- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT06262113
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 scheduled with a primary care provider (PCP) are eligible to enroll in the study.
Study Overview
Status
Conditions
Intervention / Treatment
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 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.
Study Type
Enrollment (Estimated)
Phase
- Not Applicable
Contacts and Locations
Study Contact
- Name: Paulina Arias Hernandez, MSW
- Phone Number: 6177245321
- Email: pahernandez@mgb.org
Study Contact Backup
- Name: Evan Koch, BA
- Phone Number: 6177249574
- Email: eokoch@mgh.harvard.edu
Study Locations
-
-
Massachusetts
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Boston, Massachusetts, United States, 02114
- Massachusetts General Hospital
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
- Adult
- Older Adult
Accepts Healthy Volunteers
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Health Services Research
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Double
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Intervention Arm
|
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
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Experimental: Enhanced Usual Care Arm
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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
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Receipt of Recommended NF1 Health Screenings
Time Frame: 2 weeks after PCP visit
|
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 Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Patient Activation Measure®
Time Frame: At baseline and 2 weeks after PCP visit
|
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
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Consumer Assessment of Healthcare Providers and Systems (CAHPS®) Clinician and Group Survey Version 4.0 (Beta): Rating of the Visit
Time Frame: 2 weeks after PCP 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
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Consumer Assessment of Healthcare Providers and Systems (CAHPS®) Clinician and Group Survey Version 4.0 (Beta): How Well Providers Communicate With Patients Subscale
Time Frame: 2 weeks after PCP visit
|
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
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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
Time Frame: 2 weeks after PCP visit
|
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
|
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Scott Plotkin, MD, PhD, Massachusetts General Hospital
- Principal Investigator: Vanessa Merker, PhD, Massachusetts General Hospital
Publications and helpful links
General Publications
- 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.
- 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.
Study record dates
Study Major Dates
Study Start (Estimated)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Nervous System Diseases
- Neoplasms by Histologic Type
- Neoplasms
- Genetic Diseases, Inborn
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Neoplasms, Nerve Tissue
- Peripheral Nervous System Diseases
- Nervous System Neoplasms
- Heredodegenerative Disorders, Nervous System
- Neoplastic Syndromes, Hereditary
- Nerve Sheath Neoplasms
- Neurocutaneous Syndromes
- Peripheral Nervous System Neoplasms
- Neurofibromatoses
- Neurofibromatosis 1
- Neurofibroma
Other Study ID Numbers
- 2024P000392
- AD-2022C2-24790 (Other Grant/Funding Number: PCORI)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
IPD Sharing Time Frame
IPD Sharing Access Criteria
IPD Sharing Supporting Information Type
- STUDY_PROTOCOL
- SAP
- ICF
- ANALYTIC_CODE
- CSR
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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