Neurofibromatosis (NF) Registry Portal
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT01885767|
Recruitment Status : Recruiting
First Posted : June 25, 2013
Last Update Posted : February 4, 2019
|Condition or disease|
|Neurofibromatosis 1 Neurofibromatosis 2 Schwannomatosis|
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, 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 subjects via a web-based patient portal. The portal first provides informed consent information. Following consent, the registrant creates an account by choosing a unique username and password. 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, a patient profile can be completed via on-line questionnaires. There are separate questionnaires for NF1, NF2, and Schwannomatosis. The questionnaires 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. They also ask permission from the patient or his or her parent or guardian to be contacted by email (via CTF) in regard to relevant clinical trials and studies.
Participant's responses are used to compile charts and graphics of de-identified aggregate data. Registered patients may view this data.
Researchers may request access to de-identified data. They may also request that CTF send emails of study recruitment materials to specific patient subgroups.
Data capture and security for the NF Registry is done under contract by Invitae Corp. (San Francisco, CA), a web-based patient opt-in registry provider that also hosts 250 other rare disease registries.
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.
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||10000 participants|
|Target Follow-Up Duration:||50 Years|
|Official Title:||Neurofibromatosis (NF) Registry Portal Funded by Children's Tumor Foundation|
|Study Start Date :||June 2012|
|Estimated Primary Completion Date :||June 2022|
|Estimated Study Completion Date :||June 2050|
Patients meeting clinical and/or genetic criteria for Neurofibromatosis 1
Patients meeting clinical and/or genetic criteria for Neurofibromatosis 2
Patients meeting clinical and/or genetic criteria for Schwannomatosis
- to create a natural history of NF1, NF2, and schwannomatosis [ Time Frame: final report in 2050 with descriptive statistics ]patients will input medical information and treatment information about their NF and update at least yearly in an ongoing natural history study
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01885767
|Contact: Pamela B Knight, M.S.||212-344-6633 ext firstname.lastname@example.org|
|Contact: Annette Bakker, Ph.D.||212-344-6633 ext email@example.com|
|United States, New York|
|Children's Tumor Fundation||Recruiting|
|New York, New York, United States, 10005|
|Principal Investigator: Pamela B Knight, M.S.|
|Principal Investigator:||Pamela B Knight, M.S.||The Children's Tumor Foundation|