Neurofibromatosis 1NCT01885767
The NF Registry Sponsored by the Children's Tumor Foundation
An observational study of Neurofibromatosis 1 and Neurofibromatosis 2, run by The Children's Tumor Foundation at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Neurofibromatosis 1, Neurofibromatosis 2, Schwannomatosis
- How many people
- 20,000 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2012to Jun 2050
- How long will I take part?
- 50 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients affected with NF1, NF2-related schwannomatosis, or Schwannomatosis
Who may be able to join
- Diagnosed with NF1
- Diagnosed with NF2-related schwannomatosis
- Diagnosed with Schwannomatosis
Who cannot join
- Failure to complete account registration and consent
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Children's Tumor Fundation
New York, New York, 10017
Kate Kelts, B.S.N.
About this study
Official title: The NF Registry Sponsored by Children's Tumor Foundation
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.
Read the detailed description
Patients and parents of patients will be made aware of the 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-related schwannomatosis, and all other forms of 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 the NF Registry Advisory Board 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 the National Organization for Rare Disease (NORD) using their IAMRARE web-based platform.
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.
- Keywords
- Neurofibromatosis, NF, Neurofibromatosis 1, NF1, Neurofibromatosis 2, NF2, Schwannomatosis, NF2-related schwannomatosis, LZTR1, SMARCB1
- Medical subject headings
- Neurofibromatosis 1, Neurofibromatosis 2, Schwannomatosis, Neurofibromatoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| NF1 | Patients meeting clinical and/or genetic criteria for Neurofibromatosis 1 | |
| SchW | Patients meeting clinical and/or genetic criteria for Schwannomatosis | |
| NF2-related schwannomatosis | Individuals diagnosis or suspected to have NF2-related schwannomatosis |
What is being measured
The primary outcome is the main question the study is designed to answer.
To create a natural history of NF1, NF2-related schwannomatosis, and schwannomatosis (all other forms).
patients will input medical information and treatment information about their NF and update at least yearly in an ongoing natural history study
Measured: final report in 2050 with descriptive statistics
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jun 2013 (estimated)
- Study startJun 2012
- Primary completionJun 2030 (estimated)
- Study completionJun 2050 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Other
- Enrolment
- 20,000 participants (estimated)
- Target duration
- 50 Years
Sponsor and investigators
- Lead sponsor
- The Children's Tumor Foundation
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Kate Kelts, B.S.N., Principal investigator, The Children's Tumor Foundation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01885767
- Sponsor's protocol ID
- CTF001
- Status last verified
- Oct 2026
- First submitted
- 22 May 2013
- Last updated
- 22 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No: deidentified data only may be shared
Study at a glance
From the registry record, last updated by the sponsor on 22 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The NF Registry Sponsored by Children's Tumor Foundation
- ClinicalTrials.gov ID
- NCT01885767
- Acronym / sponsor ID
- CTF001
- Keywords
- Neurofibromatosis, NF, Neurofibromatosis 1, NF1, Neurofibromatosis 2, NF2, Schwannomatosis, NF2-related schwannomatosis
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 20,000 participants (estimated)
- Lead sponsor
- The Children's Tumor FoundationUniversity, hospital or other
- Study start
- Jun 2012
- Primary completion
- Jun 2030 (estimated)
- Study completion
- Jun 2050 (estimated)
- First posted
- 25 Jun 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Patients and parents of patients will be made aware of the 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-related schwannomatosis, and all other forms of 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 the NF Registry Advisory Board 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 the National Organization for Rare Disease (NORD) using their IAMRARE web-based platform.
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 design
- Observational model
- Case only
- Time perspective
- Other
- Target follow-up
- 50 Years
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| NF1 | – | –Patients meeting clinical and/or genetic criteria for Neurofibromatosis 1 |
| SchW | – | –Patients meeting clinical and/or genetic criteria for Schwannomatosis |
| NF2-related schwannomatosis | – | –Individuals diagnosis or suspected to have NF2-related schwannomatosis |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients affected with NF1, NF2-related schwannomatosis, or Schwannomatosis
Key inclusion criteria (3 listed)
- Diagnosed with NF1
- Diagnosed with NF2-related schwannomatosis
- Diagnosed with Schwannomatosis
Key exclusion criteria (1 listed)
- Failure to complete account registration and consent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
To create a natural history of NF1, NF2-related schwannomatosis, and schwannomatosis (all other forms).
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Oct 2026.
- NF RegistryLink provided by the sponsor.
- Individual participant dataSharing planned: No. deidentified data only may be shared
Related trials for Neurofibromatosis 1
Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
RecruitingPhase 3
A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
Low Grade Astrocytoma, Low Grade Glioma
RecruitingPhase 1
Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...
Neurofibroma, Plexiform, Precursor Cell Lymphoblastic Leukemia-Lymphoma
Active, not recruitingPhase 2
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
RecruitingObservational study
Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)
Neurofibromatosis 1, Nerve Sheath Neoplasms
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Kate Kelts, B.S.N. principal investigator
The Children's Tumor Foundation
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Neurofibromatosis 1 and Neurofibromatosis 2, run by The Children's Tumor Foundation at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kate Kelts, B.S.N. (646-738-8567, kkelts@ctf.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To create a natural history of NF1, NF2-related schwannomatosis, and schwannomatosis (all other forms). (time frame: final report in 2050 with descriptive statistics). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Oct 2026.