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

Neurofibromatosis 1 Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06262113
• Other study ID #: 2024P000392
• Secondary ID: AD-2022C2-24790
• Status: Not yet recruiting
• Phase: N/A
• Start date: November 2024
• Est. completion date: August 2026

Study information

• Verified date: February 2024
• Source: Massachusetts General Hospital
• Contact: Paulina Arias Hernandez, MSW
• Phone: 6177245321
• Email: pahernandez[@]mgb.org
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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 scheduled with a primary care provider (PCP) are eligible to enroll in the study.

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 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 a follow-up survey. A small subsample of participants will also be asked to do a qualitative interview.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 360
• Est. completion date: August 2026
• Est. primary completion date: June 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Neurofibroma
• Neurofibromatoses
• Neurofibromatosis 1

Intervention

Other:
• Letters about NF1 Care (Content Type 1)
Participants will receive two letters about NF1 care, one for themselves and one for their primary care clinician, to read in advance of the patient's annual wellness visit
• Letters about NF1 Care (Content Type 2)
Participants will receive two letters about NF1 care, one for themselves and one for their primary care clinician, to read in advance of the patient's annual wellness visit

Locations

Country	Name	City	State
United States	Massachusetts General Hospital	Boston	Massachusetts

Sponsors (2)

Lead Sponsor	Collaborator
Massachusetts General Hospital	Patient-Centered Outcomes Research Institute

Country where clinical trial is conducted

United States, 

References & Publications (2)

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. — View 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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Receipt of Recommended NF1 Health Screenings	Number of AAP and ACMG recommended health screenings received by the person with NF1 at the annual wellness visit with their PCP, as assessed by patient/parent self-report	2 weeks after PCP visit
Secondary	Patient Activation Measure®	The Patient Activation Measure will be used to assess patient's and parent's self-efficacy managing their or their child's care. The patient form has 13 items and the parent form has 10 items. The measure has scores ranging from 0-100, where higher scores indicate higher patient/parent activation (e.g. self-efficacy in managing your own or your child's healthcare).	At baseline and 2 weeks after PCP visit
Secondary	Consumer Assessment of Healthcare Providers and Systems (CAHPS®) Clinician and Group Survey Version 4.0 (Beta): Rating of the Visit	The CAHPS® Rating of the Visit will be used to assess patient/parent satisfaction with the visit. This rating is a single item scored from 0 to 10, where higher scores indicated a better visit.	2 weeks after PCP visit
Secondary	Consumer Assessment of Healthcare Providers and Systems (CAHPS®) Clinician and Group Survey Version 4.0 (Beta): How Well Providers Communicate With Patients Subscale	The subscale will be used to assess How Well Providers Communicate With Patients. This scale will be scored from 0 to 100, where higher scores indicated better communication with patients.	2 weeks after PCP visit
Secondary	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	The subscale will be used to assess Providers' Use of Information to Coordinate Patient Care. This scale will be scored from 0 to 100, where higher scores indicate better use of information to coordinate care.	2 weeks after PCP visit