Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults with Neurofibromatosis Type 1

Description

The main goal of this protocol is to develop a well-phenotyped genetic biobank to identify genetic variants associated with the heterogeneous clinical presentations of Neurofibromatosis Type 1 (NF1). This will allow for improve understanding of NF1 pathogenesis and more personalized disease management. The investigators will conduct a GWAS analysis to identify common genetic risk variants associated with the development of cutaneous neurofibromas.

Conditions

Neurofibromatosis 1

Study Overview

Study Details

Study overview

The main goal of this protocol is to develop a well-phenotyped genetic biobank to identify genetic variants associated with the heterogeneous clinical presentations of Neurofibromatosis Type 1 (NF1). This will allow for improve understanding of NF1 pathogenesis and more personalized disease management. The investigators will conduct a GWAS analysis to identify common genetic risk variants associated with the development of cutaneous neurofibromas.

Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults with Neurofibromatosis Type 1

Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults with Neurofibromatosis Type 1

Condition
Neurofibromatosis 1
Intervention / Treatment

-

Contacts and Locations

Baltimore

Johns Hopkins University School of Medicine, Baltimore, California, United States, 21218

Redwood City

Stanford University, Redwood City, California, United States, 94063

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Age 40 or older.
  • * NF type 1 diagnosed using clinical criteria.
  • * At least one neurofibroma present at time of enrollment.
  • * Patient able to read and understand consent form (or equivalent translation) and able to give consent.
  • * Patient able and willing to complete all study procedures.

Ages Eligible for Study

40 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Stanford University,

Kavita Sarin, MD PhD, PRINCIPAL_INVESTIGATOR, Stanford University

Jaishri Blakely, MD, PRINCIPAL_INVESTIGATOR, Johns Hopkins University

Carlos Romo, MD, PRINCIPAL_INVESTIGATOR, Johns Hopkins University

Study Record Dates

2025-09-30