary objective of the study is to determine which intervention leads to higher rates of evidenced-based health screenings for NF1 patients in primary care settings.

Adults with NF1 and parents/guardians of children with NF1 from across the U.S. who do not go to a specialized NF clinic and who have an upcoming annual wellness visits (e.g. an annual physical, a well-child visit, etc.) scheduled with a primary care provider (PCP) are eligible to enroll in the study. To see if you might be eligible, fill out a prescreening survey here: https://redcap.link/mynfguide

Detailed description: Background: The majority of individuals with Neurofibromatosis 1 (NF1) in the United States lack access to specialized NF1 clinics and consequently don't receive care aligned with national recommendations. To address this gap in care, researchers are evaluating two interventions to determine which one helps people get recommended NF1-related health screenings at their annual wellness visit with a primary care provider.

Methods: Participants (adult patients or parents) who enroll in the study will complete baseline survey assessments online before their PCP visit and then be randomized to one of two groups. Both groups will be given letters, one for themselves and one for their clinician, that describe NF1 care recommendations. After attending their annual wellness visit, all participants will be asked to complete an online follow-up survey. A small subsample of participants will also be asked to do a virtual qualitative interview. No visits to Massachusetts General Hospital are required for this study.

Eligibility criteria: Adult Inclusion Criteria:

* Currently lives in the United States (including Puerto Rico and other United States territories)
* Has a clinical diagnosis of neurofibromatosis 1
* Does not attend an NF clinic within the Children's Tumor Foundation NF Clinic Network
* Has an in-person, well-person visit with a primary care provider scheduled within 3 months of their consent
* Speaks English or Spanish

Parent/Guardian of a Child with NF1 Inclusion Criteria:

* Currently lives in the United States (including Puerto Rico and other United States territories)
* Cares for a child (age \<18 years) with a clinical diagnosis of neurofibromatosis 1
* Does not attend an NF clinic within the Children's Tumor Foundation NF Clinic Network
* Their child has an in-person, well-person visit with a primary care provider scheduled within 3 months of their consent
* Speaks English or Spanish

Exclusion Criteria:

* Only one person per household may participate in the study
* Unwilling or unable to give informed consent

Primary outcomes:
measure: Receipt of Recommended NF1 Health Screenings
timeFrame: 2 weeks after PCP visit
Secondary outcomes:
measure: Patient Activation Measure®
timeFrame: At baseline and 2 weeks after PCP visit
measure: Consumer Assessment of Healthcare Providers and Systems (CAHPS®) Clinician and Group Survey Version 4.0 (Beta): Rating of the Visit
timeFrame: 2 weeks after PCP visit
measure: Consumer Assessment of Healthcare Providers and Systems (CAHPS®) Clinician and Group Survey Version 4.0 (Beta): How Well Providers Communicate With Patients Subscale
timeFrame: 2 weeks after PCP visit
measure: Consumer Assessment of Healthcare Providers and Systems (CAHPS®) Clinician and Group Survey Version 4.0 (Beta): Providers' Use of Information to Coordinate Patient Care Subscale
timeFrame: 2 weeks after PCP visit
Other outcomes:
Locations:
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
Study officials:
affiliation: Massachusetts General Hospital
name: Vanessa Merker, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Massachusetts General Hospital
name: Scott Plotkin, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Merker VL, Dai A, Radtke HB, Knight P, Jordan JT, Plotkin SR. Increasing access to specialty care for rare diseases: a case study using a foundation sponsored clinic network for patients with neurofibromatosis 1, neurofibromatosis 2, and schwannomatosis. BMC Health Serv Res. 2018 Aug 29;18(1):668. doi: 10.1186/s12913-018-3471-5.
citation: Merker VL, Knight P, Radtke HB, Yohay K, Ullrich NJ, Plotkin SR, Jordan JT. Awareness and agreement with neurofibromatosis care guidelines among U.S. neurofibromatosis specialists. Orphanet J Rare Dis. 2022 Feb 10;17(1):44. doi: 10.1186/s13023-022-02196-x.