A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1

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

Sex

All

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

• BACKGROUNDMerker VL, Ma Y, Chibnik LB, Radtke HB, Kelts K, Yohay K, Ullrich NJ, Plotkin SR, Jordan JT. Self-reported access to specialty clinics and receipt of health surveillance among U.S. patients with neurofibromatosis 1: a national survey. Orphanet J Rare Dis. 2025 Apr 16;20(1):185. doi: 10.1186/s13023-025-03677-5. PMID 40241092
• BACKGROUNDMerker 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. PMID 30157837
• BACKGROUNDMerker 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. PMID 35144646