Neurofibromatosis 1NCT04774289
Incidence of Malignant Peripheral Nerve Sheath Tumor (MPNST) Development in Participants With Neurofibromatosis Type 1 (NF1) Receiving and Not Receiving Medical Therapies Directed at Plexiform Neurofibromas (PN)
An observational study of Neurofibromatosis 1 and Peripheral Nerve Neoplasms, Malignant, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
Background: NF1 is a genetic syndrome. Tumors appear early in life. Many people with NF1 develop PN. These tumors can become an aggressive cancer called MPNST. People with MPNST may benefit from treatment with a MEK inhibitor (MEKi). Read more about this study
- Who is it for?
- Ages 1 month and older
- Conditions the study is for
- Neurofibromatosis 1, Peripheral Nerve Neoplasms, Malignant
- How many people
- 296 participants
- Where
- 1 siteUnited States
- Study dates
- 24 Feb 2021to 8 Dec 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
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 1 month and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: primary clinical
Who may be able to join
- All participants seen at the NIH from 1/1/1998 to 1/1/2020 with a clinical or laboratory diagnosis of NF1
- Participants beyond the neonatal period (4 weeks).
Who cannot join
- -All participants seen at the NIH from 1/1/1998 to 1/1/2020 with a clinical or laboratory diagnosis of NF1 who opted out of future use.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Cancer Institute (NCI)
Bethesda, Maryland, 20892
About this study
Official title: Incidence of Malignant Peripheral Nerve Sheath Tumors Development in Patients With Neurofibromatosis Type 1 Receiving and Not Receiving Medical Therapies Directed at Plexiform Neurofibromas
Background:
NF1 is a genetic syndrome. Tumors appear early in life. Many people with NF1 develop PN. These tumors can become an aggressive cancer called MPNST. People with MPNST may benefit from treatment with a MEK inhibitor (MEKi). Researchers want to learn if there is an increased risk of MPNST formation from MEKi treatment in people with NF1. To do this, they will review data that has been collected in NIH NF1 studies.
Read more
Objective:
To describe the characteristics of people who have taken part in NF1 studies at NIH and to compare the risk of MPNST formation in those treated with MEKi or other PN-directed treatment.
Eligibility:
People with NF1 who were seen at NIH from Jan. 1, 1998, to Jan. 1, 2020.
Design:
Participants medical records will be reviewed. Participants who opted out of future use of their data will not be included.
Demographic data, like sex, race, and date of birth, will be collected.
Data about MEKi and non-MEKi treatments will be collected.
Clinical data, such as surgery and treatment details, will be collected.
The differences between all participants who were seen at NIH for any NF1 related study will be compared. Participants will be put into 4 groups:
History of MEKi therapy
Treatment with tumor directed therapy other than MEKi
Treatment with both MEKi and non-MEKi tumor directed therapies
No tumor directed medical therapy
Participants with NF1 who were treated for PN with either a MEKi treatment or a non-MEKi treatment will also be compared.
The study will last for 3 to 6 months.
Read the detailed description
Study Description: This is a retrospective review that includes all patients with NF1 enrolled on natural history and treatment trials at the National Cancer Institute (NCI) from 1/1/1998 to 1/1/2020, excluding patients referred for treatment of MPNST.
Objective: To describe the clinical and demographic characteristics of NF1 study participants who come for evaluation at the NCI and to compare the risk of MPNST formation in patients treated with MEK 1/2 inhibitor (MEKi) or other PN-directed treatment.
Study Population: All participants with NF1 seen at the NIH from 1/1/1998 to 1/1/2020
Description of Sites/Facilities conducting research: This is a single site study initiated by the Pediatric Oncology Branch at the National Cancer Institute
Study Duration: Data abstraction to completion of data analyses will take 3-6 months.
- Keywords
- Retrospective Review, Natural History, genetic tumor predisposition syndrome
- Medical subject headings
- Neurofibromatosis 1, Peripheral Nervous System Neoplasms
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 |
|---|---|---|
| 1 | Participants with NF1 seen at the NIH from 1/1/1998 to 1/1/2020 |
What is being measured
The primary outcome is the main question the study is designed to answer.
risk of MPNST formation
descriptive statistics and frequency distribution of selected variables
Measured: ongoing
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Mar 2021
- Study start24 Feb 2021
- Primary completion1 Mar 2022
- Study completion8 Dec 2022
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.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- 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
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 296 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Brigitte C Widemann, M.D., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04774289
- Sponsor's protocol ID
- 10000271
- Other IDs
- 000271-C
- Status last verified
- 24 Jun 2024
- First submitted
- 26 Feb 2021
- Last updated
- 19 Sep 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Incidence of Malignant Peripheral Nerve Sheath Tumors Development in Patients With Neurofibromatosis Type 1 Receiving and Not Receiving Medical Therapies Directed at Plexiform Neurofibromas
- ClinicalTrials.gov ID
- NCT04774289
- Acronym / sponsor ID
- 10000271
- Keywords
- Retrospective Review, Natural History, genetic tumor predisposition syndrome
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 296 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 24 Feb 2021 (actual)
- Primary completion
- 1 Mar 2022 (actual)
- Study completion
- 8 Dec 2022 (actual)
- First posted
- 1 Mar 2021 (actual)
- 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.
Background:
NF1 is a genetic syndrome. Tumors appear early in life. Many people with NF1 develop PN. These tumors can become an aggressive cancer called MPNST. People with MPNST may benefit from treatment with a MEK inhibitor (MEKi). Researchers want to learn if there is an increased risk of MPNST formation from MEKi treatment in people with NF1. To do this, they will review data that has been collected in NIH NF1 studies.
Objective:
To describe the characteristics of people who have taken part in NF1 studies at NIH and to compare the risk of MPNST formation in those treated with MEKi or other PN-directed treatment.
Eligibility:
People with NF1 who were seen at NIH from Jan. 1, 1998, to Jan. 1, 2020.
Design:
Participants medical records will be reviewed. Participants who opted out of future use of their data will not be included.
Demographic data, like sex, race, and date of birth, will be collected.
Data about MEKi and non-MEKi treatments will be collected.
Clinical data, such as surgery and treatment details, will be collected.
The differences between all participants who were seen at NIH for any NF1 related study will be compared. Participants will be put into 4 groups:
History of MEKi therapy
Treatment with tumor directed therapy other than MEKi
Treatment with both MEKi and non-MEKi tumor directed therapies
No tumor directed medical therapy
Participants with NF1 who were treated for PN with either a MEKi treatment or a non-MEKi treatment will also be compared.
The study will last for 3 to 6 months.
Detailed description
Study Description: This is a retrospective review that includes all patients with NF1 enrolled on natural history and treatment trials at the National Cancer Institute (NCI) from 1/1/1998 to 1/1/2020, excluding patients referred for treatment of MPNST.
Objective: To describe the clinical and demographic characteristics of NF1 study participants who come for evaluation at the NCI and to compare the risk of MPNST formation in patients treated with MEK 1/2 inhibitor (MEKi) or other PN-directed treatment.
Study Population: All participants with NF1 seen at the NIH from 1/1/1998 to 1/1/2020
Description of Sites/Facilities conducting research: This is a single site study initiated by the Pediatric Oncology Branch at the National Cancer Institute
Study Duration: Data abstraction to completion of data analyses will take 3-6 months.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | – | –Participants with NF1 seen at the NIH from 1/1/1998 to 1/1/2020 |
Who may qualify
- Ages
- 1 month and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- primary clinical
Key inclusion criteria (2 listed)
- All participants seen at the NIH from 1/1/1998 to 1/1/2020 with a clinical or laboratory diagnosis of NF1
- Participants beyond the neonatal period (4 weeks).
Key exclusion criteria (1 listed)
- -All participants seen at the NIH from 1/1/1998 to 1/1/2020 with a clinical or laboratory diagnosis of NF1 who opted out of future use.
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
risk of MPNST formation
Time frame: ongoing
descriptive statistics and frequency distribution of selected variables
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
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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. This study is not recruiting at the moment.
Brigitte C Widemann, M.D. principal investigator
National Cancer Institute (NCI)
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 Peripheral Nerve Neoplasms, Malignant, run by National Cancer Institute (NCI) 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 2 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: finished, as of the sponsor's last update on 19 Sep 2024. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
risk of MPNST formation (time frame: ongoing). 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 19 Sep 2024.