Plexiform NeurofibromasNCT00006435
Study of Plexiform Neurofibromas in Neurofibromatosis Type 1
An observational study of Plexiform Neurofibromas and NF1, run by National Cancer Institute (NCI) at 1 site in United States.
Trial summary
Background: -Plexiform neurofibromas in patients with NF1 are a significant cause of morbidity but little is known about the natural history of these lesions. Objectives: The purpose of this study is to monitor the natural history of plexiform neurofibromas and to evaluate the usefulness of volumetric MRI tumor… Read more about this study
- Who is it for?
- Ages Up to 100 years
- Conditions the study is for
- Plexiform Neurofibromas, NF1
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 18 May 2001to 1 May 2020
- 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 Up to 100 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: A total of 300 plexiform neurofibromas will be studied, consisting of 100 tumors in the head/neck and trunk/limbs (externally visible and internal)
Who may be able to join
- Diagnosis of Neurofibromatosis: All study subjects will fulfill two or more of the diagnostic criteria listed below for NF1.
- Six or more caf(SqrRoot)(Copyright)-au-lait macules
- 1.5cm or larger in postpubertal individuals
- 0.5 cm or larger in prepubertal individuals
- Two or more neurofibromas of any type or 1 or more plexiform neurofibroma
- Freckling in the axilla or groin
Show all 18Show fewer
- Optic glioma (tumor of the optic pathway)
- Two or more Lisch nodules (benign iris hamartomas)
- A distinctive bony lesion
- Dysplasia of the sphenoid bone
- Dysplasia or thinning of long bone cortex
- A first degree relative with NF-1
- Plexiform Neurofibroma: A plexiform neurofibroma fulfilling entry criteria for the study will be defined as a diffuse soft tissue or nerve enlargement in a patient with NF1 that is causing, or has potential to cause, disfigurement or functional disability.
- Distribution of Plexiform Neurofibromas by site: A total of 300 plexiform neurofibromas will be studied, consisting of 100 tumors in the following three groups (based on region of maximal involvement):
- Head and Neck
- Trunk and Limbs (externally visible)
- Trunk and Limbs (internal) [spinal plexiform neurofibromas involve two or more levels with connection between the levels or extending laterally along the nerve]
- Subject Ascertainment: Study subjects will be ascertained at any of the participating clinical centers. It is expected that these will include subjects already followed in these clinics, as well as newly diagnosed patients
Who cannot join
- Presence of metallic implant(s) that will make the patient unable to have MRI studies
- Presence of medical or psychological condition that will make the patient unable to tolerate MRI studies or anesthesia (if needed)
- Inability to image tumor or define tumor margins by MRI (which may be determined after the initial study)
- Failure to obtain initial MRI within 60 days of enrollment
- Previous radiation therapy to site of plexiform neurofibroma
- Surgery involving the plexiform neurofibroma (excluding biopsy) within a six month period before enrollment
- Current antineoplastic therapy
- Entry of more than one member of the same family into the study is not permitted
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: Natural History of Plexiform in Neurofibromatosis Type I
Background:
-Plexiform neurofibromas in patients with NF1 are a significant cause of morbidity but little is known about the natural history of these lesions.
Read more
Objectives:
- The purpose of this study is to monitor the natural history of plexiform neurofibromas and to evaluate the usefulness of volumetric MRI tumor measurements in this disease.
- Other goals of the study are to provide a body of normative data on the growth rate of plexiform neurofibromas and to establish a tissue repository and pathology review center to allow future studies of the pathogenesis of neurofibromas and clinical trials of potential therapeutic agents.
Design
- This study is coordinated by Dr. Bruce Korf, and was initiated when he was at the Partners Center for Human Genetics, Boston, MA.
Read the detailed description
Background:
-Plexiform neurofibromas in patients with NF1 are a significant cause of morbidity but little is known about the natural history of these lesions.
Objectives:
- The purpose of this study is to monitor the natural history of plexiform neurofibromas and to evaluate the usefulness of volumetric MRI tumor measurements in this disease.
- Other goals of the study are to provide a body of normative data on the growth rate of plexiform neurofibromas and to establish a tissue repository and pathology review center to allow future studies of the pathogenesis of neurofibromas and clinical trials of potential therapeutic agents.
Design
- This study is coordinated by Dr. Bruce Korf, and was initiated when he was at the Partners Center for Human Genetics, Boston, MA.
- Conditions
- Plexiform Neurofibromas, NF1
- Keywords
- Tissue Repository, Growth Rate, Volumetric MRI
- Medical subject headings
- Neurofibroma, Plexiform
What is being measured
The primary outcome is the main question the study is designed to answer.
Monitor natural history of plexiform neurofibromas
Monitor natural history of plexiform neurofibromas
Measured: 3 years
Usefulness of volumetric MRI measurements
Usefulness of volumetric MRI measurements
Measured: 4 years
Secondary outcomes (2)
Establish tissue repository & pathology reviews center
Measured: 3 years
Body of data regarding growth rate of plexiform neurofibromas
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Nov 2000 (estimated)
- Study start18 May 2001
- Primary completion7 Sep 2007
- Study completion1 May 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Plexiform NeurofibromasStudies 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
- Prospective
- Enrolment
- 24 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
- NCT00006435
- Sponsor's protocol ID
- 010027
- Other IDs
- 01-C-0027
- Status last verified
- May 2020
- First submitted
- 3 Nov 2000
- Last updated
- 4 May 2020
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Natural History of Plexiform in Neurofibromatosis Type I
- ClinicalTrials.gov ID
- NCT00006435
- Acronym / sponsor ID
- 010027
- Conditions
- Plexiform Neurofibromas, NF1
- Keywords
- Tissue Repository, Growth Rate, Volumetric MRI
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- National Cancer Institute (NCI)US National Institutes of Health
- Study start
- 18 May 2001 (actual)
- Primary completion
- 7 Sep 2007 (actual)
- Study completion
- 1 May 2020 (actual)
- First posted
- 6 Nov 2000 (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.
Background:
-Plexiform neurofibromas in patients with NF1 are a significant cause of morbidity but little is known about the natural history of these lesions.
Objectives:
- The purpose of this study is to monitor the natural history of plexiform neurofibromas and to evaluate the usefulness of volumetric MRI tumor measurements in this disease.
- Other goals of the study are to provide a body of normative data on the growth rate of plexiform neurofibromas and to establish a tissue repository and pathology review center to allow future studies of the pathogenesis of neurofibromas and clinical trials of potential therapeutic agents.
Design
- This study is coordinated by Dr. Bruce Korf, and was initiated when he was at the Partners Center for Human Genetics, Boston, MA.
Detailed description
Background:
-Plexiform neurofibromas in patients with NF1 are a significant cause of morbidity but little is known about the natural history of these lesions.
Objectives:
- The purpose of this study is to monitor the natural history of plexiform neurofibromas and to evaluate the usefulness of volumetric MRI tumor measurements in this disease.
- Other goals of the study are to provide a body of normative data on the growth rate of plexiform neurofibromas and to establish a tissue repository and pathology review center to allow future studies of the pathogenesis of neurofibromas and clinical trials of potential therapeutic agents.
Design
- This study is coordinated by Dr. Bruce Korf, and was initiated when he was at the Partners Center for Human Genetics, Boston, MA.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- Up to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- A total of 300 plexiform neurofibromas will be studied, consisting of 100 tumors in the head/neck and trunk/limbs (externally visible and internal)
Key inclusion criteria (18 listed)
- Diagnosis of Neurofibromatosis: All study subjects will fulfill two or more of the diagnostic criteria listed below for NF1.
- Plexiform Neurofibroma: A plexiform neurofibroma fulfilling entry criteria for the study will be defined as a diffuse soft tissue or nerve enlargement in a patient with NF1 that is causing, or has potential to cause, disfigurement or functional disability.
- Distribution of Plexiform Neurofibromas by site: A total of 300 plexiform neurofibromas will be studied, consisting of 100 tumors in the following three groups (based on region of maximal involvement):
- Subject Ascertainment: Study subjects will be ascertained at any of the participating clinical centers. It is expected that these will include subjects already followed in these clinics, as well as newly diagnosed patients
Key exclusion criteria (8 listed)
- Presence of metallic implant(s) that will make the patient unable to have MRI studies
- Presence of medical or psychological condition that will make the patient unable to tolerate MRI studies or anesthesia (if needed)
- Inability to image tumor or define tumor margins by MRI (which may be determined after the initial study)
- Failure to obtain initial MRI within 60 days of enrollment
- Previous radiation therapy to site of plexiform neurofibroma
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
Monitor natural history of plexiform neurofibromas
Time frame: 3 years
Monitor natural history of plexiform neurofibromas
Usefulness of volumetric MRI measurements
Time frame: 4 years
Usefulness of volumetric MRI measurements
2 secondary outcomes
Establish tissue repository & pathology reviews center
Time frame: 3 years
Body of data regarding growth rate of plexiform neurofibromas
Time frame: 3 years
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 4 May 2020.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
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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 Plexiform Neurofibromas and NF1, 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 18 inclusion and 8 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 4 May 2020. 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?
Monitor natural history of plexiform neurofibromas (time frame: 3 years). The study lists 2 primary outcomes in all. 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 4 May 2020.