Neurofibromatosis (NF) Registry Portal

NCT ID: NCT01885767

Last Updated: 2023-08-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

20000 participants

Study Classification

OBSERVATIONAL

Study Start Date

2012-06-30

Study Completion Date

2050-06-30

Brief Summary

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The NF Registry is a database of patient-reported symptoms, treatments, and experiences with their neurofibromatosis disease. It is a contact registry to relay clinical trial opportunities to targeted patient subgroups, and to supply de-identified disease data to researchers. It has the potential to become a natural history resource.

Detailed Description

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Patients and parents of patients will be made aware of the Neurofibromatosis (NF) Registry through various non-commercial information sources such as the Children's Tumor Foundation (CTF) website, CTF-affiliated NF clinics, social media, CTF educational and fundraising events, and other nonprofit organizations and foundations such as the National Organization for Rare Diseases (NORD) and social media.

The NF Registry will be accessed by individual adult (over age 18) subjects via a web-based patient portal. The portal contains an IRB-approved informed consent form. Following consent, the registrant creates an account which is activated after email confirmation. An account can be created by an adult patient with the disorder, or by the parent or guardian of a child with the disorder. Account creators are required to enter identifiable contact and demographic data.

After the account is created, the account owner enrolls themselves or a minor family member (or both) and completes an on-line survey. There are separate surveys for NF1, NF2, and Schwannomatosis. The surveys ask about about the affected individual's medical and family history of the disease, testing and diagnosis, clinical manifestations (e.g., tumor types and locations) interventions and therapies, and quality of life. The account holder chooses whether to receive emails from the Registry with information about relevant clinical trials and studies for which they may be eligible.

Participant's responses are used to compile charts and graphics of de-identified aggregate data. Registered patients may view this data. Researchers may apply to our Data Use Committee for access to de-identified data or for subject recruitment emails to be sent to specific patient subgroups. Data capture and security for the NF Registry is done under contract by OpenApp (Dublin, Ireland), a web-based patient opt-in registry provider.Participants will be asked to update their information at least once a year. Their information will be stored in the NF Registry for an indefinite period of time. This longitudinal study is intended as a resource for patients and researchers. There is no specific outcome measure or anticipated endpoint.

Conditions

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Neurofibromatosis 1 Neurofibromatosis 2 Schwannomatosis

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

OTHER

Study Groups

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NF1

Patients meeting clinical and/or genetic criteria for Neurofibromatosis 1

No interventions assigned to this group

NF2

Patients meeting clinical and/or genetic criteria for Neurofibromatosis 2

No interventions assigned to this group

SchW

Patients meeting clinical and/or genetic criteria for Schwannomatosis

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

* Diagnosed with NF1
* Diagnosed with NF2
* Diagnosed with Schwannomatosis

Exclusion Criteria

* Failure to complete account registration
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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The Children's Tumor Foundation

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Kate Kelts, B.S.N.

Role: PRINCIPAL_INVESTIGATOR

The Children's Tumor Foundation

Locations

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Children's Tumor Fundation

New York, New York, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Kate Kelts, B.S.N.

Role: CONTACT

646-738-8567

Annette Bakker, Ph.D.

Role: CONTACT

212-344-7029

Related Links

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Other Identifiers

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CTF001

Identifier Type: -

Identifier Source: org_study_id

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